Giant axonal neuropathy

Taysha Gene Therapies Provides Update on Deprioritized Pipeline Programs

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星期四, 二月 15, 2024
NINDS, Central nervous system, Rush University Medical Center, Giant axonal neuropathy, GM2, Rett syndrome, CNS, IP, GAN, IND, AAV, DSM-IV codes, Pharmaceutical industry

DALLAS, Feb. 15, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (“Taysha” or “the Company”), a clinical-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), today provided an update on its deprioritized pipeline programs as part of an ongoing effort to help support their further potential development.

Key Points: 
  • DALLAS, Feb. 15, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (“Taysha” or “the Company”), a clinical-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), today provided an update on its deprioritized pipeline programs as part of an ongoing effort to help support their further potential development.
  • Taysha has been working to find ways to advance its deprioritized programs.
  • On November 13, 2023, Taysha terminated its existing loan and security agreement and entered into a new loan and security agreement that provides consent to allow the Company to transfer intellectual property (IP) for several deprioritized programs to third parties in a more efficient manner.
  • “Today’s announcement demonstrates meaningful progress to advance important development work for several deprioritized programs.

Taysha Gene Therapies Provides Update on TSHA-120 Program in Giant Axonal Neuropathy (GAN)

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星期二, 九月 19, 2023
Exercise, Regulation, Partnership, Central nervous system, Natural history, Senior, Disease, Option, Rett syndrome, Food and Drug Administration, Neurodevelopmental disorder, South Tyrol Option Agreement, Type, AAV, CNS, Bangladesh Technical Education Board, Giant axonal neuropathy, GAN, DPM, HIV disease progression rates, Patient, FDA, Therapy, Bias, BLA, NIH, Food, Pharmaceutical industry, DALLAS, Astellas Pharma

DALLAS, Sept. 19, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-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), today announced that subsequent to the receipt of Type C meeting feedback from the United States (U.S.) Food and Drug Administration (FDA) regarding a registrational path for TSHA-120, the Company will discontinue the development of its TSHA-120 program in evaluation for the treatment of giant axonal neuropathy (GAN). Further, Taysha announced that Astellas Gene Therapies, Inc. (f/k/a Audentes Therapeutics, Inc. (d/b/a Astellas Gene Therapy)) (Astellas) has elected not to exercise its option to obtain an exclusive license to TSHA-120 under the Option Agreement between Astellas and Taysha.

Key Points: 
  • Further, Taysha announced that Astellas Gene Therapies, Inc. (f/k/a Audentes Therapeutics, Inc. (d/b/a Astellas Gene Therapy)) (Astellas) has elected not to exercise its option to obtain an exclusive license to TSHA-120 under the Option Agreement between Astellas and Taysha.
  • “We believe we have made significant progress in demonstrating the therapeutic potential of TSHA-120 and identifying a potential registrational path.
  • FDA feedback included the need to address the heterogeneity of disease progression in GAN and the effort-dependent nature of MFM32 as a primary endpoint in an unblinded study.
  • FDA Type C meeting feedback indicated that the FDA continues to recommend a randomized, double-blind, placebo-controlled trial as the optimal path to demonstrate efficacy in TSHA-120.

GordonMD® Invests in Private Placement for Taysha Gene Therapies

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星期一, 八月 21, 2023
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, NOT, Central nervous system, Civil service commission, Investment, RESPECT, Rett syndrome, MECP2, AAV, CNS, Giant axonal neuropathy, Orphan drug, GAN, MD, Risk, ANY, Rare, FDA, Notre Dame Institute for Global Investing, European Commission, Pharmaceutical industry

GordonMD® Global Investments LP announced today it has participated in a $150 million private placement financing for Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-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).

Key Points: 
  • GordonMD® Global Investments LP announced today it has participated in a $150 million private placement financing for Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-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).
  • Rett syndrome is a rare genetic neurodevelopmental disorder caused by mutations in the X-linked MECP2 gene.
  • TSHA-102 utilizes a novel miRARE platform designed to mediate levels of MECP2 in the CNS on a cell-by-cell basis without risk of overexpression.
  • The firm manages a private fund and a public fund, each focused on differentiated investment opportunities in biopharmaceutical companies primarily located in the U.S., Europe and Japan.

Taysha Gene Therapies Reports First Quarter 2023 Financial Results and Provides Corporate Update

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星期四, 五月 11, 2023
Central nervous system, Civil service commission, CMC, Annual Meetings of the International Monetary Fund and the World Bank Group, MECP2, Rett syndrome, Food and Drug Administration, CTA, Research, AAV, ID, CNS, Giant axonal neuropathy, U.S. FDA, MHRA, Development, Acute motor axonal neuropathy, State Administrative Expenses, Orphan drug, Investigational New Drug, GAN, Patient, Administration, Webcast, FDA, Safety, IND, Food, European Commission, Pharmaceutical industry, Financial adviser, Medical device, DALLAS

“Screening is completed, and dosing is now scheduled for our first potential patient in the adult Rett syndrome study.

Key Points: 
  • “Screening is completed, and dosing is now scheduled for our first potential patient in the adult Rett syndrome study.
  • Cash and cash equivalents: As of March 31, 2023, Taysha had $63.4 million in cash and cash equivalents.
  • Taysha continues to expect that its current cash resources will support planned operating expenses and capital requirements into the first quarter of 2024.
  • Taysha management will hold a conference call and webcast today at 4:30 pm ET to review its financial and operating results and to provide a corporate update.

Taysha Gene Therapies Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Corporate Update

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星期二, 三月 28, 2023
Central nervous system, Civil service commission, Clinical trial, MECP2, Rett syndrome, Food and Drug Administration, CTA, Research, AAV, ID, CNS, Giant axonal neuropathy, U.S. FDA, Development, State Administrative Expenses, Orphan drug, Investigational New Drug, GAN, Patient, Administration, Agency, Webcast, FDA, Safety, IND, Food, European Commission, MHRA, Pharmaceutical industry, Cryptocurrency, DALLAS

DALLAS, March 28, 2023 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-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), today reported financial results for the fourth quarter and full-year ended December 31, 2022, and provided a corporate update.

Key Points: 
  • TSHA-102 utilizes a novel miRNA-Responsive Auto-Regulatory Element (miRARE) platform designed to regulate cellular MECP2 expression.
  • In November 2022, we recorded a $36.4 million non-cash, non-recurring impairment charge related to the North Carolina manufacturing facility.
  • Cash and cash equivalents: As of December 31, 2022, Taysha had $87.9 million in cash and cash equivalents.
  • Taysha management will hold a conference call and webcast today at 4:30 pm ET to review its financial and operating results and to provide a corporate update.

Taysha Gene Therapies Provides Update on TSHA-120 Program in Giant Axonal Neuropathy and a 2023 Corporate Outlook

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星期二, 一月 31, 2023
Organization, B, Giant axonal neuropathy, ID, Food and Drug Administration, Safety, Rett syndrome, CNS, IND, AAV, CTA, CMC, Webcast, Central nervous system, BLA, Patient, GAN, FDA, MHRA, Pharmaceutical industry, Chemistry, DALLAS

Their feedback will help inform next steps for the program in this ultra-rare indication with no approved treatments,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha.

Key Points: 
  • Their feedback will help inform next steps for the program in this ultra-rare indication with no approved treatments,” said Sean P. Nolan, Chairman and Chief Executive Officer of Taysha.
  • The dial-in number for the conference call is 877-407-0792 (U.S./Canada) or 201-689-8263 (international).
  • The live webcast and replay may be accessed by visiting Taysha’s website at https://ir.tayshagtx.com/news-events/events-presentations .
  • An archived version of the webcast will be available on the website for 30 days.

Taysha Gene Therapies Reports Third Quarter 2022 Financial Results and Provides Corporate Update

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星期二, 十一月 8, 2022
Quality of life, GLOBE, Private Securities Litigation Reform Act, Forward-looking statement, Adult, GLP, Giant axonal neuropathy, Research and development, FDA, Partnership, Nasdaq, Webcast, Research, Rett syndrome, Technology, GAN, R, SEC, Compte rendu, Annual report, ID, U.S. Securities and Exchange Commission, AAV, Company, Drug development, Disease, Female, B, Patient, GMP, CEO, Administration, Central nervous system, CNS, COVID-19, Medical device, Pharmaceutical industry, Astellas Pharma, DALLAS

In 2023, we expect to provide an update on the regulatory pathway for TSHA-120 in GAN in January following our Type B end-of-Phase 2 meeting with the FDA.

Key Points: 
  • In 2023, we expect to provide an update on the regulatory pathway for TSHA-120 in GAN in January following our Type B end-of-Phase 2 meeting with the FDA.
  • Cash and cash equivalents: As of September 30, 2022, the Company had cash and cash equivalents of $34.3 million.
  • Taysha management will hold a conference call and webcast with slides today at8:00 am ET/7:00 am CTto review its financial and operating results and to provide a corporate update.
  • Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease.

AskBio Co-Founder Jude Samulski Receives Inaugural European Society of Gene and Cell Therapy Founders Award

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星期五, 十月 28, 2022
Cell, PARK, Therapy, Recombinant, University, Kaay Raav Tumhi, Clinical trial, Bayer, LinkedIn, Society, Congress, Goal, Disagreement, CEO, Canavan disease, Patent, Founder's Award, United States Patent and Trademark Office, Research Triangle Park, European Society of Gene and Cell Therapy, Haemophilia, Giant axonal neuropathy, Feedback, RAC, Patient, PBR322, Technology, Glycogen storage disease type II, Multimedia, COVID-19, Research, Capsid, ITR, Heart, FDA, Duchenne muscular dystrophy, RJ, ESGCT, Founder, AAV, Gene, Parkinson's disease, RESEARCH, Entrepreneurship, Science, Central nervous system, Pharmaceutical industry, Vaccine, Video game, Vatican

RESEARCH TRIANGLE PARK, N.C., Oct. 28, 2022 /PRNewswire/ -- Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, today announced that R. Jude Samulski, Ph.D., AskBio President, Co-Founder and Chief Scientific Officer, received the inaugural European Society of Gene and Cell Therapy (ESGCT) Founders Award in Edinburgh, Scotland, during the recent ESGCT 29th Congress.

Key Points: 
  • "ESGCT created the Founders Award this year to mark the Society's 30th anniversary," said Professor Hildegard Bning, ESGCT Board President.
  • "The award highlights benchmark milestones in gene and cell therapy.
  • The Founders Award 2022 recognizes the first cloning of an AAV plasmid, which Dr. Samulski and his team accomplished in 1982.
  • In 2008, Dr. Samulski was recognized by the American Society of Gene & Cell Therapy (ASGCT) with the inaugural Outstanding Achievement Award.

Taysha Gene Therapies to Host Conference Call to Discuss Astellas Pharma’s Strategic Investment to Support the Development of Taysha’s AAV-based Gene Therapy Programs

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星期二, 十月 25, 2022
CNS, GLOBE, AAV, Webcast, Rett syndrome, Giant axonal neuropathy, GAN, Central nervous system, Nasdaq, Pharmaceutical industry

DALLAS, Oct. 24, 2022 (GLOBE NEWSWIRE) -- 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 that it will host a conference call and webcast with slides to discuss Astellas Pharmas strategic investment to support the development of two of Tayshas AAV-based gene therapy programs, TSHA-102 for Rett syndrome and TSHA-120 for giant axonal neuropathy (GAN), on Tuesday, October 25, 2022, at 8:00 AM Eastern Time.

Key Points: 
  • DALLAS, Oct. 24, 2022 (GLOBE NEWSWIRE) -- 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 that it will host a conference call and webcast with slides to discuss Astellas Pharmas strategic investment to support the development of two of Tayshas AAV-based gene therapy programs, TSHA-102 for Rett syndrome and TSHA-120 for giant axonal neuropathy (GAN), on Tuesday, October 25, 2022, at 8:00 AM Eastern Time.

Taysha Gene Therapies Reports Second Quarter 2022 Financial Results and Provides Corporate Update

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星期四, 八月 11, 2022
FDA, U.S. Securities and Exchange Commission, Compte rendu, Survival, GLP, CEO, CNS, Accounting, GLOBE, R, Forward-looking statement, Private Securities Litigation Reform Act, SEC, Clinical trial, Visual acuity, Behavior, GAN, Research, Annual report, Research and development, Life, Mirare, Patient, Quality of life, COVID-19, Company, Giant axonal neuropathy, SNAP, Central nervous system, Natural history, Drug development, Â, MHRA, Phenotype, Webcast, Acute motor axonal neuropathy, Nasdaq, Royal commission, Rett syndrome, ID, European Commission, Administration, Adult, AAV, Disease, Medical imaging, Medical device, Pharmaceutical industry, DALLAS

We look forward to reporting preliminary Phase 1/2 clinical data in adult females with Rett syndrome by year-end 2022.

Key Points: 
  • We look forward to reporting preliminary Phase 1/2 clinical data in adult females with Rett syndrome by year-end 2022.
  • TSHA-120 for giant axonal neuropathy (GAN): an intrathecally dosed AAV9 gene therapy in clinical development for the rare inherited genetic disorder GAN.
  • Taysha management will hold a conference call and webcast with slides today at8:00 am ET/7:00 am CTto review its financial and operating results and to provide a corporate update.
  • Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease.