Category Archives: Genetic Therapy

As the world haphazardly endeavors to defeat a deadly virus, the pharma industry has found itself charged with delivering a miracle cure in a compressed, unprecedented time frame, no less. But as drug developers swim hard and fast to neutralize COVID-19 with vaccines and therapeutics, they are also working to get other important drugs approved amid looming threats of halts and delays.

Indeed, even as the industry prioritizes everything essential as the pandemic rages, could the current emphasis on speed and efficiency spur a new (and faster) normal for drug development timelines, approvals and regulations? Time will tell, but 2021 is looking to be a big year for the pharma pipeline.

COVID-19 vaccines have been in the spotlight this year, a focus which is only expected to intensify in 2021. While usual vaccine development and approvals take decades, multiple drug developers are aiming to shorten this process to around 18 months. But while Pfizer, Moderna and Johnson & Johnson are among the current frontrunners with novel vaccine approaches, the lack of precedence for both mRNA and adenoviral vector vaccines render them high-risk, high-reward endeavors. With emergency use authorizations (EUAs) expected by year end or early 2021, we should be hearing major Phase III data verdicts for these vaccines around mid-2021.

Meanwhile, significant progress has been made with COVID-19 therapeutics, particularly in the wake of Gileads Veklury (remdesivir) approval in October. While analysts anticipate the drug to bring in around $1.5 billion by the end of 2020, new mixed data and a high price has drawn some skepticism on whether Gilead can sustain its market traction. This may pave the way for other candidates, such as Regenerons antibody cocktail REGN-COV2 or InflaRxs C5a inhibitor IFX-1.

Beyond COVID-19, many promising drugs continue to tick along in late-stage trials and approval discussions. Oncology, ever an essential priority for drug developers, boasts a robust pipeline, led by J&J/Legend Biotechs CAR-T therapy Cilta-cel. Its expected to be approved next year for multiple myeloma, along with Bristol Myers Squibbs direct competitor Ide-cel.

Next year could also be a pivotal year for Iovance, which is set to release data and file for the approval of Lifileucel, potentially the first cell therapy approved for solid tumors. New combination therapies could also offer incremental upside to the current treatment paradigm, with Nektar/Bristol Myers Squibbs bempegaldesleukin/nivolumab combo vying for approval to treat melanoma and other solid tumors.

Meanwhile in neurology, a final verdict on Biogens controversial resurrection of aducanumab for Alzheimers disease a potential $15 billion dollar drug is expected in March 2021. However, a negative vote in early November by the FDA advisory committee doesnt bode well for its chances. Though a future approval hasnt been ruled out, more studies could be a likely scenario. Meanwhile, AbbVie and Biohaven Pharmaceuticals continue to evolve their migraine franchises with 2021 regulatory filings for atogepant and zavegepant, respectively.

Non-COVID immunology drug development continues to be dominated by skin conditions, with UCBs bimekizumab on the cusp of approval for moderate-to-severe psoriasis patients. Similarly, BMS is awaiting additional Phase III data for its oral psoriasis drug BMS-986165, which could be a game-changer, in terms of convenience, for patients with moderate-to-severe disease.

Several other candidates are expected to be approved for atopic dermatitis. The wild card might be Arenas etrasimod, currently in Phase III trials for ulcerative colitis, which has potential in other autoimmune indications.

Gene therapy continues to make its mark in blood disorders, with both UniQure and Biomarin developing hemophilia candidates dangling the promise of one-time therapies for patients who are used to aggressive treatment regimens. Meanwhile, Bluebird Bios LentiGlobin awaits further approvals for sickle cell disease and beta thalassemia next year.

Elsewhere, Myovant is attempting to battle AbbVie in the womens health space, via an expected approval of Relugolix for uterine fibroids. Merck looks forward to a long-awaited approval verdict for its heart failure drug vericiguat in a niche population. And Fabrys patients could have a new and safer enzyme replacement therapy option with the impending approval of Protalixs PRX-102.

In other words, buckle up in advance of another epic and unpredictable year for pharma.

(Agents profiled in this report were chosen in consultation with inThought Research, inDemic, Adis R&D Insight, Ipsos Healthcare and GlobalData. Analyses of featured products include the latest clinical data, revenue forecasts, expected launch dates and likelihood of success as of early November 2020.)

Most anticipated products

Pfizer/BioNtech, Phase III

Type: mRNA vaccine

Where the clinical trials are: BNT162b2 is an mRNA vaccine, which encodes an optimized SARS-CoV-2 full-length spike glycoprotein at a 30g dose level in a two-dose regimen. Interim data as of early November showed that the vaccine was 95% effective. While emergency use authorization could arrive as early as late-Q4 2020, the primary analysis of the Phase III trial is expected in Q2 2021, which could enable mass vaccinations thereafter.

InDemic comment: Pfizers criteria for what constitutes a case of COVID-19 is less stringent than that of its competitors, and this will allow the company to tally up cases more quickly for an earlier readout. While it could generate some skepticism from regulators and the scientific community, Pfizer is also tracking severe cases of COVID-19 and other metrics for efficacy in its vaccine trial. Pfizers refusal to accept federal money for its vaccine development shows confidence in its technology. Dr. Sam Sun, director, InDemic Foundation

What physicians are saying: This candidate was poised to apply for emergency use authorization by late November. Orders for millions of doses have already been placed by U.S., EU and Japanese governments, highlighting the readiness to react to the imminent availability of this much-needed product. Rhoda Schmuecking, head, virology and vaccines CoE, Ipsos

Moderna, Phase III

Type: mRNA vaccine

Where the clinical trials are: mRNA-1273 is an mRNA vaccine that encodes a SARS-CoV-2 spike protein that is stabilized in the prefusion (before infection) conformation. The vaccine is in an ongoing Phase III trial with early reports showing a 94.5% efficacy rate and, even if interim analyses spur emergency use authorization for high-risk individuals in late Q4 2020 or early Q1 2021, a full analysis for widespread approval is due in Q2 2021.

InDemic comment: Moderna is a pioneer in developing mRNA therapeutics. Overall, Moderna has shown consistency and transparency with its vaccine program, which may aid in its uptake and eventual rollout to the public. Its strict definition of what constitutes a COVID-19 case could mean it takes longer for Moderna to produce a final readout. However, that sacrifice could generate greater significance for its readout. Sun

What physicians are saying: With the first interim analysis in November, the possibility of vaccines from Pfizer/BioNTech and Moderna being made available for emergency use authorization before the end of this year certainly makes this a very dynamic space to watch. Schmuecking

Johnson & Johnson, Phase III

Type: human adenoviral vector

Where the clinical trials are: Ad26.COV2.S is composed of a recombinant, replication-incompetent adenovirus type 26 (Ad26) vector, constructed to encode the SARS-CoV-2 spike protein. J&Js 60,000-patient Phase III trial is testing the single-dose vaccine versus placebo. However, the trial was paused in mid-October due to a safety signal of unknown significance. While this could impact its future in the race, the trial was resumed in late October.

InDemic comment: The vaccines one-dose regimen makes it attractive compared to other candidates. Its recent safety event may not be as serious as the rare neurological reactions experienced by two patients on AstraZeneca/Oxford Universitys adenoviral vaccine trial, which was paused in September. J&Js vaccine technology, Advac, has been used in other approved vaccines, and 100,000 patients have been vaccinated with this technology without a concern. Sun

What physicians are saying: Should the late-stage study prove positive, J&J expects to be able to supply 1 billion doses of its vaccine beginning early next year. While this is later than the mRNA vaccine candidates, J&Js boldness with the size of trial and one-shot dose makes it a very interesting option added to the mix. Schmuecking

Gilead, post-approval

Type: anti-viral

What the clinical trials have shown: Remdesivir (brand name Veklury) received full approval from the FDA in October, making it the first antiviral available to treat COVID-19. That approval was based on data from the ACTT-1 study sponsored by the National Institutes of Health, which found a significant benefit in time to recovery and a non-significant trend toward improved survival at 29 days. However, preliminary findings from the WHO Solidarity trial, which followed 11,366 adults at 405 hospitals in 30 countries, found that remdesivir has little or no impact on survival.

InDemic comment: The available data indicate that remdesivir does not improve mortality and might even worsen mortality in COVID-19 patients with severe disease who require high levels of supplemental oxygen or mechanical ventilation. For that reason, it may only be appropriate to consider this drug for hospitalized COVID-19 patients with mild to moderate disease. The conflicting data on survival may also impact Gileads ability to command the high price of $2,600 per course. Sun

What physicians are saying: In lieu of an available vaccine for COVID-19, remedisvir, along with other antivirals in development, provides a valuable option in physicians treatment armamentarium. Schmuecking

Regeneron, Phase III

Type: passive immunotherapy

What the clinical trials have shown: Results from Regenerons ongoing Phase II/III trial in non-hospitalized patients showed that its antibody cocktail significantly reduced viral load and patient medical visits. The company will use this data for an emergency use authorization of REGN-COV2 low-dose to treat mild-moderate patients, for potential 2021 approval. However, the drugs potential is less clear in severe patients: In late October, an independent monitoring committee recommended halting enrollment of patients with high oxygen requirements on its hospitalized patient trial, due to an unfavorable risk/benefit profile.

InDemic comment: REGN-COV2 and other antibody therapies against COVID-19 are expected to show substantial efficacy in reducing hospitalizations. REGN-COV2 also fills an unmet need in that it can be used for outpatients with COVID-19, whereas both remdesivir and dexamethasone are used for hospitalized patients. Sun

What physicians are saying: A premature EUA could hamper ongoing investigations needed to solidify efficacy data and offer better utility insight, according to physicians interviewed by Manasi Vaidya and Reynald Castaneda, associate editors of GlobalDatas Pharma investigative news team. Larger datasets on clinically relevant secondary endpoints, such as reduced hospitalization, are needed for stronger EUA consideration. The ability to manufacture adequate mAbs to provide access to patients is likely to be an issue in the event of a premature EUA, the experts added.

InflaRx, Phase III

Type: complement inhibitor

What clinical trials have shown: In previous trials, IFX-1 was shown to reduce kidney injury, lung emboli and mortality from multi-organ failure, so IFX-1s Phase III trial was initiated to enroll severe COVID-19 patients, with a primary endpoint of mortality. Interim data is expected in March 2021, dependent on enrollment.

InThought comment: The Phase III trial of IFX-1 is enrolling a specific population of very sick COVID-19 patients. Its primary endpoint is objective and clinically meaningful, and its Phase II trial showed that IFX-1 may reduce mortality of COVID-19 multi-organ failure. With a dearth of treatments for severe/critically ill COVID-19 patients, we believe IFX-1s interim data in early 2021 will support the use of complement inhibitors for severe COVID-19 pneumonia. It could also be synergistic with the use of dexamethasone. Sun

Most anticipated products

Johnson & Johnson, pre-registration

Indication: late-stage multiple myeloma

What the clinical trials have found: Cilta-cel is a CAR-T therapy targeting BCMA (B-cell maturation antigen) and a Phase Ib/II study in refractory multiple myeloma is expected to yield a 2021 approval. Longer-term follow-up data from the study demonstrated a 100% overall response rate and an 86% stringent complete response rate at a median of 11.5 months.

InThought comment: The 100% response rate is the main focus of this drug, which, along with its FDA breakthrough therapy designation, has placed Cilta-cel as an emerging frontrunner in the competitive BCMA space. Amanda Weyerbacher, senior principal, inThought Research

Credit Suisse revenue forecast: $427 million by 2025

What physicians are saying: Despite its race (with Bristol Myers Squibbs Ide-cel) to be the first CAR-T therapy approved for multiple myeloma, there is likely to be high anticipation for Cilta-cel as physicians and patients (who have progressed through a number of drug classes) look for strong and durable outcomes Amy Butcher, director, global oncology monitor, Ipsos

Roche, Phase I/IIb

Indication: relapsed or refractory follicular lymphoma

What the clinical trials have found: Mosunetuzumab is a bispecific T-cell engager that targets CD3 and CD20. The FDA granted breakthrough therapy designation for relapsed or refractory follicular lymphoma in July based on a Phase I/Ib GO29781 study demonstrating high response rates and durable complete remissions in people with relapsed or refractory non-Hodgkins lymphoma.

InThought comment: Roche is in active discussions with the FDA for accelerated approval in this indication. Continued updates are expected to build on already promising data, while a subcutaneous formulation could offer at-home administration and patient convenience, which has been set at a higher bar in the time of COVID. Weyerbacher

Credit Suisse revenue forecast: $250 million by 2025

What physicians are saying: Trial results garnered much attention because it achieved efficacy in patients who had already been through four to five different treatments, and even included patients who had undergone CAR-T therapy. This will be a welcome new weapon for patients with an extremely poor prognosis. Alessandra Franceschetti, director, global oncology monitor, Ipsos

Nektar, Bristol Myers Squibb, pre-registration

Indication: melanoma, RCC, NSCLC

What the clinical trials have found: Nektar and BMS are examining the drug combination for a range of solid tumors, including first-line melanoma, renal cell cancer and lung cancer. Phase II melanoma data showed an overall response rate of 53%.

InThought comment: This combination has shown promising activity across a number of solid tumor indications that improve upon current immunotherapy approaches. While melanoma is the most advanced, renal cancer is looking to come out on top. Matt Presby, senior analyst, InThought Research

Credit Suisse revenue forecast: $1.75 billion by 2025 in all indications

What physicians are saying: Although early-stage, the data indicate that the combination could lead to durable responses with a possible advantageous toxicity profile over some other immuno-oncology combinations. Eric Blouin, SVP, Ipsos

Iovance, Phase II

Indication: metastatic melanoma

What the clinical trials have found: Lifileucel is an autologous cell therapy technology, which uses tumor-infiltrating lymphocytes (TILs) from a patients own tumors. In a Phase II metastatic melanoma trial of 66 heavily treated patients, the objective response rate (ORR) was 36%.

InThought comment: Lifileucel could be on track to be the first cell therapy approved for solid tumors. The TILs technology appears to achieve responses on par with or better than currently approved options for second- and third-line melanoma, while responses in cervical cancer (LN-145) are three times greater than current immuno-oncology agents. Presby

What physicians are saying: While Phase II data has shown good antitumor activity and durable response rates, the drug carries a complex manufacturing and delivery process. A Biologics License Application (BLA) submission is expected in 2021 following the FDAs request for additional data on the proposed potency assays. Mo Muhsin, director, oncology, Ipsos

Most anticipated products

Biogen, pre-registration

Indication: Alzheimers disease

What the clinical trials have found: Aducanumab is a monoclonal antibody targeting aggregated forms of beta-amyloid found in the brain. Phase III development of the drug started in late 2016 and was stopped in March 2019 after an independent group analysis deemed the trials not worth continuing because the drug was not expected to reach the trials primary endpoint.

However, in October 2019, Biogen decided it would submit the data for regulatory approval after re-analyzing a larger dataset that showed aducanumab reduced clinical decline in patients at higher doses. On November 6, the FDA advisory committee verdict voted strongly against approving aducanumab on its current dataset. A final verdict from the FDA is due on March 7.

InThought Comment: It will be more difficult, although not impossible, for the agency to ignore this strong no-confidence stance. Standards need to be kept as high as established. For this population, at least in the beginning, the effects of a pharmaceutical intervention proven efficacious in trials is highly likely to have almost imperceptible clinical effectiveness in real life in many cases, if not the majority. That will be difficult enough. We really need to be as certain as humanly possible here. Leon Henderson-MacLennan, medical adviser, InThought Research

Credit Suisse revenue forecast: $15.4 billion by 2025

What physicians are saying: There are currently no disease-modifying treatments available for Alzheimers. If aducanumab gains approval by the FDA, it could represent the first real signs of hope for the millions of people impacted by this devastating disease. Scott Morano, VP, healthcare, Ipsos

AbbVie, pre-registration

Indication: migraine

What the clinical trials have found: AbbVies atogepant is an oral calcitonin gene-related peptide (CGRP) receptor antagonist being developed for first-line migraine prevention. A positive Phase III trial showed a significant reduction in mean monthly migraine days over 12 weeks compared to placebo. AbbVie is planning regulatory filings in the U.S. and Europe in 2021.

InThought comment: Atogepant has a good shot to ride on the coattails of the rapid adoption of oral migraine and carve out a significant portion of the multibillion-dollar migraine market. Yet more mature data is awaited to elucidate its exact differentiation and positioning to competitors and which patient setting this drug will truly be used for. Henderson-MacLennan

Credit Suisse revenue forecast: $723 million by 2025

What physicians are saying: The past two years have seen radical improvements in the treatment options available for the prevention of migraine. Atogepant represents another giant step forward with its oral formulation that could make the CGRP receptor agonist much more palatable for PCPs and therefore more accessible to patients. Morano

Biohaven Pharmaceuticals, pre-registration

Indication: migraine

What the clinical trials have found: A pivotal Phase II/III study showed that zavegepant at 10mg and 20mg doses was statistically superior to placebo on co-primary endpoints of pain freedom and freedom from most bothersome symptoms at two hours. It also showed rapid onset of pain relief at 15 minutes with a favorable toxicity profile and no signal of hepatotoxicity. An NDA filing is expected in the second half of 2021.

InThought Comment: While more data is still awaited, zavegepant is being watched keenly because of its differentiated intranasal route of administration. This is likely a safe choice and could allow Biohaven to carve out a niche portion of the migraine market. Henderson-MacLennan

GlobalData consensus forecast: $563 million in 2026

What physicians are saying: CGRP treatments have proven effective for migraine. Zavegepant represents another innovation in not only providing potentially both acute and preventive treatment, but also in an administration-friendly intranasal formulation. Its also potentially a treatment for COVID-19. Morano

Most anticipated products

UCB, pre-registration

Indication: moderate to severe psoriasis

What the clinical trials have found: Bimekizumab is an IL-17 inhibitor that suppresses both IL-17A and IL-17F, which are two key cytokines driving inflammatory processes. UCB has completed three successful Phase III trials of bimekizumab, showing superior skin clearance at week 16 compared to placebo and AbbVies blockbuster Humira. The drug has a PDUFA approval date in March 2021 and is also being examined for treatment of psoriatic arthritis and ankylosing spondylitis.

InThought comment: Bimekizumab has shown stronger efficacy compared to IL-17a inhibitors such as Novartis Cosentyx and Lillys Taltz, while theoretical concerns of suicidal ideation associated with suppressing more of the IL-17 pathway (sparked by Amgens IL-17RA inhibitor Siliq) have so far not played out. Therefore, strong efficacy with a fast onset of action and clean safety profile makes bimekizumab likely to be approved and an exciting new option for psoriasis patients. Adam Schaffner, senior principal, InThought Research

Credit Suisse revenue forecasts: $308 million in psoriasis and $692 million in all indications by 2025

What physicians are saying: While Cosentyx was the first-in-class IL17 to market in psoriasis and has been very successful to date, recent Phase IIIB head-to head trials showed superiority to Cosentyx for PASI 100 at week 16 and week 48. It will be interesting to see how bimekizumab fares on the psoriasis market versus other IL17s and IL23s. Nicola Bailey, SVP, head of autoimmune CoE, Ipsos

Bristol Myers Squibb, Phase III

Indication: psoriasis

What clinical trials have found: BMS-986165 is an oral TYK2 inhibitor currently in Phase III trials for moderate to severe psoriasis. Phase II trials showed significant skin clearance after 12 weeks, while topline data from its first Phase III study announced in early November showed superiority to blockbuster drug Otezla. Thats a drug that BMS would have inherited through its acquisition of Celgene in 2019, but was instead sold to Amgen for $13 billion. That now seems like a worthwhile trade-off. The second Phase III data are expected in the Q1 2021.

InThought comment: BMS-986165 demonstrated impressive efficacy similar to IL-23 injectable drugs such as AbbVies Skyrizi and J&Js Tremfya, but as an oral option it could be a game changer for patients with moderate-to-severe disease looking for a more convenient self-administration option. The drug has a lot of buzz around it as it is being pursued in other indications, including inflammatory bowel diseases. Schaffner

Credit Suisse revenue forecast: $2.3 billion in all indications

What physicians are saying: BMS-986165 is touted as a future best-in-class oral drug with a new mechanism not seen in psoriasis. In addition to its oral convenience, it is exciting in that it is so far noted for a good side-effect profile, with possibly no sacrifice on efficacy. Bailey

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Pipeline Report 2021: Promising drugs tick along across a range of conditions - Features - MM&M - Medical Marketing and Media

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics (NASDAQ: MGTA) and bluebird bio, Inc. (NASDAQ: BLUE) today announced an exclusive clinical trial collaboration to evaluate the utility of MGTA-145, in combination with plerixafor, for mobilization and collection of stem cells in adults and adolescents with sickle cell disease (SCD). The data from this clinical trial could provide proof-of-concept for MGTA-145, in combination with plerixafor, as the preferred mobilization regimen for patients with SCD. bluebird bios experience with plerixafor as a mobilization agent in sickle cell disease aligns with Magentas combination therapy approach, utilizing MGTA-145 plus plerixafor with potential to achieve safe, rapid and reliable mobilization of sufficient quantities of high-quality stem cells to improve outcomes associated with stem cell transplantation. Under the collaboration, the stem cells will be fully characterized, and Magenta will undertake preclinical studies to evaluate the ability of these cells to be gene corrected and engrafted in mouse models. The companies will co-fund the clinical trial and Magenta will retain all rights to its product candidate.

We are excited to build upon our leading position in the field of ex-vivo gene therapy and the promising clinical data with LentiGlobin in SCD with a collaboration focused on achieving improved stem cell mobilization, said Dave Davidson, M.D., chief medical officer, bluebird bio. In this initial study, we hope to establish whether the combination of plerixafor with MGTA-145 can generate appropriate CD34+ stem cells with a single round of mobilization. If successful, we hope to evaluate this novel mobilization regimen with LentiGlobin to make another step forward in the treatment of patients with SCD.

Achieving reliable and rapid stem cell mobilization and a simplified collection process can ensure the entire patient experience is optimal with respect to therapeutic outcome. The incorporation of bluebird bios experience in this area of treatment will be immensely valuable in further developing MGTA-145 plus plerixafor to address the remaining unmet needs in gene therapy approaches for diseases like sickle cell disease, said John Davis Jr., M.D., M.P.H., M.S., Head of Research & Development and Chief Medical Officer, Magenta Therapeutics. We look forward to collaborating with bluebird bio to evaluate MGTA-145 as the preferred mobilization option for people with sickle cell disease.

SCD is a serious, progressive and debilitating genetic disease caused by a mutation in the -globin gene that leads to the production of abnormal sickle hemoglobin (HbS), causing red blood cells (RBCs) to become sickled and fragile, resulting in chronic hemolytic anemia, vasculopathy and painful vaso-occlusive events (VOEs). For adults and children living with SCD, this means unpredictable episodes of excruciating pain due to vaso-occlusion as well as other acute complicationssuch as acute chest syndrome (ACS), stroke, and infections, which can contribute to early mortality in these patients.

Currently available mobilization drugs, including granulocyte-colony stimulating factor (G-CSF), a commonly used mobilization agent administered over the course of five to seven days in other transplant settings, is not used in sickle cell disease because it can trigger vaso-occlusive crises and even death in adults and adolescents. Plerixafor is used to mobilize a patients stem cells for collection prior to transplant and while an available treatment option, multiple cycles of apheresis and collection may sometimes be required to generate sufficient stem cells for gene therapy. Magenta is developing MGTA-145, in combination with plerixafor, to be the preferred mobilization regimen for rapid and reliable mobilization and collection of hematopoietic stem cells (HSCs) to improve stem cell transplantation outcomes in multiple disease areas, including genetic diseases such as sickle cell disease, as well as blood cancers and autoimmune diseases.

About Magenta Therapeutics MGTA-145

MGTA-145, in combination with plerixafor, has demonstrated, in a recently completed Phase 1 study in healthy volunteers, it can rapidly and reliably mobilize high numbers of functional stem cells in a single day, without the need for G-CSF. MGTA-145 works in combination with plerixafor to harness a physiological mechanism of stem cell mobilization to rapidly and reliably mobilize HSCs for collection and transplant across multiple indications.

Additionally, as shown in preclinical studies, stem cells mobilized with MGTA-145 can be efficiently gene-modified and are able to engraft, potentially allowing for safer and more efficient mobilization for gene therapy approaches to treat sickle cell disease and other genetic diseases.

Magenta completed its Phase 1 trial of MGTA-145 in healthy volunteers, demonstrating MGTA-145 was well tolerated and enables same-day dosing, mobilization and simplified collection of sufficient stem cells for transplant, meeting all primary and secondary endpoints.

About bluebird bio, Inc.

bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, were developing gene and cell therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, were working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.

bluebird bio is a human company powered by human stories. Were putting our care and expertise to work across a spectrum of disorders: cerebral adrenoleukodystrophy, sickle cell disease, -thalassemia and multiple myeloma, using gene and cell therapy technologies including gene addition, and (megaTAL-enabled) gene editing.

bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.

Follow bluebird bio on social media: @bluebirdbio, LinkedIn, Instagram and YouTube.

LentiGlobin and bluebird bio are trademarks of bluebird bio, Inc.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavour, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption Risk Factors in Magentas Annual Report on Form 10-K filed on March 3, 2020, and in bluebird bios Annual Report on Form 10-K filed on February 18, 2020, as updated by each companys most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta and bluebird bio believe that the expectations reflected in the forward-looking statements are reasonable, neither Magenta nor bluebird bio can guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta or bluebird bio, nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. Neither Magenta nor bluebird undertake any obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

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Magenta Therapeutics and bluebird bio Announce a Phase 2 Clinical Trial Collaboration to Evaluate Magenta's MGTA-145 for Mobilizing and Collecting...

-- Results from the multiple-ascending dose trial demonstrate proof-of-concept for SRP-5051 and support continued dose escalation --

-- At a total dose exposure approximately 10x lower than eteplirsen, SRP-5051 at 20 mgs/kg showed enhanced tissue exposure, greater exon skipping, and greater dystrophin production with no negative renal or other laboratory findings --

-- These are the first clinical results from the Companys peptide phosphorodiamidate morpholino oligomer (PPMO) technology, its next-generation chemistry platform --

CAMBRIDGE, Mass., Dec. 07, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced results from the ongoing MOMENTUM study (Study 5051-201), a global Phase 2 clinical trial, of SRP-5051, its next-generation treatment for patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping. This is the first clinical data from SRP-5051, an investigational treatment that uses Sareptas peptide phosphorodiamidate morpholino oligomer (PPMO) technology. PPMO technology includes a proprietary cell-penetrating peptide that is conjugated to Sareptas PMO backbone with the goal of increasing cellular uptake of drug in the muscle tissue.

Results from Part A of the multi-ascending dose MOMENTUM study found consistently higher tissue exposure, exon-skipping and dystrophin production in patients taking a monthly dose of SRP-5051 compared to baseline. SRP-5051 was generally well-tolerated across all doses studied, with no clinical or laboratory findings reported. The results support continued dose escalation of SRP-5051 and further clinical development.

Sareptas PMO RNA technology is a vital platform on which we design therapies to treat those with Duchenne muscular dystrophy. Our next-generation PPMO technology is designed to increase cell penetration with the goal of offering significantly improved efficacy with more convenient dosing in Duchenne patients amenable to exon skipping, said Doug Ingram, president and chief executive officer, Sarepta. While patient numbers in each dose arm are small, the higher tissue concentration, exon skipping and dystrophin production in the 20 mg/kg dosing group were observed at an early 12-week timepoint and with far less cumulative drug exposure when compared to our current PMO technology. We know from our experience with PMOs that exon-skipping and dystrophin increase over time, and these results along with our preclinical experience, give us confidence as we dose escalate and continue to advance our PPMO exon-skipping therapies for Duchenne, including another five potential therapies that have already been designed, and explore the utility of the PPMO RNA platform for new disease indications.

When compared to a control group of Duchenne patients from the PROMOVI study who received biopsies at 24 weeks after taking a weekly 30 mg/kg dose of eteplirsen, once-monthly dosing of SRP-5051 resulted in higher muscle concentration, increased exon-skipping and dystrophin at 12 weeks. A dose-dependent increase in exon-skipping and dystrophin was observed, with patients in the 20 mg/kg dose group of SRP-5051 seeing a 1.6-fold increase in exon skipping (n=4) and a 5-fold increase in the % of normal dystrophin (n=2) when compared to the group taking eteplirsenat 24 weeks.

The incidence of adverse events in the MOMENTUM study was similar across all dosed cohorts and does not suggest dose dependency. One treatment-emergent event, unrelated to study drug, occurred in the 4 mg/kg dose group. No clinical or laboratory findings were observed. Full results will be presented at a future medical meeting.

About MOMENTUM (SRP-5051-201) MOMENTUM is a multi-arm, ascending dose study designed to identify the maximum tolerated dose of SRP-5051. Informed by Study 5051-101, a single-ascending dose study of SRP-5051, patients in the MOMENTUM study will receive monthly intravenous (IV) infusions of SRP-5051, starting at 4 mg/kg and ascending to 40 mg/kg. The study will enroll up to 24 patients, both ambulant and non-ambulant, between the ages of 7 to 21 at sites in the U.S., Canada, Australia and European Union. The primary endpoint is safety, and secondary and exploratory endpoints include exon-skipping, dystrophin expression and tissue concentration. All patients will undergo a muscle biopsy at baseline and 12 weeks in Part A and at baseline and 24 weeks in Part B. More information can be found on http://www.clinicaltrials.gov.

About SRP-5051SRP-5051 uses Sareptas PPMO chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. SRP-5051 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. PPMO is Sareptas next-generation chemistry platform designed around a proprietary cell-penetrating peptide conjugated to the PMO backbone, with the goal of increasing tissue penetration, increasing exon skipping and significantly increasing dystrophin production. Around 13% of DMD patients have mutations which make them amenable to skipping exon 51. If successful, the PPMO offers the potential for improved efficacy and less frequent dosing for patients.

About Duchenne Muscular DystrophyDMD is an X-linked rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500 - 5,000 male births worldwide. A devastating and incurable muscle-wasting disease, DMD is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. Progressive muscle weakness in the lower limbs spreads to the arms, neck and other areas of the body. The condition is universally fatal, and death usually occurs before the age of 30 due to respiratory or cardiac failure.

About EXONDYS 51 EXONDYS 51 (eteplirsen) uses Sareptas proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 51 of dystrophin pre-mRNA, resulting in skipping of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.

EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.

This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in some patients treated with EXONDYS 51. Continued approval may be contingent upon verification of a clinical benefit in confirmatory trials.

EXONDYS 51 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental.

Important Safety Information About EXONDYS 51Hypersensitivity reactions, including rash and urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension, have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.

Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 mg or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.

In the 88 patients who received 30 mg/kg/week of EXONDYS 51 for up to 208 weeks in clinical studies, the following events were reported in 10% of patients and occurred more frequently than on the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.

For further information, please see the fullPrescribing Information.

AboutSarepta TherapeuticsAt Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Forward-Looking StatementsThis press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the potential benefits of PPMO, including increasing cellular uptake of drug in the muscle tissue and offering significantly improved efficacy with less frequent dosing; our plan to continue to dose escalate and advance our PPMO exon-skipping therapies for Duchenne, including another five potential therapies that have already been designed, and to explore the utility of the PPMO RNA platform for new disease indications; and potential market opportunities.

These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: success in preclinical trials and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; the data presented in this release may not be consistent with the final data set and analysis thereof or result in a safe or effective treatment benefit; different methodologies, assumptions and applications we utilize to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or foreign regulatory authorities; if the actual number of patients suffering from DMD is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, due to a variety of reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates and the COVID-19 pandemic; and even if Sareptas programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052, iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566,tsorrentino@sarepta.com

See the article here:
Sarepta Therapeutics Announces Positive Clinical Results from MOMENTUM, a Phase 2 Clinical Trial of SRP-5051 in Patients with Duchenne Muscular...

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that it will host a webcast on Monday, December 7 at 6:00PM EST to discuss presentations at the 62nd American Society of Hematology (ASH) Annual Meeting being held virtually December 5-8, 2020. The ASH presentations will highlight data from the Fanconi Anemia, Leukocyte Adhesion Deficiency-I and the Pyruvate Kinase Deficiency programs.

Following the discussion, Rocket management and key opinion leaders will be available for a brief Q&A session.

To access the live webcast and presentation, please click here. The webcast replay will be available on the Rocket website following the completion of the call.

Investors may listen to the call by dialing (866) 866-1333 from locations in the United States or +1 (404) 260-1421 from outside the United States. Please refer to conference ID number 50038102.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding data updates at ASH, its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, filed November 6, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

Link:
Rocket Pharmaceuticals to Hold Webcast for Investors Highlighting Data Presented at the 62nd American Society of Hematology Annual Meeting - Business...

MedicalResearch.com Interview with:

Steven Pipe, MDProfessor of Pediatrics and PathologyLaurence A. Boxer Research Professor of Pediatrics and Communicable DiseasesPediatric Medical Director, Hemophilia and Coagulation Disorders ProgramDirector, Special Coagulation LaboratoryUniversity of Michigan

MedicalResearch.com: What is the background for this study?

Response: Hemophilia B is an inherited bleeding disorder where patients are missing clotting factor IX (9), a critical blood clotting protein.Patients with a severe deficiency are at risk for traumatic and spontaneous bleeds primarily into joints.Repeated bleeding into joints causes more than acute pain and swelling but also leads to inflammatory and degenerative changes in joints that eventually leads to severe debilitating arthritis that can be crippling.To try to prevent this, patients as young as infants are placed on regular infusions of clotting factor IX concentrates.The relatively short half-life of factor IX means patients must infuse on average once to twice a week.These can only be delivered intravenously parents and then patients themselves have to learn this.Prophylaxis must be continued lifelong to try to prevent bleeding events and protect joint health over the lifespan.This is a tremendous burden on the patient and their caregivers.

Even with regular prophylaxis, joint bleeds may still occur and arthropathy may still ensue.This is because the blood levels often reach critically low levels prior to the next infusion.Gene therapy aims to deliver a functional copy of the factor IX gene such that the patients own liver will make a continuous supply of factor IX that is delivered to the bloodstream.At steady state with levels close to or within the normal range, patients would no longer be subject to bleeding events and would not require prophylaxis any longer.We hope that such a one-time treatment would produced durable, functionally curative levels of factor IX.

MedicalResearch.com: What are the main findings?

Response: The 3 key takeaways are:

MedicalResearch.com: What should readers take away from your report?

Response: I think it is best to characterize this as a treatment that liberates patients with hemophilia from the burden of repeated prophylactic infusions of clotting factor to protect them from recurrent bleeding events.Gene therapy offers a chance to have steady state levels of factor in the blood that would eliminate risk for spontaneous and even traumatic bleeding events.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Response: These are very encouraging results maximizes eligibility for patients by being able to treat in the presence of pre-existing neutralizing antibodies.Results are in a range that allow for cessation of prophylaxis and continued bleed protection.Results appear to be durable through the follow up period to date.The 26 week endpoint was a co-primary endpoint.The other endpoints are factor IX activity at 52 weeks and annualized bleeding rate over the 52 weeks post-dosing. Those endpoints should be analyzed in 2021 and decisions could then be made on submission for regulatory review.

MedicalResearch.com: Is there anything else you would like to add? Any disclosures?

Response: This could be potentially clinical practice changing, if approved. This looks to be a transformative therapy for patients and could be an option for patients across the adult lifespan we treated patients from age 19 through 75. Our patient population have been looking forward for a long time for a one-time therapy like this that would provide lasting protection from bleeds.

I have served as a paid consultant to uniQure and chair the Steering Committee for the global clinical trial program

Citation: ASH2020 Oral Abstract

First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A BAY 2599023 has Broad Patient Eligibility and Stable and Sustained Long-Term Expression of FVIII

Steven W. Pipe, MD1, Francesca Ferrante, MD2*, Muriel Reis3*, Sara Wiegmann4*, Claudia Lange5*, Manuela Braun5*and Lisa A Michaels6*https://ash.confex.com/ash/2020/webprogram/Paper139803.html

The information on MedicalResearch.com is provided for educational purposes only, and is in no way intended to diagnose, cure, or treat any medical or other condition. Always seek the advice of your physician or other qualified health and ask your doctor any questions you may have regarding a medical condition. In addition to all other limitations and disclaimers in this agreement, service provider and its third party providers disclaim any liability or loss in connection with the content provided on this website.

4

Link:
Gene Therapy Liberates Hemophilia B Patients from Requiring Regular Infusions of Clotting Factor - MedicalResearch.com

Dublin, Nov. 27, 2020 (GLOBE NEWSWIRE) -- The "Gene Therapy - Global Market Trajectory & Analytics" report has been added to ResearchAndMarkets.com's offering.

In a Major Setback to the Healthcare System, Non-COVID-19 Care Delivery Bears the Brunt of the Pandemic. Gene Therapy Market Slumps by -13.6%

The global market for Gene Therapy is expected to decline by -13.6% in the year 2020 and thereafter recover and grow to reach US$3.3 billion by the year 2027, trailing a post COVID-19 CAGR of 19.5% over the analysis period 2020 through 2027.

Governments worldwide are focusing all healthcare resources on fighting the global pandemic. Billions of dollars have poured into researching COVID-19 drugs, therapies and vaccines. Over US$8 billion globally excluding the U.S. has been pledged only for vaccine development. The U.S. has independently pumped billions of dollars into COVID-19 research and response. The massive reallocation of funds and reprioritization of efforts has left a glaring gap in other sectors of healthcare.

Gene therapy which holds promise for treating cancer, cystic fibrosis, heart disease, diabetes, hemophilia & AIDS, is slumping due to lack of research funds & reduced footfall of patients seeking treatment. Given the complex and fragile manufacturing and delivery system along with funding models of the industry, COVID-19 has emerged as a black swan event. Various players still find it challenging to ensure timely delivery of gene therapy to patients and clinical sites.

There are concerns regarding administration of cell and gene therapies. The chances of virus transmission, mainly to people in the high-risk group, coerced hospitals to delay or cancel appointments. In addition, travel restrictions and stay-at-home orders discouraged patients from visiting to treatment centers. Treatments intended to be delivered into ICUs are being impacted by bed reservations made for patients with COVID-19 infection.

R&D and preclinical activities are also affected by supply shortages as a result of strong demand for consumables like reagents and PPE from COVID-19 laboratories. The clinical development segment suffered the most due to concerns regarding recruitment of patients and suspension of trial enrollments for protecting participants from the risk of infection. These issues are delaying activation of new sites, prompting players to postpone new clinical trials.

However, the intensity of disruptions for cell and gene therapy trials was less in comparison to the pharmaceutical industry due to association of the former with rare and serious medical conditions, enabling participants to continue trials. While companies targeting paediatric diseases suspended trials, others dealing with oncology maintained the pace. COVID-19 has also impacted patient assessment and has made it difficult for companies to perform follow-up evaluations for trial participants. These issues are attributed to confluence of various factors like travel ban, withdrawal of several services from healthcare sites and the risk of virus transmission.

In addition, these disruptions are anticipated to threaten existence of certain cell and gene therapy companies, particularly small-scale biotech players that are in pre-commercial phase and rely on external funding. As governments, stakeholders, pharmaceutical companies and venture capitalists invest in these players on the basis of research milestones, pipeline progress and data readouts, ability of these companies to secure future funding will also be affected.

In the post COVID-19 period, growth will be led by therapy indications in the field of oncology. Gene therapies hold promise to improve the condition of patients where traditional cancer treatments such as radiation and chemotherapy are not effective. Blood and lymphatic cancers hold huge potential as gene therapies can manipulate the genetic information to target the cancerous proteins, thereby enabling the body to fight against the cancers. Oncology will remain the key area of focus for gene therapy applications. Cancer therapies represent the leading category, as is gauged through robust rise in the number of molecules being tested across numerous clinical trials.

Novartis which recently bagged the U.S. FDA approval for Kymriah, a gene therapy designed for the treatment of hematological cancer, is seeking to gain commercial approval in established and emerging countries. Similarly, Kite Pharma, the developer of YESCARTA, the first CAR T-cell therapy approved for certain types of non-Hodgkin lymphoma in adults, has formed a separate team to provide end-to-end support for its Yescarta customers including hospitals and clinics. Such efforts by developers would augment the use case of gene therapies in treatment of large B-cell lymphoma and acute lymphoblastic leukemia (ALL), the high potential cancer treatment verticals.

More developmental focus will also be shed on monogenic rare diseases which have clearer genomic targets and the unmet need in smaller patient populations. Majority gene therapies so far have come to market through accelerated review pathways of regulatory authorities. In the year 2018 alone, over 150 applications for investigational new drugs for gene therapies were filed.

In the coming years, there will be significant improvement in the number of approvals for new gene therapies. The growth is anticipated to emerge from different modalities including RNAi, ASOs and CRISPR gene editing based therapeutics which offer long term opportunities for growth. These technologies are generating much excitement for investors.

Competitors identified in this market include, among others:

Key Topics Covered:

1. MARKET OVERVIEW

2. FOCUS ON SELECT PLAYERS

3. MARKET TRENDS & DRIVERS

4. GLOBAL MARKET PERSPECTIVE

For more information about this report visit https://www.researchandmarkets.com/r/x4ed1e

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

Original post:
Global Gene Therapy Market Report 2020-2027: Market is Projected to Reach a Revised $3.3 Billion - GlobeNewswire