Category Archives: Genetic Therapy

By

LINDA A. JOHNSON AP Medical Writer

August 19, 2020, 7:23 PM

4 min read

FAIRLESS HILLS, PA -- Investors fled drug developer BioMarin in droves on Wednesday, driving shares down by a third after U.S. regulators rejected the companys potentially game-changing hemophilia A gene therapy over concerns it might not really be a one-and-done lifetime treatment.

The U.S. Food and Drug Administration's rejection late Tuesday means the San Rafael, California-based company will have to complete an ongoing late-stage patient study, likely delaying possible approval till late in 2022.

The infused therapy, called Roctavian, could have freed hemophilia A patients from frequent, extremely expensive infusions of a blood-clotting therapy to prevent dangerous internal bleeding. It had been highly anticipated by doctors, patients and investors.

In a statement, BioMarin said the company and the FDA previously agreed on how much patient testing data the agency required to review the therapy, but in its rejection letter the FDA for the first time recommended Biomarin finish the late-stage study and provide two years of follow-up data on the therapys safety and efficacy in preventing internal bleeding for all study participants.

The company added that FDA concluded differences between the results of a small, early-stage study and interim data from the late-stage study left unclear how long the therapys effect would last.

Roctavian was meant to free patients with severe hemophilia A from 100 to 150 IV infusions of Factor VIII per year to prevent or at least reduce painful, spontaneous bleeding into joints and muscles, which can cause permanent damage to them.

Also known as valoctocogene roxaparvovec or valrox for short, it would have been the first gene therapy approved in the U.S. for any type of hemophilia. That's a rare, genetic bleeding disorder in which people dont have enough of a clotting protein called Factor VIII due to a mutation in the gene responsible for producing it. They repeatedly suffer spontaneous internal bleeding. About 1 in 10,000 people, mostly males, have hemophilia A, including about 20,000 in the U.S. About half have severe disease.

The gene therapy works by using an inactivated virus, created in a lab, to deliver to liver cells a working gene via a one-time IV infusion meant to enable the body to produce FVIII on its own.

Questions about whether it would work for a lifetime or just a few years came amid rumors that Biomarin might set a price tag as high as $3 million per patient. That would top the price for the most expensive therapy ever approved by the FDA, Swiss drugmaker Novartis AGs gene therapy for spinal muscular atrophy, Zolgensma, which was launched in spring 2019 with a $2.125 million price tag per patient.

Biomarin has estimated the lifetime cost of current treatments to prevent bleeding at about $25 million, arguing its gene therapy would save far more than its cost.

Several other drugmakers are developing gene therapies for hemophilia A but are further behind in testing: partners Pfizer Inc. and Sangamo Therapeutics, Spark Therapeutics and Generation Bio.

SVB Leerink analyst Joseph Schwartz on Wednesday slashed his 12-month price target for BioMarin's stock from $140 to $113 per share. He called the FDA ruling a major negative surprise, but added, We would not sell the stock here, as disappointing as this is.

Schwartz noted that after BioMarin disclosed disappointing data from an interim analysis of the ongoing late-stage study, company shares dropped to $63. Schwartz believes BioMarins other assets are worth an $88 share price.

The company currently sells six medicines in the U.S., all for rare genetic or enzyme disorders. It posted a profit of $52 million on revenue of $932 million in the first six months of 2020.

In mid-afternoon trading, Biomarin Pharmaceutical Inc. shares plunged $42.62, or 36%, to $75.92. Trading volume by then was about 19 times the usual number of BioMarin shares traded in a day.

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FDA blocks much-anticipated BioMarin hemophilia gene therapy - ABC News

Gyroscope Therapeutics Limited, a clinical-stage retinal gene therapy company, today announced the initiation of its Phase II programme evaluating its investigational gene therapy, GT005, for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). Dry AMD is a leading cause of permanent vision loss in people over the age of 50. GT005 is a one-time AAV-based gene therapy that is delivered under the retina. The goal of the Phase II clinical trial programme is to determine if GT005 has the potential to slow the progression of GA.

Gyroscope plans to conduct two Phase II trials evaluating GT005 in people with GA. The first, called EXPLORE, is enrolling people who have a mutation in their Complement Factor I (CFI) gene [NCT04437368]. The first patient to receive GT005 in EXPLORE was enrolled and dosed by Dr. Arshad M. Khanani at Sierra Eye Associates in Reno, Nev., USA.

Geographic atrophy is a devastating diagnosis, as there are no approved treatments for this gradual and irreversible loss of vision, said Arshad M. Khanani, M.D., M.A., Director of Clinical Research at Sierra Eye Associates and Clinical Associate Professor at the University of Nevada, Reno School of Medicine, and an investigator in the EXPLORE trial. We are excited to participate in this trial evaluating GT005 for the potential to slow progression of geographic atrophy. We believe one-time gene therapies could be a major advancement in the field of retinal disease.

GT005 is designed to restore balance to an overactive complement system, a part of the immune system, by increasing production of the CFI protein. An overactive complement system has been implicated in the development of AMD. The CFI protein regulates the activity of the complement system. It is believed that increasing CFI production will dampen the systems overactivity and reduce inflammation, with the goal of preserving a persons eyesight.

Research has found that approximately 3% of people with dry AMD have certain CFI mutations that correlate with low CFI levels in the blood and a higher risk of developing AMD.1 Gyroscope estimates that more than 100,000 people with GA in the United States and EU5 European countries may have these mutations.1,2,3,4 The EXPLORE trial will evaluate GT005 in this group of people with mutations in their CFI gene.

Research has also shown that a small supplementation of CFI can normalise complement activity in the blood,5 suggesting GT005 may also be applicable for a broader group of people with GA. It is estimated that approximately one million people in the United States alone have GA.4 Gyroscope therefore plans to initiate a second Phase II trial in 2020 that will evaluate GT005 in a broader GA population.

We are excited about the potential of GT005 for people with dry AMD. Research suggests GT005 may be best suited for people with certain mutations in their CFI gene. However, evidence also suggests it may have potential for a broader population of people with geographic atrophy, said Nadia Waheed, M.D., MPH, Chief Medical Officer of Gyroscope. We have designed our clinical programme to evaluate these groups in two distinct Phase II trials, with the goal of determining which patients GT005 may be most appropriate for and to further our understanding of the role of the complement system in AMD.

About the EXPLORE Trial

EXPLORE is a Phase II, multicentre, randomised trial evaluating the safety and efficacy of GT005 administered as a single subretinal injection.

EXPLORE is enrolling people who are aged 55 or older and have a clinical diagnosis of GA secondary to dry AMD and who have a mutation of the CFI gene. People being screened for the trial will be genotyped for the mutations. Trial participants will be randomised to one of three treatment arms: GT005 dose 1, GT005 dose 2 or a control arm. Participants in the control arm will receive current standard of care. The primary endpoint of EXPLORE is progression of GA over 48 weeks. The study will also evaluate GT005 for various safety and tolerability measures. Gyroscope plans to enroll approximately 75 patients at approximately 40 centres based in the United States, United Kingdom, Europe and Australia.

Gyroscope will announce details about the second Phase II trial of GT005 at a later date.

About Age-Related Macular Degeneration (AMD) and Geographic Atrophy (GA)

AMD is a leading cause of blindness affecting an estimated 196 million people globally.6 AMD typically affects people aged 50 and older, and causes a gradual and permanent loss of central vision that worsens over time.7 There are no approved treatments for the dry form of AMD, which is the most common, impacting approximately 90% of people with the disease.8 As dry AMD advances it leads to GA, an irreversible degeneration of retinal cells. This vision loss can be devastating, severely impacting a persons daily life as they lose the ability to drive, read and even see the faces of loved ones.

About Gyroscope Therapeutics: Vision for Life

Gyroscope Therapeutics is a clinical-stage retinal gene therapy company developing and delivering gene therapy beyond rare disease to treat a leading cause of blindness, dry AMD. Our lead investigational gene therapy, GT005, is a one-time therapy delivered under the retina. GT005 is designed to restore balance to an overactive complement system by increasing production of the Complement Factor I protein. GT005 is currently being evaluated in a Phase I/II clinical trial called FOCUS and a Phase II clinical trial called EXPLORE.

Syncona Ltd, our lead investor, helped us create the only retinal gene therapy company to combine discovery, research, drug development, a manufacturing platform and surgical delivery capabilities. Headquartered in London with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness. For more information visit: http://www.gyroscopetx.com and follow us on Twitter (@GyroscopeTx) and on LinkedIn.

References

1 Kavanagh D, Yu Y, Schramm EC, et al. Rare genetic variants in the CFI gene are associated with advanced age-related macular degeneration and commonly result in reduced serum factor I levels. Hum Mol Genet. 2015;24(13):3861-3870.

2 Data on File.

3 Friedman DS, OColmain BJ, Muoz B, et al. Prevalence of age-related macular degeneration in the United States [published correction appears in Arch Ophthalmol. 2011 Sep;129(9):1188]. Arch Ophthalmol. 2004;122(4):564-572.

4 Rudnicka AR, Kapetanakis VV et al. Incidence of late-stage age-related macular degeneration in American whites: systematic review and meta-analysis. Am J Ophthalmol 2015;160:85-93.

5 Lachmann PJ, Lay E, Seilly DJ, Buchberger A, Schwaeble W, Khadake J. Further studies of the down-regulation by Factor I of the C3b feedback cycle using endotoxin as a soluble activator and red cells as a source of CR1 on sera of different complotype. Clin Exp Immunol. 2016;183(1):150-156.

6 Wong WL, Su X, Li X, et al. Global prevalence of age-related macular degeneration and disease burden projection for 2020 and 2040: a systematic review and meta-analysis. Lancet Glob Health 2014;2:e106116.

7 National Eye Institute. Age-Related Macular Degeneration. https://www.nei.nih.gov/learn-about-eye-health/eye-conditions-and-diseases/age-related-macular-degeneration. Accessed July 16, 2020.

8 Centers for Disease Control and Prevention. Age-Related Macular Degeneration. https://www.cdc.gov/visionhealth/basics/ced/. Page last reviewed June 3, 2020. Accessed August 4, 2020.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200812005751/en/

Charlotte Arnold

VP, Corporate Affairs

Gyroscope Therapeutics

media@gyroscopetx.com

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Gyroscope Therapeutics Announces Initiation of Phase II Programme Evaluating its Investigational Gene Therapy, GT005, for Dry Age-Related Macular...

NEW YORK, Aug. 12, 2020 /PRNewswire/ --

INTRODUCTION Since the approval and launch of cell and gene therapy products, such as Zolgensma (2019), ZYNTEGLO (2019), Luxturna (2017), KYMRIAH (2017) and YESCARTA (2017), there has been a significant increase in demand for viral vectors. Experts believe that the number of such therapies is likely to double over the next couple of years. It is also worth mentioning that this particular field of medical research received close to USD 10 billion in funding in 2019. Currently, over 1,000 cell and gene therapy-related clinical trials are being conducted, worldwide. Genetic manipulation is a basic requirement of cell and gene therapy development, and, as a result, gene delivery vectors are considered to be of utmost importance in this domain. So far, viral vectors (such as those based on adeno-associated viruses (AAV), adenoviruses, lentivirus, retroviruses and other viruses) have been shown to be the most versatile gene delivery tools available, having demonstrated high transfection efficiencies in both preclinical and clinical settings. Further, taking into account the therapeutic efficacy and low side effects profiles of cell and gene therapies, the demand for such interventions is anticipated to grow at a rapid pace, resulting in a proportional increase in need for appropriate vector systems, as well. However, viral vector development and manufacturing is a complex and cost intensive process. One of the primary concerns associated with viral vector production is related to yield; in fact, a singular batch run is estimated to incur losses of up to 70% during the purification step alone.

Read the full report: https://www.reportlinker.com/p05950929/?utm_source=PRN

A number of techniques are presently used for viral vector purification. Over the years, size-based viral purification strategies, such as density-gradient ultracentrifugation, ultrafiltration, precipitation and size-exclusion chromatography (SEC), have become part of the accepted industry standard. However, recently, stakeholders have begun relying more on affinity chromatography-based purification regimens, given its robustness and high selectivity. Presently, several companies claim to offer a diverse range of virus purification solutions, including, filter plates, prepacked chromatography columns and resins, and consolidated kits, for viral vector (virus) purification. As indicated earlier, downstream processing of viral vector products is challenging. Existing separation and purification techniques are not efficient when it comes to purifying viral vectors at large scales. Moreover, these techniques are often unable to separate complete viruses from empty capsids, thereby, compromising product recovery. Therefore, it has become essential for stakeholders to develop innovative ways to optimize the purification process, in order to further improve virus recovery and facilitate effective removal of contaminants / impurities. Currently, the viral vector purification products market is driven by abovementioned surge in demand for viral vectors. In this context, it is worth mentioning that there are several virus based vaccines under development against the novel SARS-CoV-2 coronavirus strain. In fact, according to the WHO, there are over 20 viral vaccines currently being investigated against the novel coronavirus. Developing the means to better purify viruses is, therefore, likely to significantly impact viable vaccine yield. The current crisis is definitely going to have a strong impact on the virus purification products market.

SCOPE OF THE REPORT The 'Viral Vector Purification / Virus Purification Products Market (kit, prepacked column, resin, cassette, filter plate, capsule, reagent and others), 2020-2030' report features an extensive study of various products available for viral vector purification, in addition to the current market landscape and future potential of product developers.

Amongst other elements, the report features: - An overview of the current market landscape of companies providing products for purification of viruses / viral vectors, using different techniques, such as chromatography, centrifugation and filtration. It features information on the type of product (kit, prepacked column, resin, cassette, filter plate, capsule and reagent), type of purification technique (chromatography, centrifugation and filtration), scale of operation (lab-scale, clinical and commercial), type of viral vector (AAV, adenovirus, lentivirus, retrovirus and others) and details on other physical and operational parameters of the product (such as matrix, pore size, volume of bed, flow rate, operating pressure, working temperature, pH, filtration area and process time). In addition, the chapter includes information on the purification product developers, including details on the year of establishment, company size and location of headquarters. - Elaborate profiles of key players, including an overview of the company, product portfolio (viral vector purification products), recent developments and an informed future outlook. - An analysis evaluating the potential strategic partners (comprising of viral vector-based therapy developers and viral vector manufacturers) for viral vector purification product developers, based on several parameters, such as type of viral vector, developer strength, operational strength, therapeutic area, strength of clinical pipeline and strength of preclinical pipeline. - A clinical trial analysis of completed, ongoing and planned studies of various viral vector-based cell therapies, gene therapies and vaccines (approved / under development). It features detailed analyses of clinical studies of different viral-vector based therapies on the basis of their registration year, phase of development, trial status, type of therapy, therapeutic area, type of sponsor / collaborator, geographical location, number of patients enrolled and key players. - An informed estimate of the annual clinical and commercial demand (in terms of number of patients) for viral vectors, taking into account the marketed gene-based therapies and clinical studies evaluating vector-based therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength. Further, the demand has been segregated on the basis of type of viral vector, type of therapy, therapeutic are and geographical location. - A case study on tangential flow filtration (TFF), highlighting the role, advantages and disadvantages of the technique for purification of viral vectors; the chapter features details of products used for TFF, including product type, scale of operation, membrane material, flow rate and filtration area. - A case study featuring the viral vector manufacturers providing commercial scale production, highlighting details on their year of establishment, company size, type of viral vector (AAV, adenovirus, lentivirus, retrovirus and others), purpose of production (in-house and contract-basis), and location of headquarters and manufacturing facilities.

One of the key objectives of the report was to estimate the existing market size and identify potential growth opportunities for viral vector purification product developers, over the coming decade. Based on various parameters, such as the likely increase in number of clinical studies related to viral vector-based therapies, anticipated growth in target patient population, existing price variations across different purification techniques, and the success of cell and gene therapy products (considering both approved and late-stage clinical candidates), we have provided an informed estimate of the likely evolution of the market in the short to mid-term and long term, for the period 2020-2030. In order to provide a detailed future outlook, our projections have been segmented on the basis of [A] type of viral vector (AAV, adenovirus, lentivirus, retrovirus and others), [B] type of purification technique (chromatography, centrifugation and filtration), [C] type of therapy (gene therapy, cell therapy and viral vaccines) [D] therapeutic area (oncological disorders, cardiovascular disorders, ophthalmic disorders, metabolic disorders, inflammation & immunological diseases and others), [E] scale of operation (preclinical / clinical and commercial) and [F] key geographical regions (North America, Europe, Asia Pacific and rest of the world).

The research, analysis and insights presented in this report are backed by a deep understanding of key insights gathered from both secondary and primary research. The opinions and insights presented in the report were influenced by discussions held with several players in this industry. The study includes detailed transcripts of discussions held with the following individuals: - Haifeng Chen (Chief Executive Officer, Virovek) - Jeffrey Hung (Chief Commercial Officer, Vigene Biosciences) - Kai Lipinski (Chief Scientific Officer, Vibalogics)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information. The secondary sources of information include - Annual reports - Investor presentations - SEC filings - Industry databases - News releases from company websites - Government policy documents - Industry analysts' views While the focus has been on forecasting the market till 2030, the report also provides our independent view on various non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various secondary and primary sources of information.

CHAPTER OUTLINES Chapter 2 provides an executive summary of the insights captured in our research. It offers a high-level view on the likely evolution of the viral vector purification product in the short to mid-term, and long term.

Chapter 3 provides a general overview of various types of viral and non-viral vectors. It includes a detailed discussion on the methods of gene transfer, type of viral vectors and applications of viral vectors in various advanced therapies. It provides information on the different stages of the viral vector purification process, type of purification techniques used and the key challenges associated with the purification of viral vectors. Further, it includes a review of the latest trends and innovations in the vector purification market.

Chapter 4 is an overview of the various types of products used for purification techniques based on principles of chromatography, centrifugation and filtration. It highlights product specific information, such as type of product (kit, prepacked column, resin, cassette, filter plate, capsule and reagent), type of purification technique (chromatography, centrifugation and filtration), scale of operation (lab-scale, clinical and commercial), type of viral vector (adenovirus, AAV, lentivirus, retrovirus and others), type of chromatographic technique (affinity chromatography, ion-exchange chromatography, size-exclusion chromatography and hydrophilic interaction chromatography), pack size (wherever specified) and price (wherever specified). In addition, the chapter provides details on certain additional parameters (based on the purification technique, wherever applicable), such as type of matrix, binding capacity, particle size, volume of bed, flow rate, operating pressure, working temperature, process time and pH. The chapter also provides information on the purification product developers, including year of establishment, company size and location of headquarters.

Chapter 5 includes detailed profiles of players offering products for viral vector purification. Each profile features an overview of the company, , information on its product portfolio, recent developments and an informed future outlook. Chapter 6 highlights potential strategic partners (comprising of viral vector-based therapy developers and viral vector manufacturers) for viral vector purification product developers, based on multiple parameters, such as type of viral vector, developer strength, operational strength, therapeutic areas, strength of clinical pipeline and strength of preclinical pipeline. The analysis aims to provide the necessary inputs to the product developers, enabling them to make the right decisions to collaborate with industry stakeholders with relatively more initiatives in the domain.

Chapter 7 features a geographical clinical trial analysis of completed, ongoing and planned studies of viral vector-based cell therapies, gene therapies and vaccines (approved / under development). The analysis provides details related to the different types of viral vectors investigated / being investigated across various geographies, based on the number of clinical trials, registration year, phase of development, trial status, type of therapy, therapeutic area, type of sponsor / collaborator, number of patients enrolled, key players and type of viral vector.

Chapter 8 features an informed estimate of the annual demand for viral vectors, taking into account the viral vector based marketed therapies and clinical studies. For the purpose of estimating the current clinical demand, we considered the active clinical studies of different types of vector-based therapies that have been registered till date. The data was analyzed on the basis of various parameters, such as number of annual clinical doses, trial location, and the enrolled patient population across different geographies. Further, in order to estimate the commercial demand, we considered the marketed vector-based therapies, based on various parameters, such as target patient population, dosing frequency and dose strength. The chapter features analyses demand on the basis of type of viral vector, type of therapy, therapeutic are and geographical location.

Chapter 9 is a detailed case study which provides an overview of the tangential flow filtration, highlighting its role, advantages and disadvantages. It features key details of the products used for tangential flow filtration, such as type of product, scale of operation, membrane material, flow rate and filtration area.

Chapter 10 is a detailed case study which provides an overview of the viral vector manufacturers. It features an in-depth analysis of the commercial scale viral vector manufacturers, highlighting details on their year of establishment, company size, types of vectors (AAV, adenovirus, lentivirus, retrovirus and others), purpose of production (in-house and contract-basis), headquarters and manufacturing facilities.

Chapter 11 presents an insightful market forecast analysis, highlighting the future potential of the virus purification products market till the year 2030. We have segregated the opportunity of the market on the basis of [A] type of viral vector (AAV, adenovirus, lentivirus, retrovirus and others), [B] type of purification technique (chromatography, centrifugation and filtration), [C] type of therapy (gene therapy, cell therapy and viral vaccines) [D] therapeutic area (oncological disorders, cardiovascular disorders, ophthalmic disorders, metabolic disorders, inflammation & immunological diseases and others), [E] scale of operation (preclinical / clinical and commercial) and [F] geographical location (North America, Europe, Asia Pacific and rest of the world).

Chapter 12 summarizes the overall report. In this chapter, we have provided a list of key takeaways from the report, and expressed our independent opinion related to the research and analysis described in the previous chapters.

Chapter 13 is a collection of interview transcripts of the discussions held with key stakeholders in this market. In this chapter, we have presented the details of our conversations with (in alphabetical order) Haifeng Chen (Chief Executive Officer, Virovek), Jeffrey Hung (Chief Commercial Officer, Vigene Biosciences) and Kai Lipinski (Chief Scientific Officer, Vibalogics).

Chapter 14 is an appendix, which provides tabulated data and numbers for all the figures provided in the report.

Chapter 15 is an appendix, which provides a list of companies and organizations mentioned in this report.

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Every week, some of the most exciting tech companies in the world post their latest and greatest job opportunities on Jobs.VentureBeat, powered by Jobbio. From transformative tech to gaming and beyond, Jobs.VentureBeat helps businesses from all over the US hire top tech talent.

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AveXis is advancing cutting-edge science, starting with our proprietary gene therapy for the treatment of spinal muscular atrophy.

They are in the midst of an incredible journey and are looking for passionate individuals to join them on this important mission. AveXis, a Novartis company, is dedicated to developing and commercialising novel treatments for patients suffering from rare and life-threatening neurological genetic diseases. In addition to developing a treatment for SMA, AveXis also plans to develop other novel treatments for rare neurological diseases, including Rett syndrome and a genetic form of amyotrophic lateral sclerosis caused by mutations in the superoxide dismutase 1 (SOD1) gene.

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Companies of the week: Here are some brilliant transformative tech companies that are hiring right now - VentureBeat

HONG KONG Singapore-based Tessa Therapeutics Pte. Ltd. released the results of two investigator-initiated phase I/II trials that found its autologous CD30 CAR T-cell therapy TT-11 showed a high rate of durable complete responses and a favorable safety profile in patients with relapsed/refractory Hodgkin lymphoma.

The studies, funded by Tessa, were led by researchers at the Baylor College of Medicine and the University of North Carolina (UNC) Lineberger Comprehensive Cancer Center.

The trials enrolled 41 adult patients who received CD30 CAR T-cell therapy following lymphodepletion with chemotherapy. The overall response rate in the 32 patients with active disease who received fludarabine-based lymphodepletion was 72%, including 19 patients (59%) with complete responses.

Among those who participated in the studies, 94% were still alive a year after treatment and 61% of those who had a complete response also reported no evidence of recurrence a year later.

None of the patients experienced complications that have been seen with several CD19 CAR T cell trials. The results have been published in the Journal of Clinical Oncology.

These data are significant, as they demonstrate that CAR T-cell therapy may be a safe and effective treatment option for patients with Hodgkin lymphoma and potentially other lymphomas expressing the CD30 antigen, said Natalie Grover, study co-first author, assistant professor in the UNC Department of Medicine and a UNC Lineberger member.

The highest dose treatment led to the complete disappearance of tumors in the majority of patients, and almost all subjects had clinical benefit. As such, we believe further study of this treatment approach is warranted, said Carlos Ramos, study co-first author, professor at the Center for Cell and Gene Therapy at Baylor College of Medicine, Houston Methodist Hospital and Texas Childrens Hospital.

Expanded testing to come

Ivan Horak, Tessas president of R&D, told BioWorld that the company plans to further explore the results in a Tessa-sponsored regulatory phase II trial, which it aims to initiate this year. The TT-11 trial would be conducted out of 22-plus sites in U.S. and Europe, he said.

The companys clinical trial partners include institutions in the U.S. such as MD Anderson, Baylor College of Medicine, Stanford Cancer Institute, City of Hope National Medical Center, UCSF Medical Center and Baylor Scott & White Health, as well as other centers in Massachusetts, Texas and California.

The University of North Carolina has granted Tessa an exclusive license to its patents, data and know-how, while Baylor College of Medicine has granted Tessa the rights to use its data and know-how, for the further development and commercialization of this therapy. Tessa declined to speak on the financial arrangements given confidentiality considerations.

Tessa's pipeline currently has four IND-approved clinical trials, covering the evaluation of TT-11 in Hodgkins lymphoma the companys lead program, which has a regenerative medicine advanced therapy designation and non-Hodgkin lymphomas, as well as trials for TT-11X and TT-16. It also has one preclinical program for a candidate known as TT-20X, which will explore allogeneic dual CAR Epstein-Bar virus-specific T cells in solid tumors. The company also plans to test TT-11 (HL) in pediatric patients, Horak said.

TT-11X, the companys allogeneic candidate, combines the properties of CD30-CAR and virus specific T-cells (VSTs) in an allogeneic setting. VSTs can be used for allogeneic application without T-cell antigen receptor (TCR) deletion or other genetic modifications, with a minimal risk of graft rejection, Horak said. CD30-CAR as a target has shown strong efficacy in Hodgkin lymphoma and further increases the persistence and expansion of VST cells, he said.

TT-16 is in an upcoming investigator-initiated phase I trial in HER2-positive solid tumors. The FDA has approved the trial to commence at Baylor College of Medicine.

Tessa also is building an in-house 90,000-square-foot GMP facility, which will be one of the companys commercial-scale cell therapy manufacturing facilities in Asia.

More:
Tessa enjoys positive results from two phase I/II trials of CD30 CAR T-cell therapy - BioWorld Online

RALEIGH Beam Therapeutics, a Boston-based biotechnology company developing precision medicines through DNA base editing, plans to build a $83 million manufacturing facility in North Carolinas Research Triangle Park, it was announced today.

The uptick: the addition of 201 jobs with an average salary of around$102,654.

North Carolina is a leader in biotechnology, from the research in our labs to the states biomanufacturers, said Governor Roy Cooper, in a statement.Companies like Beam Therapeutics work in developing precision medicines will help keep North Carolina on the cutting edge of this industry.

Beam is developing precision genetic medicines through a pioneering CRISPR technology known as base editing. Its using this proprietary gene editing approach to create therapies aimed at cures for serious diseases with precise edits to single base pairs in DNA and RNA.

Its targeting gene therapies in the fields of hematology, oncology, liver diseases, and ocular and central nervous system (CNS) diseases. The company believes its approach provides greater control of the gene editing process with a lower risk of unintended off-target effects. Based on the most effective method for each target organ, Beam uses a range of gene delivery strategies, including electroporation for blood and immune cells, non-viral (LNP) for liver and potentially other organs, and viral (AAV) for delivery to the eye and CNS.

Backstory: A look at RTPs growing gene therapy hub as startup brings 200+ $102,000 jobs to Durham

Beams technology platform incorporates gene editing and gene delivery modalities found across North Carolinas broad and growing cell- and gene-based therapy community. In the gene editing space, North Carolina has both home-grown companies like Precision BioSciences and production-phase companies including Cellectis.

We believe investment in strategic manufacturing capabilitiesis an important component of fully realizing the power of our base editing technology and achieving our vision to provide life-long cures to patients suffering from serious diseases, said John Evans, CEO of Beam Therapeutics, in a statement. Research Triangle Park is a thriving biopharmaceutical hub, providing significant access to the broad range of talent we will need to make this vision a reality.

Beam Therapeutics project in North Carolina will be facilitated, in part, by a Job Development Investment Grant (JDIG) approved by the states Economic Investment Committee earlier today.

The agreement authorizes the potential reimbursement to the company of up to $3.2 million spread over 12 years.

NC Biotech Center contributed to this report.

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Beam Therapeutics to build $83M manufacturing facility in RTP, create hundreds of jobs - WRAL Tech Wire