Tay–Sachs disease

Sio Gene Therapies to Participate in Upcoming Investor & Scientific Conferences

Retrieved on: 
Thursday, March 11, 2021
Branches of biology, Rare diseases, Lipid storage disorders, Health sciences, Life sciences, Neurodegenerative disorders, Congenital disorders, Applied genetics, Sandhoff disease, Tay–Sachs disease, Gene therapy, Therapy

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.

Key Points: 
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies.
  • We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally.
  • Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies.

Sio Gene Therapies to Present at the 10th Annual SVB Leerink Global Healthcare Conference

Retrieved on: 
Tuesday, February 16, 2021
Branches of biology, Rare diseases, Lipid storage disorders, Health sciences, Life sciences, Neurodegenerative disorders, Congenital disorders, Medical genetics, Sandhoff disease, Tay–Sachs disease, Gene therapy, Therapy

Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.

Key Points: 
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies.
  • We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally.
  • Led by an experienced team of gene therapy development experts, and supported by collaborations with premier academic, industry and patient advocacy organizations, Sio is focused on accelerating its candidates through clinical trials to liberate patients with debilitating diseases through the transformational power of gene therapies.

Sio Gene Therapies Announces Receipt of $11.6 Million from Closing of the Sale of Arvelle Therapeutics

Retrieved on: 
Thursday, February 4, 2021
Rare diseases, Lipid storage disorders, Neurodegenerative disorders, Health, Branches of biology, Clinical medicine, Sandhoff disease, Tay–Sachs disease, Gangliosidosis, European Medicines Agency, GM2 gangliosidoses

Per the terms of the sale, additional payments to Sio Gene Therapies Inc. are expected over time, including a payment of approximately $4.8 million by mid-2021 upon marketing approval of cenobamate by the European Medicines Agency (EMA).

Key Points: 
  • Per the terms of the sale, additional payments to Sio Gene Therapies Inc. are expected over time, including a payment of approximately $4.8 million by mid-2021 upon marketing approval of cenobamate by the European Medicines Agency (EMA).
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • Our current pipeline of clinical-stage candidates includes the first potentially curative AAV-based gene therapies for GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases, which are rare and uniformly fatal pediatric conditions caused by single gene deficiencies.
  • Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Sio Gene Therapies Announces Corporate Updates and Financial Results for Second Fiscal Quarter Ended September 30, 2020

Retrieved on: 
Friday, November 13, 2020
Lipid storage disorders, Rare diseases, Branches of biology, Health, Clinical medicine, Sandhoff disease, Tay–Sachs disease, Gangliosidosis, GM1, Axovant Sciences

In recent months, we have taken significant strides forward with our AAV-based gene therapy programs in GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases.

Key Points: 
  • In recent months, we have taken significant strides forward with our AAV-based gene therapy programs in GM1 gangliosidosis and Tay-Sachs/Sandhoff diseases.
  • Expect to complete dosing of juvenile (Type II) patients in the high-dose cohort of the ongoing AXO-AAV-GM1 clinical study before year-end 2020.
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected.

Axovant Gene Therapies Announces FDA Clearance of IND for AXO-AAV-GM2 Gene Therapy in Tay-Sachs and Sandhoff Diseases

Retrieved on: 
Monday, November 9, 2020
Branches of biology, Rare diseases, Lipid storage disorders, Health sciences, Life sciences, Neurodegenerative disorders, Axovant Sciences, Sandhoff disease, Gangliosidosis, Tay–Sachs disease, GM1, Therapy

AXO-AAV-GM2 is the first investigational gene therapy to achieve IND clearance for Tay-Sachs and Sandhoff diseases.

Key Points: 
  • AXO-AAV-GM2 is the first investigational gene therapy to achieve IND clearance for Tay-Sachs and Sandhoff diseases.
  • Along with AXO-AAV-GM1 for GM1 gangliosidosis, todays announcement represents the second IND clearance for Axovants gene therapy pipeline in the last 12 months, said Gavin Corcoran, M.D., Chief R&D Officer of Axovant.
  • Each program captures the essence of our mission to liberate patients from debilitating neurological diseases by rapidly advancing these gene therapy programs into clinical development.
  • Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases.

Axovant Gene Therapies to Present AXO-Lenti-PD Program Update During Virtual Parkinson’s Disease R&D Day on October 30, 2020

Retrieved on: 
Thursday, October 29, 2020
Branches of biology, Lipid storage disorders, Rare diseases, Health, Clinical medicine, GM2 gangliosidoses, Sandhoff disease, Gangliosidosis, Tay–Sachs disease, GM1, Axovant Sciences, Degenerative disease

The Company expects to provide an update on program timelines in the first quarter of 2021 or as program timelines are clarified.

Key Points: 
  • The Company expects to provide an update on program timelines in the first quarter of 2021 or as program timelines are clarified.
  • Axovant Gene Therapies is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurodegenerative diseases.
  • Our current pipeline of gene therapy candidates target GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease.
  • Axovant undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.

Axovant Gene Therapies Receives Rare Pediatric Disease Designation for AXO-AAV-GM2 for Tay-Sachs and Sandhoff Disease

Retrieved on: 
Tuesday, October 13, 2020
Lipid storage disorders, Rare diseases, Branches of biology, Health, Clinical medicine, GM2 gangliosidoses, Sandhoff disease, Tay–Sachs disease, Gangliosidosis, GM2, GM1, Axovant Sciences

In addition to the Rare Pediatric Disease designation, AXO-AAV-GM2 has Orphan Drug Designation (ODD) and is the first gene therapy that has been administered to children with Tay-Sachs disease.

Key Points: 
  • In addition to the Rare Pediatric Disease designation, AXO-AAV-GM2 has Orphan Drug Designation (ODD) and is the first gene therapy that has been administered to children with Tay-Sachs disease.
  • We are thrilled to bring AXO-AAV-GM2 one step closer to patients in need through this Rare Pediatric Disease designation.
  • Previously, Axovant reported the first evidence for potential disease modification in Tay-Sachs disease from an expanded access study administering investigational AXO-AAV-GM2 gene therapy in two patients with infantile (Type I) Tay-Sachs disease.
  • Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease.

Kantar Health and CATS Foundation Present Updated GM2 Disease Registry at 2020 NORD Rare Diseases & Orphan Products Breakthrough Summit

Retrieved on: 
Monday, October 12, 2020
Technology, General Health, Health, Data management, Rare diseases, Lipid storage disorders, Branches of biology, Neurodegenerative disorders, Health, Medicine, Tay–Sachs disease, Sandhoff disease, Kantar Health, Rare disease, Disease, GM2 gangliosidoses

Kantar Health, a leading global healthcare data, analytics and research provider, and the Cure & Action for Tay-Sachs (CATS) Foundation presented the latest version of the GM2 Disease Registry at the 2020 NORD Rare Diseases & Orphan Products Breakthrough Summit, held October 8-9.

Key Points: 
  • Kantar Health, a leading global healthcare data, analytics and research provider, and the Cure & Action for Tay-Sachs (CATS) Foundation presented the latest version of the GM2 Disease Registry at the 2020 NORD Rare Diseases & Orphan Products Breakthrough Summit, held October 8-9.
  • The GM2 Disease Registry (GM2DR) was established in 2015 to increase awareness of and support for clinical research on Tay-Sachs and Sandhoff Disease.
  • In the United States a rare disease is defined as a condition that affects fewer than 200,000 people.
  • As real-world evidence and commercialization partners, Kantar Health enables clients to bring safe and effective treatment to patients around the world.

Axovant Gene Therapies Receives Rare Pediatric Disease Designation for AXO-AAV-GM1 for GM1 Gangliosidosis

Retrieved on: 
Friday, October 9, 2020
Lipid storage disorders, Rare diseases, Branches of biology, Clinical medicine, Health, GM2 gangliosidoses, Lysosomal storage disease, Sandhoff disease, Gangliosidosis, GLB1, GM1, Tay–Sachs disease

In addition to the Rare Pediatric Disease designation, AXO-AAV-GM1 has Orphan Drug designation and is the only gene therapy in clinical development for both infantile (Type I) and juvenile (Type II) GM1 gangliosidosis.

Key Points: 
  • In addition to the Rare Pediatric Disease designation, AXO-AAV-GM1 has Orphan Drug designation and is the only gene therapy in clinical development for both infantile (Type I) and juvenile (Type II) GM1 gangliosidosis.
  • Receiving Rare Pediatric Disease Designation in addition to the previously granted Orphan Drug Designation for AXO-AAV-GM1 highlights the importance of expediting a therapy for GM1 Gangliosidosis, a progressive, fatal, pediatric disease with no approved treatment options, said Sean OBryan, Senior Vice President, Regulatory Affairs & Quality.
  • GM1 Gangliosidosis is a progressive and fatal pediatric lysosomal storage disorder caused by mutations in the GLB1 gene leading to impaired production of the -galactosidase enzyme.
  • Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease.

Oxford Biomedica notes Axovant’s positive six-month follow-up from second cohort of SUNRISE-PD Phase 2 trial of AXO-Lenti-PD

Retrieved on: 
Tuesday, October 6, 2020
Branches of biology, Lipid storage disorders, Rare diseases, Health, Articles, GM2 gangliosidoses, Sandhoff disease, Gangliosidosis, Oxford BioMedica, Tay–Sachs disease, OXB, GM1

Oxford Biomedica (LSE:OXB) is a leading, fully integrated, gene and cell therapy group focused on developing life changing treatments for serious diseases.

Key Points: 
  • Oxford Biomedica (LSE:OXB) is a leading, fully integrated, gene and cell therapy group focused on developing life changing treatments for serious diseases.
  • The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology, CNS disorders, liver diseases and respiratory disease.
  • Oxford Biomedica is based across several locations in Oxfordshire, UK and employs more than 580 people.
  • Our current pipeline of gene therapy candidates targets GM1 gangliosidosis, GM2 gangliosidosis (also known as Tay-Sachs disease and Sandhoff disease), and Parkinsons disease.