StemCell Therapy

DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced new data for multiple preclinical programs and a planned R&D Day, which will be held in June 2021.

Collectively, these new preclinical data highlight Tayshas next wave of novel gene therapies that have the potential to impact meaningful patient populations. The promising data underscore our ability to rapidly and reproducibly investigate disease biology, design innovative gene therapies and efficiently advance the development of these drug candidates, said RA Session II, President, Founder and Chief Executive Officer of Taysha. Among the compelling new data, for the first time, we have shown that TSHA-113, an AAV9 gene therapy that utilizes AAV-mediated gene silencing, reduced tau expression in mouse models of human tauopathies. The potential implications of these data are far reaching, and we intend to further evaluate TSHA-113 in additional preclinical studies. The totality of the preclinical data presented today support the fundamental elements of our scientific approach of coupling validated technology with novel targeted payload design while utilizing a proven HEK293 suspension manufacturing process. We believe our deep pipeline and innovative scientific engine hold tremendous potential, and we are poised to continue delivering meaningful value to patients with monogenic CNS diseases.

Todays data demonstrate the breadth, depth and velocity of our development engine as a sustainable pivotal-stage gene therapy company. There are no approved disease modifying therapies for any of the programs in our portfolio and we are encouraged by the results of our gene therapy approach of vectorized RNA and gene replacement therapies across our portfolio, said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. We are very excited to further develop TSHA-113 in tauopathies, including Alzheimers disease, MAPT-associated frontotemporal dementia and progressive supranuclear palsy, based on the significant reduction in tau expression demonstrated in transgenic mouse models of human tauopathies. In addition, to date, we have advanced five programs into IND/CTA-enabling studies, including TSHA-105 in SLC13A5 deficiency, TSHA-111-LAFORIN in Lafora disease, TSHA-111-MALIN in Lafora disease, TSHA-112 in APBD and TSHA-119 in GM2 AB variant. We intend to file an IND/CTA for one of these five named programs by the end of 2021. By mid-year, we intend to select a development candidate for Angelman syndrome and obtain interim expression and safety data from confirmatory non-human primate studies by year-end. We remain on track to report Phase 1/2 biomarker data for TSHA-101 in GM2 gangliosidosis in the second half of this year and to provide a clinical and regulatory update for TSHA-120 in giant axonal neuropathy by year-end. Finally, in the second half of the year, we continue to expect dosing of the first patient with CLN1 disease in a Phase 1/2 trial for TSHA-118 under an already open IND, filing an IND/CTA for TSHA-102 in Rett syndrome and TSHA-104 in SURF1-associated Leigh syndrome, and filing an IND for TSHA-101 in GM2 gangliosidosis in the U.S. These anticipated clinical and regulatory milestones are expected to be followed by the initiation of Phase 1/2 clinical trials for each of these indications. We look forward to providing additional updates at our R&D Day in June.

TSHA-113 for Tauopathies

Taysha is developing tau-specific microRNA (miRNA) shuttles designed to target tau mRNA for all six isoforms found in the human brain and/or mouse brain. TSHA-113 is an AAV9 capsid that packages these miRNA shuttles and is delivered in the CSF for the treatment of tauopathies.

TSHA-105 for SLC13A5 deficiency

TSHA-105 is a recombinant self-complementary AAV9 vector that expresses the human SLC13A5 protein under the control of a ubiquitous promoter. The drug candidate is being developed for the treatment of SLC13A5 deficiency.

TSHA-106 for Angelman syndrome

TSHA-106 is an intrathecally delivered AAV9 viral vector designed for shRNA-mediated knockdown of UBE3A-ATS, the antisense transcript governing the expression of UBE3A through the paternal allele.

TSHA-112 for Adult Polyglucosan Body Disease (APBD)

TSHA-112 is an intrathecally delivered AAV9 viral vector designed for miRNA-mediated knockdown of the GYS1 gene to treat APBD.

TSHA-111-LAFORIN for EPM2A and TSHA-111-MALIN for EPM2B for Lafora disease

TSHA-111-LAFORIN and TSHA-111-MALIN are intrathecally delivered AAV9 viral vectors designed for miRNA-mediated knockdown of the GYS1 gene to treat Lafora disease.

TSHA-119 for GM2 AB variant

TSHA-119 is a self-complementary AAV9 vector designed to deliver a functional copy of the GM2A gene to treat GM2 AB variant.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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This entry was posted on April 17, 2021 at 1:52 am and is filed under Gene therapy. You can follow any responses to this entry through the RSS 2.0 feed. Both comments and pings are currently closed. |