Category Archives: Genetic Therapy

LEIDEN, Netherlands & CAMBRIDGE, Mass., April 26, 2022 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the Company), a company dedicated to changing lives through the creation of transformative RNA therapies, today announced several presentations at the 7th Annual Retinal Cell and Gene Therapy Innovation Summit being held Friday, April 29, 2022 and the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) being held May 1-4, 2022, both in Denver, CO, U.S.

Presentations at the Retinal Cell and Gene Therapy Innovation Summit

Presentation title: AON treatment for CEP290-LCA: Efficacy, safety, and durabilityPresenter: Dr. Artur Cideciyan, University of PennsylvaniaPresentation type: Pre-recorded talkSession: Session 4: Antisense Oligonucleotide TherapyDate: April 29, 2022 at 4:00pm MDT

Presentation title: Efficacy and safety of sepofarsen, an intravitreal RNA antisense oligonucleotide, for the treatment of CEP290-associated inherited retinal disease called Leber congenital amaurosis (LCA10): A randomized, double-masked, sham-controlled, phase III study (ILLUMINATE)Presenter: Dr. Bart Leroy, Ghent University HospitalPresentation type: Oral presentationSession: Session 4: Antisense Oligonucleotide TherapyDate: April 29, 2022 at 4:15pm MDT

Presentations at ARVO

Presentation title: Long-term safety and efficacy of sepofarsen in a Ph1b/2 INSIGHT extension trial in CEP290-associated Leber congenital amaurosis (LCA10)Presenter: Dr. Stephen R. Russell, Iowa UniversityPresentation type: Oral presentationDate: May 1, 2022 at 3:36pm MDT

Presentation title: QR-1011 corrects splicing in the Stargardt disease type 1-causing variant ABCA4 c.5461-10T>CPresenter: Melita Kaltak, ProQR TherapeuticsPresentation type: Oral presentationDate: May 4, 2022 at 10:34am MDT

Presentation title: Efficacy and safety of sepofarsen, an intravitreal RNA antisense oligonucleotide, for the treatment of CEP290-associated Leber congenital amaurosis (LCA10): a randomized, double-masked, sham-controlled, Phase 3 study (ILLUMINATE)Presenter: Dr. Bart Leroy, Ghent University HospitalPresentation type: Poster presentationDate: May 4, 2022 at 3:00pm MDT

CEP290-mediated Leber congenital amaurosis 10, or LCA10, is a difficult to treat ultra-rare inherited retinal disease for which there is currently no treatment, said Dr. Bart Leroy, Head of the Ophthalmology Department and Professor of Ophthalmology and Ophthalmic Genetics at Ghent University in Belgium and Attending Physician at The Children's Hospital of Philadelphia. After missing the primary endpoint in the Illuminate Phase 2/3 clinical trial, sepofarsen showed an encouraging efficacy signal across multiple endpoints in post-hoc analyses comparing sepofarsen treated and sham treated eyes to their corresponding untreated contralateral eyes. I look forward to continuing to work with ProQR on this investigational treatment.

About Leber congenital amaurosis 10 (LCA10)

Leber congenital amaurosis (LCA) is the most common cause of blindness due to genetic disease in children. It consists of a group of diseases of which LCA10 is the most frequent and one of the most severe forms. LCA10 is caused by mutations in the CEP290 gene, of which the c.2991+1655A>G (p.Cys998X) mutation has the highest prevalence. LCA10 leads to early loss of vision causing most people to lose their sight in the first few years of life. To date, there are no treatments approved that treat the underlying cause of the disease. Approximately 2,000 people in the Western world have LCA10 because of this mutation.

About sepofarsen

Sepofarsen (QR-110) is an investigational RNA therapy designed to restore vision in Leber congenital amaurosis 10 due to the c.2991+1655A>G mutation (p.Cys998X) in the CEP290 gene. The mutation leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to enable normal splicing, resulting in restoration of normal (wild type) CEP290 mRNA and subsequent production of functional CEP290 protein. Sepofarsen is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation and rare pediatric disease designation from the FDA as well as access to the PRIME scheme by the EMA.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA therapies. ProQR is pioneering a next-generation RNA technology called Axiomer, which uses a cells own editing machinery called ADAR (adenosine deaminase acting on RNA) to make specific single nucleotide edits in RNA to reverse a mutation or modulate protein expression and could potentially yield a new class of medicines for genetic diseases. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.Learn more about ProQR at http://www.proqr.com.

FORWARD-LOOKING STATEMENTS

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding participation in these conferences as well as sepofarsen (QR-110) and the clinical development and the therapeutic potential thereof, and our planned interactions with regulatory authorities relating to our programs. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities by us and our collaborative partners whose operations and activities may be slowed or halted by the COVID-19 pandemic; the likelihood of our clinical programs being executed on timelines provided and reliance on our contract research organizations and predictability of timely enrollment of subjects and patients to advance our clinical trials and maintain their own operations; our reliance on contract manufacturers to supply materials for research and development and the risk of supply interruption from a contract manufacturer; the potential for later data to alter initial and preliminary results of early-stage clinical trials, including as a result of differences in the trial designs and protocols across different trials; the unpredictability of the duration and results of the regulatory review of applications or clearances that are necessary to initiate and continue to advance and progress our clinical programs; the outcomes of our planned interactions with regulatory authorities; the ability to secure, maintain and realize the intended benefits of collaborations with partners; the possible impairment of, inability to obtain, and costs to obtain intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in research and development; our ability to maintain and service our loan facility with Pontifax and Kreos; general business, operational, financial and accounting risks; and risks related to litigation and disputes with third parties. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future, except as required by law.

ProQR Therapeutics N.V.

Investor Contact:Sarah KielyProQR Therapeutics N.V.T: +1 617 599 6228skiely@proqr.comorHans VitzthumLifeSci AdvisorsT: +1 617 430 7578hans@lifesciadvisors.com

Media Contact:Robert StanislaroFTI ConsultingT: +1 212 850 5657robert.stanislaro@fticonsulting.com

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ProQR to Present at the Retinal Cell and Gene Therapy Innovation Summit and the Association for Research in Vision and Ophthalmology (ARVO) 2022 -...

The trends in the hemophilia treatment market are gaining momentum with increasing incidences of hemophilia A and B and staggering need for their treatments, including gene, anti-tissue factor pathway inhibitor, and factor replacement therapies.

With novel developments in hemophilia treatment progressing at an unprecedented pace, the industry is touted to seize an overall remuneration of USD 16 billion by 2028 end- Global Market Insights Inc.

The trends in the hemophilia treatment market are gaining momentum with increasing incidences of hemophilia A and B and staggering need for their treatments (Figure). As per reliable estimates, the global prevalence of hemophilia A is announced to be approximately 1 case per 5000 males, with nearly 1 third of affected individuals not having a family history of the blood disorder. The rare disease frequency also varies with the reporting country, with a range of 5.4 to 14.5 cases per 100,000 males.

Hemophilia is a rare condition for which the approved treatment options have remained practically unchanged for quite a while now. Recently, however, the hemophilia treatment market is observing an explosion of innovation in the treatment options that are either under development or have already been approved, including some medications and injections.

In 2018, the FDA had approved emicizumab-kxwh which was intended to reduce or avert the frequency of bleeding episodes in people with hemophilia A. Some other options might include desmopressin, a manufactured hormone that stimulates the release of stored factor 8, and antifibrinolytic medications, that prevent clots from breaking down.

While oral medication and injections are recognized to treat the condition, its prolonged impact on human health or side effects are not known in detail. In this case, another welcome leap in the business space is the advancement in therapy options.

Given below are some of the latest advancements in the hemophilia treatment that are expected to push different economies toward healthy living and reduce the prevalence of the rare disease:

Gene Therapy

Since hemophilia is coined as a genetic disorder, gene therapy for its treatment could emerge as a possible breakthrough in the health care fraternity. The treatment offers a potential cure for hemophilia patients by establishing constant endogenic expression of factor 8 or 9 following transmission of functional gene to swap the hemophilic patients own flawed gene.

While only about 25% to 30% of the worlds hemophilia population has access to factor replacement owing to its costly alternative, complications, and burden, the ability of gene therapy solution for hemophilia delivers hope for more global admittance to the treatment.

Having said that, results from one of the recent trials of the approach have been known to restore patients anticoagulant factor activity levels to near normal or normal levels and dropped down their annualized bleeding rates by almost 90%.

Anti-TFPI Therapy

Anti-tissue factor pathway inhibitor, or anti-TFPI, therapy is an advanced treatment that seeks to reduce bleeding by decreasing on the system that averts the blood from clotting too much. This therapy restores hemostatic balance by blocking one of the anticoagulants and preventing it from functioning normally.

Considering its success trail, different biopharmaceutical firms are now researching and analyzing ways to incorporate anti-TFPI therapy into their portfolio of hemophilia treatment. One of these companies is Pfizer.

The leading pharmaceutical firm recently announced dosing its first participant in the Phase 3 BASIS study of marstacimab, an anti-TFPI being evaluated for the treatment of people suffering from severe hemophilia A or B. The completed phase 2 study results represented that the treatment with marstacimab demonstrate more than 75% reductions in annual bleeding rates for almost all the participants in the study population.

Factor Replacement Therapy

The factor replacement therapy is deemed as one of the efficient therapies to treat hemophilia. In this, the clotting factors are injected into veins to stop severe blood loss and problems from bleeding such as joints, organs, and muscles. These therapies have been widely used before surgery or operation to avoid excessive blood loss.

According to a recent study by Global Market Insights Inc., it was revealed that the factor replacement therapy surpassed USD 9,412 million in 2021, in terms of valuation, across the overall industry. It is further anticipated to hold a prominent position worldwide owing to the ongoing R&Ds and advancements in the space.

To illustrate, the US FDA announced approval of Factor VII treatment for hemophilia A and B with inhibitors.

In a nutshell, increasing prevalence of hemophilia and surging need for associated treatment therapies globally would help hemophilia treatment market growth curve to ascend significantly through 2028.

Reference

Hemophilia treatment market size by disease (hemophilia a {severe, moderate, mild}, hemophilia b {severe, moderate, mild}), by product (recombinant factor concentrates {factor VIII, factor IX}, plasma-derived factor concentrates {factor VIII, factor IX}, extended half-life products {factor VIII, factor IX}), by patient (pediatric {0 to 4, 5 to 13, 14 to 18}, adult {19 to 44, 45+}), by treatment (prophylaxis, on demand), by therapy (factor replacement therapy, non-factor replacement therapy), by drug class (vasopressin, coagulation factors), by route of administration (injectable, nasal spray), by end-use (hospitals, clinics, hemophilia treatment centers), industry analysis report, regional outlook, application potential, COVID-19 impact analysis, price trends, competitive market share & forecast, 2022 2028. Global Markets Insights website. gminsights.com/industry-analysis/hemophilia-treatment-market. Publication February 2022. Accessed April 2022.

Link:
Contributor: Top 3 Future Therapies to Drive Hemophilia Treatment Market Growth - AJMC.com Managed Markets Network

CAR-T therapies have revolutionised the treatment of some blood cancers, but are costly as well as time- and resource-intensive. Now, US researchers think one solution could be an implant that generates T cells within the body to attack the cancer.

It is early days for the concept, but its developers from North Carolina State University and the University of North Carolina have completed a proof-of-concept study that shows it works in animal models and could start to attack the cancer in a single day.

At the moment, it can take weeks to complete the CAR-T process, which includes harvesting of T cells from the patient an shipment to a manufacturing unit, engineering, activation, and expansion of the cells, quality control checks and shipment back to the hospital for re-infusion into the patient.

In some cases where the cancer is particularly aggressive, that timeframe may simply be too long to be a viable option for the patient. It is also expensive, at up to $500,000 per procedure.

The UNC and NCSU team, led by biomedical engineer Yevgeny Brudno, report in Nature Biotechnology how they have developed an implant that can generate the CAR-T cells in vivo.

It is based on an FDA-approved biocompatible, sponge-like material called MASTER, which has antibodies that activate T cells as well as interleukins that promote proliferation.

A mixture of T cells isolated from the human donor and viral particles to engineer them is introduced into the sponge, which is then surgically implanted into the patient the same day. The T cells are modified, activated and proliferate all within the implant from which they are steadily released as fully functional CAR-T cells to attack the tumour.

In a mouse model carrying human lymphoma cells, the CAR-T cells made in the implants were better at fighting the cancer cells than a control group manufactured using the current production method and delivered intravenously.

They were also healthier, with superior sustainability in the body and more anticancer potency, and showed fewer signs of differentiation or other cellular changes associated with T-cell exhaustion, a condition in which they lose activity.

The CAR-T cells targeted CD19, working in a similar way to current commercial CAR-Ts from Gilead Sciences, Novartis and Bristol-Myers Squibb.

Our MASTER technology takes the cumbersome and time-consuming activation, reprogramming and expansion steps and performs them inside the patient, said Dr Pritha Agarwalla, one of the researchers behind the work.

The large pores and sponge-like nature of the MASTER material brings the virus and cells close together, which facilitates cellular genetic reprogramming, she added.

The researchers also found that the improvement in anticancer efficacy was particularly noticeable over the long term, when mice were reimplanted with lymphoma cells to represent a recurrence.

They now want to test the implant approach to see how it performs against solid tumours, which have proved to be a challenging target for current CAR-T therapies.

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Implants could transform CAR-T therapy into one-day procedure - - pharmaphorum

Compared to a critical organ that had been largely ignored, companies working to develop microbiome-based therapies believe there is untapped potential in the area. Ben Hargreaves speaks to companies that are developing therapies not just in gastrointestinal conditions but also in broader areas, such an immuno-oncology.

The interest in how the human microbiome influences health has grown substantially in the last decade. PubMed cites 1,822 pieces of research relating to the microbiome in 2011, which grew to 25,153 published in 2021. As more research has been conducted, closer and unexpected links have been found between the microbiome of the gut, as one example, and other areas of the body, such as brain health and the immune system.

The microbiome, as a term, can be related to the nasal, oral, skin, gastro-intestinal, and urogenital regions of the human body, each containing varied microbial communities that have an impact on health. Research into the area is being made possible through advancements in technology, such as being able to sequence genes at scale, allowing researchers to gain more detailed genetic data on the microbiome.

With more research being published, there has arrived greater interest in how individual microbiomes could influence health and there is now a growing market of prebiotic, probiotic and postbiotic commercial products based on the links being made to various health conditions. At the same time, the pharma industry has also started to note the potential for a new treatment paradigm to be developed, and investment into the space has started to increase. As a result, pharma companies such as Merck, Gilead, Genentech, Johnson & Johnson and Takeda have positioned themselves alongside partners to benefit from a potential new wave of treatments.

More recent research has also suggested that there are links to the health of the heart and to the brain, through an impact on mental health.

The possibilities

The gut contains approximately one trillion microbes and at least 160 bacterial species that are constantly interacting with one another and with the cell lining of the gut. The gut microbiome has long been linked to gastrointestinal conditions, such as inflammatory bowel disease and irritable bowel diseases, as well as obesity and type 2 diabetes. More recent research has also suggested that there are links to the health of the heart and to the brain, through an impact on mental health, with the latter becoming so established as to become known as the gut-brain axis.

Due to the variety of potential indications that can be targeted in ensuring a healthy gut, the work being carried out by pharma and biotech companies in the space is often concentrated in this area. Seres Therapeutics is a biotech that has specialised in the microbiome space and also possesses one of the assets closest to potential approval in the overall pipeline. Its treatment candidate, known as SER-109, is targeting patients with recurrent C. difficile infection (CDI).

The treatment has been granted US FDA breakthrough therapy designation and orphan drug designation, and has also completed a phase 3 clinical study. In the trial, Seres announced that the product candidate had met its primary endpoint, showing a statistically significant 30.2% absolute reduction in the rate of CDI recurrence compared to placebo. As a result, the treatment candidate is the most advanced one in the overall microbiome pipeline, with an approval holding the potential to advance the entire field, as well as Seres own prospects. Speaking to pharmaphorum, the companys CEO, Eric Shaff, said, Our investigational SER-109 has clearly validated our approach, and there is tremendous opportunity to apply our microbiome therapeutics more broadly to infection protection across multiple patient populations and disease areas.

Mid-way through last year, Nestl confirmed its conviction in the project by consolidating its existing partnership with Seres through paying $175 million upfront for North American co-marketing rights to the potential microbiome therapy. The biotech is eligible to receive a further $125 million if the FDA approves the drug and could receive another $225 million in commercial milestones. If approved by the FDA, the treatment would become the first microbiome-based treatment to receive approval.

In regard to future work, Shaff stated, Given what weve learned from our lead investigational program, SER-109, were especially focused on how to use microbiome therapeutics to provide infection protection to medically compromised patients.

Eyes bigger than the belly

Even though many microbiome-based therapies are looking to gastrointestinal conditions, the rest of the pipeline is much broader. Finch Therapeutics, which is partnered with Takeda on several drug candidates, is developing FIN-211 for autism spectrum disorder (ASD). The company states that a subset of individuals with the condition also exhibit the most common gastrointestinal (GI) disorders, with the expectation that treatment could address both the GI and behavioural symptoms by targeting multiple ASD-relevant pathways.

4D Pharma, another biotech working in the space, has a pipeline of products that predominantly target either immuno-oncology or central system disorders indications. There is now a growing body of research to suggest that the microbiome of a patient can impact responses to immunotherapy. The potential was enough for both Pfizer and Merck to decide to collaborate with 4D to test combination treatments of their immunotherapies, Bavencio (avelumab) and Keytruda (pembrolizumab), respectively. Both treatments will be tested alongside MRx0518, which is also being tested as a monotherapy, after 4D found that the microbiome therapy was able to increase key immune biomarkers associated with anti-cancer immune response and responses to therapy and was associated with better clinical outcomes in early trials.

Compelling advantages

A spokesperson for 4D Pharma explained to pharmaphorum that microbiome-based therapies, or Live Biotherapeutic Products (LBPs) as they are also known, are attractive to use in combination due to their safety profile.

As naturally occurring non-engineered strains of human commensal bacteria originally isolated from healthy human donors, they are expected to have very clean safety profiles, they said. This also means LBPs are attractive as combination therapies, driving better efficacy without additional toxicity concerns.

Beyond this, the spokesperson added that LBPs could go beyond what is therapeutically possible with traditional small molecules or biologics. This is due to their ability to hit multiple targets with the same drug, which, alongside the expected safety profile of the treatments, could offer advantages over existing treatments.

More broadly, the microbiome can be thought of as analogous to a critical organ that we previously overlooked or underappreciated, but is involved in the proper functioning of a wide range of human systems from metabolism to immunology (including immuno-oncology) and neurology via the gut-brain axis. By understanding these impacts, the microbiome offers a whole new way to think about treating many different diseases, the spokesperson concluded.

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Understanding the microbiome as an 'overlooked critical organ' - - pharmaphorum

By Reka Geczy, PhD, Aruna Balgi, Stella Park, Rita Zhao, Ethan Watt, Maggie Wong, Cooper Webb, Nikita Jain, PhD, Angela Zhang, PhD, Anitha Thomas, PhD, Samuel Clarke, PhD

The expression of the chimeric antigen receptor (CAR) on T cells turns a patients cells into cell-based cancer therapies and has revolutionized cancer treatment today1. Despite its successes and high response rates, evidence suggests an increasing need for more complex genetic engineering enabled by CRISPR/Cas-mediated genome editing technologies. Such examples include the disruption of inhibitory pathways exploited by the tumor microenvironment2, 3, improvement of CAR T cell efficiency4, 5, and manufacturing of universal CAR T cells from allogeneic donors6, 7. The desire to achieve both gene editing and transgene expression in next-generation T cell therapies emphasizes the significance of the genetic material delivery method, which plays a critical role in cell function, cell yield, ease of production, and scale-up.

A promising new approach for T cell engineering is the use of RNA to express therapeutic proteins and gene editing nucleases. RNA is typically delivered to cells using electroporation; however, the sequential electrical pulses for multi-step gene engineering leads to a dramatic trade-off between efficiency and cell viability. This type of trade-off is not observed with lipid nanoparticles (LNPs), making it an attractive alternative for RNA delivery. LNPs are entirely synthetic lipid formulations designed to encapsulate and protect RNA before delivering it into cells. The production of LNPs is well-established and is scalable for large-scale gene delivery and gene editing, which are key to meeting clinical demand now and in the future. The RNA-LNP complex structurally resembles low density lipoproteins (LDL) and can co-opt the endogenous uptake pathway of LDL to enter cells using receptor-mediated endocytosis. This gentle uptake mechanism enables successful genome engineering of T cells while maintaining high cell viability.

Herein, we report a novel method for sequential genetic engineering of T cells using the GenVoy-ILM T Cell Kit for mRNA. We utilized a manufacturing workflow optimized to deliver various RNA cargoes. In this case study, we show CRISPR/Cas9-mediated knockouts (KO) of the T cell receptor (TCR) and explore multi-step LNP engineering to produce TCR KO CAR T cells, a promising approach towards allogeneic CAR T cell therapy6, 8, 9. We describe in detail LNP production and cell culture treatment protocols, as well as optimization strategies for T cell gene editing to ensure success with the GenVoy-ILM T Cell Kit for mRNA.

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Genome Editing of Human Primary T Cells With Lipid Nanoparticles - BioProcess Online

Shares of Taysha Gene Therapies are down more than 10% after apatient'sdeath marred positive interim data for the companys experimental gene therapy treatment for Sandhoff and Tay-Sachs diseases, which are two forms of GM2 gangliosidosis.

The first patient treated with Texas-based Tayshas TSHA-101 was diagnosed with Sandhoff disease, arare inherited disorder that progressively destroys brain and spinal cord nerve cells. After administering TSHA-101, an investigational gene therapy that delivers both the HEXA and HEXB genes that make up the -hexosaminidase A enzyme, the patient demonstrated clinical improvement after three months. The patient was cleared to travel home. However, Taysha said the unvaccinated patient was exposed to a family member who was symptomatic of an upper respiratory infection, which was likely COVID-19. The Taysha patient was hospitalized with pneumonia. While there, the patient contracted a secondary hospital-acquired methicillin-resistant staphylococcus aureus (MRSA) infection and ultimately succumbed.

Trial investigators have made the initial assessment that the patients death was not likely due to the gene therapy. However, according to the company, a review from the independent data safety monitoring board is being conducted and results are anticipated in the near term.

Prior to the patients death, Taysha said their disease had realized normalization of Hex A enzyme activity by the end of the first month following treatment with the gene therapy. The levels were 58-fold above the presumed asymptomatic level of 5% of normal identified by natural history at Month 3, the company said. According to available data, the patient achieves Hex A enzyme activity of 190% and 288% of normal at month 1 and month 3, respectively.

The second patient to receive the gene therapy is a Tay-Sachs patient. Another rare genetic disorder, Tay-Sachs is caused by the absence of an enzyme that helps break down fatty substances known as gangliosides. Without the ability to break them down, gangliosides build up to toxic levels in the brain and spinal cord, leading to early death.

After dosing with TSHA-101, the Tay-Sachs patient achieved Hex A enzyme activity of 25% of normal at month 1. That data represents a 5-fold improvement above the presumed asymptomatic level of 5% of normal identified by natural history.

Despite the early death that is possibly related to COVID-19, Taysha said the data for TSHA-101 is the first-ever to support the bicistronic vector approach in humans delivering bothHEXAandHEXBgenes in the endogenous ratio. Although the cause of the patients death is still under review, the company said preliminary data suggest that TSHA-101 was well-tolerated with no significant drug-related events.

TSHA-101 is the only bicistronic vector currently in clinical development for GM2 gangliosidosis and has been granted Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration. It has also been granted Orphan Drug designation from the European Commission.

TSHA-101 is the first bicistronic vector in clinical development, representing an important first for the field of gene therapy, RA Session II, president, founder and chief executive officer of Taysha, said in a statement. TSHA-101 demonstrated expression of bothHEXAandHEXBgenes in the endogenous ratio, providing the ability to restore and normalize enzyme activity in GM2 gangliosidosis. We expect to provide continued updates on the program, with additional clinical data anticipated by the end of 2022.

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Taysha's Promising Gene Therapy Results Marred by Patient's Death - BioSpace