Category Archives: Genetic Therapy

As the world continues to battle the coronavirus pandemic, scientists are looking towards gene therapy to find ways to develop vaccines for the Covid-19 virus. Gene therapy itself was developed based on how viruses work.

When a virus attacks a host, it introduces its genetic material into the host cell as part of its replication cycle. The genetic material serves as an instruction manual on how to produce more copies of the virus, hijacking the host body's normal production machinery to serve the needs of the virus. The host cells then produce additional copies of the virus, leading to more host cells being infected.

Like animals, humans have found a way to domesticate viruses as well, i.e., direct the virus's function to achieve favorable results, which is prominent in gene therapy. Such viruses which physically insert their genes into the host's genome could instead be used to carry "good" genes into a human cell. Scientists would first remove the genes in the virus that cause diseases, and replace those genes with genes encoding the desired effect.

All of this sounds quite sci-fi but it has been done numerous times in the past. Peter Kolchinsky, a virologist and a biotechnology investor, compiled how different viruses have been used for gene therapy in the past.

Kolchinsky tweeted, "SARS2 is a scary menace, but did you know that we've domesticated viruses? Like wolves vs dogs, we've tamed them, including some deadly ones, to perform many useful functions (and may help us stop SARS2)."

The human immunodeficiency virus (HIV) has killed millions of people. It works by disabling the host body's immune system until it can't defend the person against common, normally mild pathogens. Kolchinsky explained that HIV's special trick is to integrate its genome into that of the host body's cells.

This feature of HIV is used for gene therapy, as explained before, by replacing a chunk of the virus's genome with the hemoglobin gene to insert it into bone marrow stem cells of patients with sickle cell anemia, whose hemoglobin genes are malfunctioning.

Kolchinsky also tweeted, "Adenoviruses typically cause mild infections, including common colds. These, too, we are trying to use for gene therapies, particularly when we just want to temporarily make a protein in cells. One company is developing such an adenovirus gene therapy for heart disease to induce growth of new blood vessels when old ones are clogged. Another is using this virus to make oral vaccines that would otherwise require injection (eg flu vaccine pill). When we use a virus to deliver code for making something in cells, we call that a virus vector."

There is now a wealth of clinical experience with numerous vector types that include primarily vaccinia, measles, vesicular stomatitis virus (VSV), polio, reovirus, adenovirus, lentivirus, -retrovirus, adeno-associated virus (AAV) and herpes simplex virus (HSV).

However, as with all other procedures, viral vector-gene therapy has associated risks. Viruses can usually infect more than one type of cell, so, when viralvectorsare used to carrygenesinto the body, they might infect healthy cells as well as cancer cells.

Another danger is that the new gene might be inserted in the wrong location in the DNA, possibly causing harmful mutations to the DNA or even cancer. Moreover, when viruses are used to deliver DNA to cells inside the patient's body, there is a slight chance that this DNA could unintentionally be introduced into the patients reproductive cells. If this happens, it could produce changes that may be passed on if a patient has children after treatment.

One study to help find a vaccine for Covid-19 aims to use the principles behind gene therapy to get the vaccine ready. The researchers' method uses a harmless virus as a vector to bring DNA into the patient's cells. The DNA should then instruct the cells to make a coronavirus protein that would stimulate the immune system to fight off future infections.

While a mass-produced vaccine may still take a while, this study is one of at least 90 vaccine projects around the world trying to find a cure for Covid-19. However, some experts are worried that a vaccine may never be available. According to our previous report, Dr David Nabarro, a professor of global health at Imperial College London, who also serves as a special envoy to the WHO on Covid-19, said, "There are some viruses that we still do not have vaccines against."

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Can gene therapy help develop coronavirus vaccine? Researchers banking on this technology for breakthrough - MEAWW

GAINESVILLE, Fla., and CAMBRIDGE, Mass., May 06, 2020 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it will report financial results for the fiscal quarter ended March 31, 2020 before the market opens on Wednesday, May 13, 2020. AGTC management will host a conference call beginning at 8:00 AM Eastern Time that day to review results and provide a corporate update.

To access the call, dial 877-407-6184 (US) or 201-389-0877 (outside of the US). A live webcast will be available in the Events and Presentations section of the Investor Relations page at http://ir.agtc.com/events-and-presentations. Please log in approximately 10 minutes prior to the scheduled start time. The archived webcast will be available in the Events and Presentations section of the company's website.

About AGTC

AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trial programs in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3) as well as an optogenetics program that is being developed in collaboration with Bionic Sight. In addition to its clinical trials, AGTC has preclinical programs in otology and adrenoleukodystrophy (ALD), frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), which are diseases of the central nervous system (CNS), and other ophthalmology indications including Stargardts and dry age-related macular degeneration (dry AMD). The otology program is being developed in collaboration with Otonomy. AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products.

IR/PR CONTACTS:David Carey(IR) orGlenn Silver (PR)Lazar FINN PartnersT: (212) 867-1768 or (646) 871-8485david.carey@finnpartners.comorglenn.silver@finnpartners.com

Corporate Contacts:Bill SullivanChief Financial OfficerApplied Genetic Technologies CorporationT: (617) 843-5728bsullivan@agtc.com

Stephen PotterChief Business OfficerApplied Genetic Technologies CorporationT: (617) 413-2754spotter@agtc.com

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AGTC to Host Third Quarter Financial Results Conference Call and Webcast on May 13, 2020 - Yahoo Finance

CAMBRIDGE, Mass.--(BUSINESS WIRE)--AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, today announced a new development and commercialization agreement with Saladax Biomedical, Inc. (Saladax), a leading diagnostics provider focused on developing blood tests for personalized dosing, to develop and validate a fully automated nanoparticle immunoassay kit designed to simplify and streamline therapeutic drug monitoring (TDM) for patients treated with the conditioning agent busulfan.

At AVROBIO, we push ourselves to be at the forefront of technologies advancing lentiviral gene therapy, and its in this spirit that were funding the development of this kit, said Geoff MacKay, AVROBIOs president and CEO. Our personalized conditioning approach is already delivering results. We believe this new assay kit will, for the first time, provide convenient busulfan TDM close to the patient, potentially improving both the patient experience and long-term outcomes, as well as enabling many more hospitals and clinics to become TDM-capable sites.

AVROBIOs state-of-the-art plato gene therapy platform incorporates TDM protocols designed to optimize busulfan dosing over four days, with the goal of maximizing stem cell engraftment while minimizing side effects. TDM evaluates how quickly a patient metabolizes busulfan a rate that can vary significantly from patient to patient and even from one day to the next for the same patient. Current assays that inform that dose adjustment can take hours to return results and must be processed at specialized laboratories with trained staff that may not be geographically convenient to the gene therapy dosing site.

The technology used to deliver these rapid test results is based on an extensive intellectual property portfolio developed by Saladax in the field of TDM. The new assay kit under development by Saladax, which collects a small blood sample, is able to return results on patient metabolization of busulfan in minutes using hospitals standard analytical devices, greatly expanding access to personalized conditioning with busulfan.

Personalized Gene Therapy to Optimize Durable Protein Expression including in Brain, Muscle and Bone

AVROBIOs investigational gene therapies start with collecting the patients own hematopoietic stem cells. In the companys manufacturing process, a lentiviral vector is used to integrate a therapeutic gene designed to produce functional protein essential to cellular health into the patients chromosomes. Prior to dosing, treating clinicians use busulfan, an extensively validated conditioning agent generally considered to be the gold standard for ex vivo lentiviral gene therapy, to create space in the patients bone marrow. Finally, the patient receives the gene therapy and the therapeutic stem cells are expected to engraft in the bone marrow and produce generations of daughter cells, each containing the therapeutic gene. This approach is designed to drive durable production of the functional protein throughout the patients body, including hard-to-reach tissues such as the brain, muscle and bone. A distinguishing feature of this type of gene therapy with busulfan conditioning is that some of the corrected cells are expected to cross the blood-brain barrier and thereby potentially address central nervous system manifestations.

Earlier this year, AVROBIO reported initial clinical results for the first patient conditioned with busulfan using TDM prior to dosing in AVROBIOs Phase 2 clinical trial of its investigational gene therapy, AVR-RD-01, for Fabry disease. The early data from this patient showed increased endogenous enzyme activity at one month following dosing, as compared to other patients in the trial who received a different conditioning agent. Initial data suggest side effects, including nausea, mucositis, fever, rash and hair loss, which were consistent with those expected based on clinical experience of busulfan, developed eight to 10 days after dosing with busulfan and resolved quickly.

About AVROBIO

Our mission is to free people from a lifetime of genetic disease with a single dose of gene therapy. We aim to halt or reverse disease throughout the body by driving durable expression of functional protein, even in hard-to-reach tissues and organs including the brain, muscle and bone. Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a program in Pompe disease. AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words and phrases such as aims, anticipates, believes, could, designed to, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will and variations of these words and phrases or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding our business strategy for and the potential therapeutic benefits of our prospective product candidates, anticipated benefits of our gene therapy platform including potential impact on our commercialization activities, timing and likelihood of success, the expected benefits of Saladaxs immunoassay kits, including the ability to improve, simplify and streamline therapeutics drug monitoring for patients treated with the conditioning agent busulfan and enable local commercialization of AVROBIOs proprietary platform worldwide, the expected benefits and results of our implementation of the plato platform in our clinical trials and gene therapy programs, and the expected safety profile of our investigational gene therapies. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not successfully recruit or enroll a sufficient number of patients for our clinical trials, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that AVROBIO may not realize the intended benefit of Saladaxs immunoassay kits, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, risks relating to clinical trial and business interruptions resulting from the COVID-19 outbreak or similar public health crises, including that such interruptions may materially delay our development timeline and/or increase our development costs or that data collection efforts may be impaired or otherwise impacted by such crises, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Annual or Quarterly Report, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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AVROBIO to Collaborate with Saladax Biomedical on New High-Speed Diagnostic Assay Used with Busulfan Conditioning to Enable Widespread...

A detailed study by Fact.MR on the Gene Therapy market provides an in-depth evaluation of various growth drivers, trends, and opportunities, the prevailing regulatory landscape in various regions, and the competitive scenario amidst the COVID-19 pandemic. Despite the ongoing issues, the worldwide revenue of gene therapy market will surpass US$ 5 Bn by 2026, exhibiting a stellar growth rate. At-scale investments in the field of gene-related R&D, and increasing number of late-stage gene therapy candidates in oncology and other genetic disorders are likely to oil the growth engine. The report provides insight into the major drivers and key restraining factors, emerging trends, and sources of new revenue streams over the assessment period.

To make a granular assessment of the current and projected opportunities and revenues, the report segments the Gene Therapy market on the basis of region, end-user/application, and product/service/offering type.

Request to View Sample of Research Report @ https://www.factmr.com/connectus/sample?flag=S&rep_id=4648

Regional Assessments:

The study identifies key opportunities in different key regions. It provides insights into key trends and favorable macroeconomic frameworks in prominent revenue-generating regions. Some of the key regions and countries covered in the analysis are:

Product-wise Assessments:

The report segments the Gene Therapy market based on major offerings by various players. It provides a detailed scenario of the existing demand and consumption patterns of various key product types. It also provides readers with the prevailing pricing structure in various regions, emerging manufacturing/production trends, and recent technological advancements in products/services.

The key segments covered are:

The study takes a closer look at the nature of demand in various key application areas and highlights major technological innovations in particular to certain areas. It also takes a closer look at

The study on the Gene Therapy market provides a detailed profile of offerings by key players, their key strategies, recent mergers and acquisitions, and major collaboration deals. Companies covered in the study include

To Know More Information about This Report, Ask The Analyst @ https://www.factmr.com/connectus/sample?flag=AE&rep_id=4648

How Insights and Analysis by Fact.MR Make Difference?

The in-depth assessment of various growth dynamics of the Gene Therapy market and insights on market estimations help all market participants to stay ahead in the curve of growth with a worldwide economic impact of COVID-19. Some of the insights that can make the difference are:

Apart from these insights, the authors of the report by Fact.MR shed light on the following dynamics and answers relevant questions:

About Fact.MR

Fact.MR is a fast-growing market research firm that offers the most comprehensive suite of syndicated and customized market research reports. We believe transformative intelligence can educate and inspire businesses to make smarter decisions. We know the limitations of the one-size-fits-all approach; thats why we publish multi-industry global, regional, and country-specific research reports.

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Gene Therapy Market Size, Share, Growing Demand and Rising Trends 2020 to 2026 - The Cloud Tribune

Vir and Alnylam Go Ahead with COVID-19 Gene Silencing Therapy

Vir Biotechnology (VIR) announced a new COVID-19 drug candidate, VIR-2703. The company is collaborating with Alnylam Pharmaceuticals (ALNY) for developing this treatment which is an inhalable RNAi therapeutic which works by targeting the SARS-CoV-2 genome for potentially treating and/or preventing the respiratory illness. Both companies are now planning to meet with the FDA for a probable accelerate pathway for progressing to clinical trials stage.

The collaboration also seeks to identify up to three additional drug candidates for treating COVID-19 as well as other coronavirus ailments. Vir recently received infusion of $250 million from GlaxoSmithKline Plc (NYSE:GSK). In March this year, Alnylam synthesized 350 siRNAs targeting the SARS-CoV-2 genome. Alnylam Pharmaceuticals CEO John Maraganore said, "To our knowledge, this is one of the most potent direct-acting antivirals targeting SARS-CoV-2 reported to date. As this pandemic continues to unfold, we are committed to acting with the utmost urgency to broaden and accelerate our efforts to develop investigational RNAi therapeutics against COVID-19, and potentially future coronavirus-mediated diseases." The companies are working closely to generate data for facilitating fast commercialization.

VIR-2703 takes a novel approach for treating coronavirus. It employs RNA interference or RNAi technology for silencing particular genetic material leading to the impact on production of proteins responsible for the disease. Alnylam currently has Givlaari and Onpattro, the FDA approved drugs in its portfolio and these drugs are intended to treat rare genetic diseases. The companies plan to file an Investigational New Drug (IND) or IND equivalent application for the drug candidate by the end of this year. The investigational RNAi therapeutic is being developed as an inhalation formula and would be delivered directly to the patient's lungs.

Alnylam is believed to have synthesized more than 350 small interfering RNAs or siRNAs as the company said that it is focusing on the highly conserved regions of the SARS-CoV-2 genome. VIR-2703 showed to have an effective concentration for 50% inhibition (EC50) of less than 100 picomolar in dose-response assays. It also demonstrated an EC95 of less than one nanomolar in the SARS-CoV-2 live virus model which was used for measuring the inhibition of infectious virion production.

VIR-2703 is also known as ALN-COV and showed 95 percent of its maximal effect at a concentration of one nanomolar in its dose response assays. The drug candidate has been called "one of the most potent direct-acting antivirals targeting SARS-CoV-2 reported to date" by Alnylam CEO John Maraganore. The companies are planning to develop up to three more host factor targeting development candidates for treating COVID-19.

Vir mainly focuses on developing treatments for infectious diseases while Alnylam specializes in developing RNAi treatments. Both the companies are also working on another product candidate VIR-2218, which seeks to treat hepatitis B virus. This drug candidate is an RNAi product and works by inhibiting the production of proteins such as hepatitis B virus surface antigen (HBsAg). VIR-2218 is currently in Phase 2 of development.

BioMarin Pharmaceutical Inc. (BMRN) announced that it has collaborated with a Swiss biotech firm DiNAQOR AG. The deal is mainly concerned with the development of novel gene therapies for treating rare genetic cardiomyopathies. The license initially encompasses DiNAQOR's lead program, DiNA-001 for MYBPC3 hypertrophic cardiomyopathy. However, later the collaboration will expand to cover various pipeline programs of the Swiss company.

The companies did not disclose the financial terms of the deal but DiNAQOR will be entitled to an undisclosed sum as upfront payment and may also be eligible for biobucks. BioMarin will have the option to expand the license to cover additional programs of DiNAQOR on similar terms. Jean-Jacques Bienaim, chairman and CEO at BioMarin said, " With this agreement, BioMarin is continuing to apply its gene therapy know-how and manufacturing expertise in new areas like cardiology. This collaboration extends our global leadership position in gene therapy and boosts our potential to transform the lives of patients worldwide with rare genetic cardiomyopathies." BioMarin has its own portfolio of gene therapy work.

It is estimated that nearly half a million people around the world have been diagnosed with HCM. Under the terms and conditions of the deal, DiNAQOR will also receive various other payments tied to the achievement of different development regulatory and commercial milestones in addition to tiered royalties. BioMarin expects the collaboration to help boost its position in the market and provide it global reach.

BioMarin also reiterated its 2020 GAAP net income estimates. The company expects its GAAP net income for the entire year to be in the range of $20 million and $80 million, taking the collaboration into account. DiNAQOR mainly focuses on developing gene therapies which can safely offer treatment to the heart muscle and carry out transduction of the cardiac cells while limiting the exposure of the therapy to other organs.

Teva Pharmaceutical Industries (TEVA) announced the commercial availability of its Truxima in the United States. The company has collaborated with Celltrion Healthcare for this purpose. Truxima is the biosimilar to Roche (OTCQX:RHHBY) blockbuster Rituxan for treating rheumatoid arthritis.

The drug is approved to be used for treating rheumatoid arthritis in combination with methotrexate in adult patients suffering from moderately to severely active RA and who have not shown adequate response to one or more TNF antagonist therapies. Brendan O'Grady, Executive Vice President, North America Commercial of Teva said, "We are proud to make TRUXIMA available to patients and providers as a treatment option for these indications, especially as this is the only rituximab biosimilar indicated for rheumatoid arthritis. Following the launch of our other biosimilar earlier this year, we remain focused on our commitment to lower healthcare costs and increase price competition through the availability of biosimilars." Truxima was awarded a pass-through status in the hospital outpatient setting earlier this year. The status was granted by the Centers for Medicare and Medicaid Services.

Under its October 2016 agreement, Teva owns commercialization rights for the drug in the United States and Canada. Celltrion vice chairman Hyoung-Ki Kim said, "We believe that the continued use of biosimilars in the US market will contribute to addressing unmet needs for patients and providers."

It is expected that the drug will be available through primary wholesalers at a Wholesale Acquisition Cost of $845.55 per 100mg vial and $4,227.75 per 500mg vial. However, actual costs to individual patients and providers are likely to be lower as WAC does not take additional rebates and discounts into account. Teva will also provide dedicated patient support services through the CORE program.

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Vir-Alnylam's COVID-19 Therapy, And Other News: The Good, Bad And Ugly Of Biopharma - Seeking Alpha

The team that developed Zolgensma is back for round two. A group of former AveXis executives and investors unveiled a new gene therapy company, and theyre wasting no time. With 15 programs, $30 million in seed funding and an unrivaled partnership with UT Southwestern Medical Center, Taysha Gene Therapies plans to be in the clinic by the end of the year.

Its working on adeno-associated vector (AAV) gene therapies for monogenic diseasesthat is, diseases caused by a defect in a single geneof the central nervous system. Tayshas lead program targets GM2 gangliosidosis, a very rare disorder that progressively destroys nerve cells in the brain and spinal cord, but the companys portfolio includes conditions that are more prevalent.

The company plans to start clinical trials for three more programs by the end of 2021: treatments for Rett syndrome, a neurodevelopmental disorder; SURF1 deficiency, the most frequent cause of Leigh syndrome; and a SLC6A1 genetic epilepsy, which is similar to Dravet syndrome, Taysha CEO and co-founder R. A. Session II told FierceBiotech.

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And thats not allTaysha has the option to pick up four more prospects from UT Southwestern.

The reason why we can tackle 15 programs with the option to four additional programs is were able to focus on what we do best and were able to allow our collaborators to focus on what they do best, Session said.

Under their partnership, UT Southwestern is working on discovery and preclinical work all the way through IND-enabling studies. It has its own GMP viral manufacturing site to support that work, as well as clinical development, which Taysha will pick up. The company will also take care of regulatory strategy, commercial manufacturing and commercialization. Its a skill set that Tayshas management team honed at AveXis as itdeveloped the gene therapy that eventually became Novartis spinal muscular atrophy treatment Zolgensma.

We essentially flew the plane and built it at the same time when we were developing AveXis We have people with the experience of being able to develop, manufacture and commercialize a gene therapy program and were marrying that with a best-in-class academic research institution, Session said.

RELATED: FDA lets Novartis off the hook in Zolgensma data manipulation

Dividing the labor creates this engine for innovation that allows the partners to advance many programs in parallel, Session added. With about 50 people in its gene therapy unit, UT Southwestern can develop a capsidthe protein shell of a virusfor treatment delivery or get a candidate into animal models much more quickly than a biotech going it alone, he said. The same goes for late-stage development and commercialization on Tayshas side.

For its initial programs, Taysha is focusing on AAV gene therapies because the team knows they work.

AAV9 is the best way we have to treat monogenic CNS disease. If its not broke, dont fix it, Session said. We know how to effectively dose AAV9; we know its safe, effective and efficient. And we know its scalable. These are problems we had to solve at our previous company.

But, moving forward, Taysha and UT Southwestern are working on new technologies, including an AAV delivery platform that would allow for the redosing of gene therapies as well as an AAV capsid platform aimed at improving target delivery.

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Taysha Gene Therapies hits the ground running with $30M, 15 programs - FierceBiotech