Taysha Gene Therapies to Present Biodistribution Data from an Analysis Evaluating AAV9 Gene Therapy Delivery at the Upcoming 31st Annual ESGCT Congress
Taysha Gene Therapies will present biodistribution data from an analysis of 28 non-human primates (NHPs) at the 31st Annual ESGCT Congress in Rome. The analysis, spanning five studies, evaluated AAV9 gene therapy vector delivery using intrathecal (IT) and intra-cisterna magna (ICM) administration.
Key findings include:
- Lumbar IT administration led to widespread and consistent biodistribution across brain and spinal cord regions
- IT and ICM administration achieved comparable biodistribution in NHPs
- Results support IT administration as an effective, safe, and minimally invasive approach for delivering AAV-based gene therapies for CNS diseases in children and adults
The data reinforces Taysha's clinical development strategy, particularly for their TSHA-102 program targeting Rett syndrome. The poster presentation, titled 'Broad CNS Biodistribution of AAV9-based Gene Therapies Delivered by Intrathecal Lumbar Puncture in Non-Human Primates,' will be presented on October 23, 2024.
Taysha Gene Therapies presenterà dati sulla biodistribuzione da un'analisi di 28 primati non umani (NHP) al 31° Congresso Annuale ESGCT a Roma. L'analisi, che comprende cinque studi, ha valutato la somministrazione del vettore di terapia genica AAV9 utilizzando la somministrazione intratecale (IT) e intra-cisterna magna (ICM).
Le principali scoperte includono:
- La somministrazione lombare IT ha portato a una biodistribuzione ampia e consistente nelle aree cerebrali e del midollo spinale
- Le somministrazioni IT e ICM hanno raggiunto una biodistribuzione comparabile negli NHP
- I risultati supportano la somministrazione IT come un approccio efficace, sicuro e minimamente invasivo per la somministrazione di terapie geniche basate su AAV per le malattie del SNC nei bambini e negli adulti
I dati rafforzano la strategia di sviluppo clinico di Taysha, in particolare per il loro programma TSHA-102 che mira alla sindrome di Rett. La presentazione del poster, intitolata 'Ampia biodistribuzione del SNC delle terapie geniche basate su AAV9 somministrate mediante puntura lombare intratecale in primati non umani,' sarà presentata il 23 ottobre 2024.
Taysha Gene Therapies presentará datos de biodistribución de un análisis de 28 primates no humanos (NHP) en el 31° Congreso Anual ESGCT en Roma. El análisis, que abarca cinco estudios, evaluó la administración del vector de terapia génica AAV9 utilizando métodos intratecales (IT) e intra-cisterna magna (ICM).
Los hallazgos clave incluyen:
- La administración lumbar IT condujo a una biodistribución amplia y consistente en las regiones del cerebro y la médula espinal
- La administración IT e ICM logró una biodistribución comparable en NHP
- Los resultados respaldan la administración IT como un enfoque efectivo, seguro y mínimamente invasivo para entregar terapias génicas basadas en AAV para enfermedades del SNC en niños y adultos
Los datos refuerzan la estrategia de desarrollo clínico de Taysha, especialmente para su programa TSHA-102 dirigido al síndrome de Rett. La presentación del póster, titulada 'Amplia biodistribución del SNC de las terapias génicas basadas en AAV9 entregadas por punción lumbar intratecal en primates no humanos,' se presentará el 23 de octubre de 2024.
Taysha Gene Therapies는 로마에서 열리는 제31회 ESGCT 연례회의에서 28마리의 비인간 영장류(NHP)에 대한 생물 분포 데이터를 발표할 예정입니다. 이 분석은 5개의 연구를 포함하며 AAV9 유전자 요법 벡터 전달을 척수내(IT)와 대뇌수막내(ICM) 투여를 통해 평가했습니다.
주요 발견은 다음과 같습니다:
- 요추 IT 투여는 뇌와 척수 지역에서 광범위하고 일관된 생물 분포를 유도했습니다.
- IT 및 ICM 투여는 NHP에서 비교 가능한 생물 분포를 달성했습니다.
- 결과는 IT 투여가 아동과 성인의 CNS 질환에 대한 AAV 기반 유전자 치료를 위한 효과적이고 안전하며 최소 침습적인 접근 방식으로서의 타당성을 지지합니다.
이 데이터는 Taysha의 임상 개발 전략을 강화하며, 특히 Rett 증후군을 겨냥한 TSHA-102 프로그램에 관한 것입니다. 포스터 발표 제목은 '비인간 영장류에서 척수내 요추 천자를 통해 전달된 AAV9 기반 유전자 치료의 광범위한 CNS 생물 분포'이며, 2024년 10월 23일에 발표될 예정입니다.
Taysha Gene Therapies présentera des données de biodistribution provenant d'une analyse de 28 primates non humains (NHP) lors du 31e Congrès Annuel de l'ESGCT à Rome. L'analyse, qui s'étend sur cinq études, a évalué la livraison du vecteur de thérapie génique AAV9 par administration intrathécale (IT) et intra-cisterna magna (ICM).
Les principales conclusions incluent :
- L'administration lombaire IT a conduit à une biodistribution large et cohérente dans les régions cérébrales et de la moelle épinière
- L'administration IT et ICM a atteint une biodistribution comparable chez les NHP
- Les résultats soutiennent l'administration IT comme une approche efficace, sûre et peu invasive pour la délivrance de thérapies géniques basées sur AAV pour les maladies du SNC chez les enfants et les adultes
Les données renforcent la stratégie de développement clinique de Taysha, en particulier pour leur programme TSHA-102 ciblant le syndrome de Rett. La présentation du poster, intitulée 'Large biodistribution CNS des thérapies géniques basées sur AAV9 délivrées par ponction lombaire intrathécale chez les primates non humains,' sera présentée le 23 octobre 2024.
Taysha Gene Therapies wird Daten zur Biodistribution aus einer Analyse von 28 nicht-menschlichen Primaten (NHP) beim 31. jährlichen ESGCT-Kongress in Rom präsentieren. Die Analyse, die sich über fünf Studien erstreckt, bewertete die Verabreichung des AAV9-Gentherapievectors mittels intrathekaler (IT) und intra-cisterna magna (ICM) Anwendung.
Wichtige Ergebnisse sind:
- Die lumbale IT-Anwendung führte zu einer weitreichenden und konsistenten Biodistribution in den Gehirn- und Rückenmarksregionen
- IT- und ICM-Anwendungen erzielten eine vergleichbare Biodistribution bei NHPs
- Die Ergebnisse unterstützen die IT-Anwendung als einen effektiven, sicheren und minimalinvasiven Ansatz zur Verabreichung von AAV-basierten Gentherapien für ZNS-Erkrankungen bei Kindern und Erwachsenen
Die Daten stärken die klinische Entwicklungsstrategie von Taysha, insbesondere für ihr TSHA-102-Programm, das auf das Rett-Syndrom abzielt. Die Poster-Präsentation mit dem Titel 'Breite CNS-Biodistribution von AAV9-basierten Gentherapien, die durch intrathekale lumbale Punktion in nicht-menschlichen Primaten verabreicht wurden,' wird am 23. Oktober 2024 präsentiert.
- None.
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Data from an analysis of five NHP studies showed lumbar IT administration led to widespread and consistent biodistribution of AAV9 gene therapy vectors across brain and spinal cord regions
IT and ICM administration achieved comparable biodistribution across brain and spinal cord regions in NHPs
Findings from the analysis reaffirm the clinical potential of IT administration as an effective, safe and minimally invasive approach to deliver AAV-based gene therapies designed to treat CNS diseases in both children and adults
DALLAS, Oct. 22, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (Taysha or the Company), a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system (CNS), today announced that it will present biodistribution data from an analysis of 28 non-human primates (NHP) evaluating the delivery of AAV9 gene therapy vectors across five different studies using intrathecal (IT) delivery by lumbar puncture (four studies) or intra-cisterna magna (ICM) injection (one study). The data will be presented during a poster presentation at the upcoming 31st Annual Congress of the European Society of Gene & Cell Therapy (ESGCT), taking place in Rome, Italy from October 22-25, 2024. These findings reinforce Taysha’s clinical development approach utilizing IT administration to deliver AAV-based gene therapies designed to treat the genetic root cause of CNS diseases, including the TSHA-102 program in clinical evaluation for children, adolescents and adults living with Rett syndrome.
“Findings from an analysis across five NHP studies showed that both IT and ICM administration led to comparable, consistent and widespread biodistribution of AAV9 vector throughout the brain and spinal cord regions,” said Sukumar Nagendran, M.D., President and Head of Research & Development at Taysha. “Importantly, these findings further support the clinical potential of IT administration as an effective, safe and minimally invasive delivery approach for broad targeting of the CNS that has potential for outpatient use in both children and adults. We believe the NHP biodistribution data, together with our additional preclinical data, reaffirm our clinical development strategy utilizing IT administration and provide translational support for the broad clinical effect reported following treatment with TSHA-102 in both our REVEAL adolescent/adult trial and REVEAL pediatric trial."
Poster presentation details are as follows:
Title: Broad CNS Biodistribution of AAV9-based Gene Therapies Delivered by Intrathecal Lumbar Puncture in Non-Human Primates
Presenters: Nino Devidze, MS, Ph.D., Clinical Development Lead and Emdadul Haque, Ph.D., Senior Director, Translational Sciences at Taysha Gene Therapies
Date and Time: Wednesday October 23, 2024, from 13:30 to 15:00 CEST
Poster Session: CNS & Sensory Diseases
Poster Number: P0284
Additional details on the meeting can be found at the 31st Annual ESGCT Congress website.
About TSHA-102
TSHA-102 is a self-complementary intrathecally delivered AAV9 investigational gene transfer therapy in clinical evaluation for Rett syndrome. Designed as a one-time treatment, TSHA-102 aims to address the genetic root cause of the disease by delivering a functional form of MECP2 to cells in the CNS. TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) technology designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression. TSHA-102 has received Regenerative Medicine Advanced Therapy, Fast Track and Orphan Drug and Rare Pediatric Disease designations from the FDA, Orphan Drug designation from the European Commission and Innovative Licensing and Access Pathway designation from the Medicines and Healthcare products Regulatory Agency.
About Rett Syndrome
Rett syndrome is a rare neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene encoding methyl CpG-binding protein 2 (MeCP2), which is essential for regulating neuronal and synaptic function in the brain. The disorder is characterized by loss of communication and hand function, slowing and/or regression of development, motor and respiratory impairment, seizures, intellectual disabilities and shortened life expectancy. Rett syndrome progression is divided into four key stages, beginning with early onset stagnation at 6 to 18 months of age followed by rapid regression, plateau and late motor deterioration. Rett syndrome primarily occurs in females and is one of the most common genetic causes of severe intellectual disability. Currently, there are no approved disease-modifying therapies that treat the genetic root cause of the disease. Rett syndrome caused by a pathogenic/likely pathogenic MECP2 mutation is estimated to affect between 15,000 and 20,000 patients in the U.S., EU, and U.K.
About Taysha Gene Therapies
Taysha Gene Therapies (Nasdaq: TSHA) is a clinical-stage biotechnology company focused on advancing adeno-associated virus (AAV)-based gene therapies for severe monogenic diseases of the central nervous system. Its lead clinical program TSHA-102 is in development for Rett syndrome, a rare neurodevelopmental disorder with no approved disease-modifying therapies that address the genetic root cause of the disease. With a singular focus on developing transformative medicines, Taysha aims to address severe unmet medical needs and dramatically improve the lives of patients and their caregivers. The Company’s management team has proven experience in gene therapy development and commercialization. Taysha leverages this experience, its manufacturing process and a clinically and commercially proven AAV9 capsid in an effort to rapidly translate treatments from bench to bedside. For more information, please visit 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,” “plans,” and “future” or similar expressions are intended to identify forward-looking statements. Forward-looking statements include, but are not limited to, statements concerning the potential of TSHA-102 and Taysha’s other product candidates to positively impact quality of life and alter the course of disease in the patients Taysha seeks to treat, its research, development and regulatory plans for its 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 Taysha’s product candidates. Forward-looking statements are based on management’s 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 Taysha’s business are described in detail in its SEC filings, including in Taysha’s Annual Report on Form 10-K for the full-year ended December 31, 2023 and Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, which are available on the SEC’s website at www.sec.gov. Additional information will be made available in other filings that Taysha makes from time to time with the SEC. These forward-looking statements speak only as of the date hereof, and Taysha disclaims any obligation to update these statements except as may be required by law.
Company Contact:
Hayleigh Collins
Director, Head of Corporate Communications and Investor Relations
Taysha Gene Therapies, Inc.
hcollins@tayshagtx.com
Media Contact:
Carolyn Hawley
Inizio Evoke
Carolyn.hawley@inizioevoke.com
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