Articles from Taysha Gene Therapies, Inc.

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 reported financial results for the fourth quarter and full-year ended December 31, 2021 and provided a corporate update.

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 the initiation of clinical development of TSHA-102 for the treatment of Rett syndrome under a recently approved Clinical Trial Application (CTA) by Health Canada. Sainte-Justine Mother and Child University Hospital Center in Montreal, Quebec, Canada has been selected as the initial clinical site under the direction of Dr. Elsa Rossignol, principal investigator. The company also announced positive preclinical data from IND/CTA-enabling studies including a pharmacology study in the Rett knockout mouse model (n=252) that assessed the efficacy of TSHA-102 and a Good Laboratory Practices (GLP) toxicology study of TSHA-102 in nonhuman primates (NHPs) (n=24) that explored biodistribution and mechanism of action, both of which supported authorization of the CTA.

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 report its financial results for the fourth quarter and full-year ended December 31, 2021, and host a corporate update conference call and webcast on Thursday, March 31, 2022, at 8:00 AM Eastern Time.

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 its participation in upcoming fireside chats at the Guggenheim Genomic Medicines and Rare Disease Day Conference and the 21st Annual Needham Virtual Healthcare Conference.

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 the presentation of clinical safety data supporting the first-generation construct at the highest dose of 1.0x1015 total vg for the treatment of CLN7 Batten disease at the 18th Annual WORLD Symposium, held February 7-11, 2022, in San Diego, CA by investigators from UT Southwestern.

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 reported positive clinical efficacy and safety data for the high dose cohort of 3.5x1014 total vg, as well as long-term durability data across all therapeutic doses of TSHA-120 in giant axonal neuropathy (GAN).

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 reported positive initial serum β-hexosaminidase A (Hex A) enzyme activity data for TSHA-101 in patients with Sandhoff and Tay-Sachs diseases, which represent two forms of GM2 gangliosidosis. Today’s data are the first ever to support the bicistronic vector approach in humans delivering both HEXA and HEXB genes in the endogenous ratio.

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 RA Session II, President, Founder and CEO of Taysha, will present a corporate overview at the 40th Annual J.P. Morgan Healthcare Conference on Thursday, January 13, 2022 at 9:00 AM Eastern Time.

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 RA Session II, President, Founder and CEO of Taysha, will participate in two panel discussions during the LifeSci Partners 11th Annual Corporate Access Event.

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 plans to provide clinical data updates, including its intent to host two virtual investor conference calls and webcasts in January 2022 to discuss preliminary clinical data for TSHA-101 in GM2 gangliosidosis and high dose cohort and long-term clinical data for TSHA-120 in giant axonal neuropathy (GAN) from the respective ongoing Phase 1/2 trials.

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 has been added to the ICE Biotechnology Index (NYSE:ICEBIO) in accordance with the annual reconstitution of the index, effective prior to the U.S. market open on Monday, December 20, 2021.

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 preliminary clinical safety data for the first-generation construct in CLN7 disease. Preliminary clinical efficacy and safety data will be presented at the 18th Annual WORLDSymposium in February 2022 by Dr. Ben Greenberg, Vice Chair of Clinical and Translational Research and Professor in the departments of Neurology ad Pediatrics at UT Southwestern (UTSW). In addition, UTSW has completed the design of a next-generation construct, which is expected to further improve potency, packaging efficiency and manufacturability as well as reduce risk of immunogenicity over the first-generation construct.

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 the initiation of clinical development of TSHA-118 for the treatment of CLN1 disease under a recently approved Clinical Trial Application (CTA). Queen’s University in Ontario, Canada has been selected as the initial clinical site under the direction of Dr. Jagdeep Wahlia. There is also an open investigational new drug application (IND) in the United States for TSHA-118 in CLN1 disease.

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 a late-breaking abstract and poster presentation by Dr. Rachel Bailey, Assistant Professor at UT Southwestern Medical Center on positive preclinical data for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy at the American Epilepsy Society Annual Meeting on December 6th 2021.

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 its participation in the Stifel 2021 Virtual Healthcare Conference, Jefferies London Healthcare Conference, and the 4th Annual Evercore ISI HealthCONx Virtual Conference.

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 report its financial results for the third quarter ended September 30, 2021, and host a corporate update conference call and webcast on Wednesday, November 10, 2021, at 8:00 AM Eastern Time.

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 its participation in a fireside chat at the Jefferies Gene Therapy & Editing Summit.

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 sponsored genetic testing for giant axonal neuropathy (GAN) in partnership with GeneDx, Inc., a leader in genomic analysis and a wholly owned subsidiary of BioReference Laboratories, Inc., an OPKO Health company (NASDAQOPK). Under the partnership, Taysha will sponsor the inclusion of a genetic marker to test for GAN in the GeneDx hereditary neuropathy panel free of charge to individuals at risk for or suspected of having GAN.

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 it will host a virtual key opinion leader (KOL) webinar on TSHA-106 for the treatment of Angelman syndrome on Tuesday, October 26, 2021, from 10:00 a.m. to 1:00 p.m. ET.

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 has been granted orphan drug designation from the European Commission for TSHA-101, an AAV9-based bicistronic gene replacement therapy in development for GM2 gangliosidosis, also called Tay-Sachs or Sandhoff disease.

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 its participation in a fireside chat at Chardan’s 5th Annual Genetic Medicines Conference and a panel discussion and corporate presentation at the Cell & Gene Meeting on the Mesa on October 12, 2021. Meeting on the Mesa is the largest annual partnering meeting dedicated solely to the cell and gene therapy sector, attracting more than 1,200 attendees through the three-day program and featuring presentations and talks by major companies in the space.

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 has been granted orphan drug designation from the European Commission for TSHA-102, an AAV9-based gene replacement therapy in development for Rett syndrome.

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 publication of new preclinical data for TSHA-104 in SURF1-associated Leigh syndrome in Molecular Therapy: Methods & Clinical Development.

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 it will host a virtual key opinion leader (KOL) webinar on TSHA-102 for the treatment of Rett syndrome on Wednesday, September 22, 2021, from 10:00 a.m. to 12:30 p.m. ET.

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 its participation in the following investor healthcare conferences: Citi’s 16th Annual BioPharma Conference, Morgan Stanley 19th Annual Global Healthcare Conference, 2021 Wells Fargo Virtual Healthcare Conference, HC Wainwright 23rd Annual Global Investment Conference and the 2021 Cantor Virtual Global Healthcare Conference and the SVB Leerink CybeRx Series.

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 has been granted orphan drug designation from the European Commission for TSHA-105, an AAV9-based gene therapy in development for SLC13A5-related epilepsy.

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 it will host a virtual key opinion leader (KOL) webinar on TSHA-118 for the treatment of CLN1 disease on Monday, August 30, 2021, from 10:00 a.m. to 12:30 p.m. ET.

Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system in both rare and large patient populations, today announced that it has entered into a loan and security agreement with Silicon Valley Bank (SVB) that provides Taysha with up to $100 million of borrowing capacity.

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 report its financial results for the second quarter ended June 30, 2021, and host a corporate update conference call and webcast on Monday, August 16, 2021, at 8:00 AM Eastern Time.

Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivitol-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 its participation in a fireside chat for the BTIG Virtual Biotechnology Conference and a panel for the 2021 Wedbush Pacgrow Healthcare Virtual Conference.

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 virtual manufacturing day for analysts and investors. The event will be webcast live on Tuesday, July 27, 2021, from 10:00 a.m. to 1:00 p.m. ET.

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 its participation in a virtual fireside chat for the William Blair Biotech Focus Conference 2021.

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 the publication of new analyses of natural history data for TSHA-120 in giant axonal neuropathy, or GAN. The data were published online and will be included in the June edition of Brain, a highly esteemed neurological science peer-reviewed journal.

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 its first two-day virtual research and development (R&D) day for analysts and investors. The event will be webcast live on June 28 and June 29, 2021 from 10:00 a.m. to 1:00 p.m. ET each day.

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 its participation in virtual fireside chats at the LifeSci Genetics Medicine Conference and LSX Biotech Growth CEO Forum.

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 its first annual stockholder meeting will be held June 17, 2021 at 10 am ET via webcast.

Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivitol-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 its participation in a virtual fireside chat for the Jefferies Virtual Healthcare Conference, Goldman Sachs 42nd Annual Global Healthcare Conference, and the JMP Securities Life Science Conference.

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 has joined the newly formed Rare Disease Company Coalition, a first-of-its-kind alignment of life sciences companies committed to discovering, developing and delivering rare disease treatments for the patients they collectively serve.

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 the most recent addition to its leadership team with the appointment of industry veteran Mary Newman as Chief Development Officer. Ms. Newman will oversee program and portfolio management, as well as translational sciences, and will report to Suyash Prasad, MBBS, M.Sc., MRCP, MRCPCH, FFPM, Taysha’s Chief Medical Officer and Head of Research and Development.

Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, 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) in both rare and large patient populations, today announced its participation in a virtual fireside chat at the 2021 RBC Capital Markets Global Healthcare Conference and a virtual presentation at the Oppenheimer Rare & Orphan Disease Summit.

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 reported financial results for the first quarter ended March 31, 2021 and provided a corporate update.

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 the publication of new preclinical data for TSHA-102 in Rett syndrome. The data were published online and will be included in the May edition of Brain, a highly esteemed neurological science peer-reviewed journal.

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 preclinical data from its investigational gene therapy programs will be presented at the 24th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT), which will be held virtually May 11-14, 2021.

Taysha Gene Therapies, Inc. (Nasdaq: TSHA) (“Taysha”), a patient-centric, 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) in both rare and large patient populations, today announced that it will report its financial results for the first quarter ended March 31, 2021, and host a corporate update conference call and webcast on Tuesday, May 11, 2021, at 8:00 AM Eastern Time.

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.

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 the acquisition of exclusive worldwide rights to a clinical-stage AAV9 gene therapy program, now known as TSHA-120, for the treatment of giant axonal neuropathy (GAN). TSHA-120 is an intrathecally dosed AAV9 gene therapy currently being evaluated in a clinical trial for the treatment of GAN. The trial is being conducted by the National Institutes of Health (NIH) in close collaboration with a leading patient advocacy group focused on finding treatments and cures for GAN. TSHA-120 has received rare pediatric disease and orphan drug designations from the U.S. Food and Drug Administration (FDA) for the treatment of GAN.