HEXB

Taysha Gene Therapies Announces Positive Initial Biomarker Data For TSHA-101, the First Bicistronic Gene Therapy in Clinical Development, Demonstrating Normalization of β-Hexosaminidase A Enzyme Activity in Patients With GM2 Gangliosidosis

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Thursday, January 27, 2022
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TSHA-101 demonstrated expression of both HEXA and HEXB genes in the endogenous ratio, providing the ability to restore and normalize enzyme activity in GM2 gangliosidosis.

Key Points: 
  • TSHA-101 demonstrated expression of both HEXA and HEXB genes in the endogenous ratio, providing the ability to restore and normalize enzyme activity in GM2 gangliosidosis.
  • We expect to provide continued updates on the program, with additional clinical data anticipated by the end of 2022.
  • Based on natural history data, patients with asymptomatic GM2 gangliosidosis have Hex A enzyme levels that are at least 5% of normal activity.
  • TSHA-101 is an investigational gene therapy that delivers both the HEXA and HEXB genes that comprise the -hexosaminidase A enzyme.

Sio Gene Therapies Announces Granting of FDA Fast Track Designation for Investigational AXO-AAV-GM2 Gene Therapy in Patients with GM2 Gangliosidosis

Retrieved on: 
Monday, November 1, 2021
Safety, Tay–Sachs disease, Fast track (FDA), COVID-19, SIOX, Sandhoff disease, Mutation, HEXB, Gene, Industry, Neurodegeneration, GM2, Organization, Fast Track, Security (finance), U.S. Securities and Exchange Commission, Patient, Private Securities Litigation Reform Act, NASDAQ, FDA, Spinal cord, HEXA, Food, Life expectancy, Review, University of Massachusetts, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Brain, GM1, Sio, GLOBE, Imagination, AAV, Infant, Pharmaceutical industry, Vaccine

NEW YORK and DURHAM, N.C., Nov. 01, 2021 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX), a clinical-stage company focused on developing gene therapies to radically transform the lives of patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to AXO-AAV-GM2, an investigational gene therapy for the treatment of early infantile, late infantile, and juvenile-onset Tay-Sachs and Sandhoff disease. The Fast Track designation is intended to facilitate the development and review of drugs to treat serious conditions and fill an unmet medical need.

Key Points: 
  • The Fast Track designation is intended to facilitate the development and review of drugs to treat serious conditions and fill an unmet medical need.
  • GM2 gangliosidosis is a set of rare, monogenic neurodegenerative lysosomal storage disorders caused by mutations in the genes that encode the enzyme -Hexosaminidase A.
  • AXO-AAV-GM2 has received Orphan Drug Designation, Rare Pediatric Disease Designation and Fast Track Designation from theFood and Drug Administrationand is the only gene therapy in clinical development for all pediatric forms of GM2 gangliosidosis.
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.

UPDATE: Sio Gene Therapies to Present New Data at the European Society of Gene and Cell Therapy Virtual Congress 2021

Retrieved on: 
Monday, October 4, 2021
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The Company will also present a poster review of patient-level data up to 24 months from the Phase 1/2 study of AXO-Lenti-PD gene therapy for the treatment of Parkinsons disease.

Key Points: 
  • The Company will also present a poster review of patient-level data up to 24 months from the Phase 1/2 study of AXO-Lenti-PD gene therapy for the treatment of Parkinsons disease.
  • The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well.
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally.

Sio Gene Therapies to Present New Data at the European Society of Gene and Cell Therapy Virtual Congress 2021

Retrieved on: 
Monday, October 4, 2021
GM1 gangliosidoses, Food, Sandhoff disease, Neurodegeneration, Food and Drug Administration Amendments Act of 2007, HEXA, Security (finance), NASDAQ, Private Securities Litigation Reform Act, U.S. Securities and Exchange Commission, University of Massachusetts Medical School, COVID-19, Patient, European Society of Gene and Cell Therapy, GM2, Infant, Central nervous system, Gene, Type 2, Tay–Sachs disease, SIOX, Company, University, Disease, Dopamine, Cell, GM1, Brain, Organization, AAV, Imagination, GLB1, HEXB, Sio, Industry, Enzyme, GLOBE, ESGCT, Pharmaceutical industry, Vaccine

The Company will also present a poster review of patient-level data up to 24 months from the Phase 1/2 study of AXO-Lenti-PD gene therapy for the treatment of Parkinsons disease.

Key Points: 
  • The Company will also present a poster review of patient-level data up to 24 months from the Phase 1/2 study of AXO-Lenti-PD gene therapy for the treatment of Parkinsons disease.
  • The gene therapy is delivered intravenously, which has the potential to broadly transduce the central nervous system and treat peripheral manifestations of the disease as well.
  • Sio Gene Therapies combines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.
  • We are also expanding the reach of gene therapy to highly prevalent conditions such as Parkinsons disease, which affects millions of patients globally.

Taysha Gene Therapies Receives Orphan Drug Designation from the European Commission for TSHA-101 for the Treatment of Infantile GM2 Gangliosidosis

Retrieved on: 
Wednesday, September 29, 2021
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GM2 gangliosidosis is a fatal neurodegenerative disease caused by deficiency in the lysosomal enzyme -hexosaminidase A, also known as Hex A.

Key Points: 
  • GM2 gangliosidosis is a fatal neurodegenerative disease caused by deficiency in the lysosomal enzyme -hexosaminidase A, also known as Hex A.
  • GM2 gangliosidosis is a rare and fatal monogenic lysosomal storage disorder that is part of a family of neurodegenerative genetic diseases that includes Tay-Sachs and Sandhoff diseases.
  • There are no approved therapies for the treatment of the disease, and current treatment is limited to supportive care.
  • Orphan designation in the European Union includes benefits such as protocol assistance, reduced regulatory fees and market exclusivity.

Polaryx Therapeutics Announces FDA Grants Orphan Drug Designation for PLX-200 in GM2 Gangliosidoses

Retrieved on: 
Monday, August 30, 2021
Hearing, Ganglioside, Niemann–Pick disease, Glycogen storage disease, Tay–Sachs disease, RXR, Drug development, Therapy, Lists of diseases, GM2, Gene, Lysosome, Trial of the century, Sandhoff disease, HEXB, Seizure, EB, Food, Disease, Neuron, LLM, Inflammation, Lysosomal storage disease, Board, Neurodegeneration, HEXA, Enzyme, Patient, CEO, Hexosaminidase, Apoptosis, TFEB, PPAR, FDA, Gangliosidosis, GM2 gangliosidoses, Nervous system, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Pharmaceutical industry, Vaccine

PARAMUS, N.J., Aug. 30, 2021 /PRNewswire/ --Polaryx Therapeutics, Inc. ("Polaryx"), a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation for PLX-200 to treat GM2 gangliosidoses.

Key Points: 
  • PARAMUS, N.J., Aug. 30, 2021 /PRNewswire/ --Polaryx Therapeutics, Inc. ("Polaryx"), a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation for PLX-200 to treat GM2 gangliosidoses.
  • "We are very pleased to be granted Orphan Drug Designation for PLX-200 from the FDA for the treatment of GM2 gangliosidoses.
  • Furthermore, this designation validates the rationale for clinical use of PLX-200 in GM2 gangliosidoses patients.
  • Polaryx Therapeutics, Inc. is developing drug candidates for lysosomal storage disorders, for which there are currently no safe and patient-friendly treatment options available.

Taysha Gene Therapies Receives Orphan Drug Designation and Rare Pediatric Disease Designation for TSHA-101 for GM2 Gangliosidosis

Retrieved on: 
Thursday, August 27, 2020
Children, FDA, Baby, Maternity, Genetics, Other Health, Biotechnology, General Health, Pharmaceutical, Consumer, Health, Lipid storage disorders, Rare diseases, Health, Branches of biology, Clinical medicine, GM2 gangliosidoses, Sandhoff disease, Gangliosidosis, Tay–Sachs disease, Orphan drug, GM 2 gangliosidosis, HEXB

Taysha Gene Therapies , a patient-centric gene therapy company with a mission to eradicate monogenic CNS diseases, today announced that it has received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for TSHA-101, an AAV9-based gene therapy in development for GM2 Gangliosidosis.

Key Points: 
  • Taysha Gene Therapies , a patient-centric gene therapy company with a mission to eradicate monogenic CNS diseases, today announced that it has received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for TSHA-101, an AAV9-based gene therapy in development for GM2 Gangliosidosis.
  • Receiving both Orphan Drug Designation and Rare Pediatric Disease Designation by the FDA speaks to the strength of the translational data package supporting TSHA-101 for GM2 Gangliosidosis, said RA Session II, President, CEO and Founder of Taysha.
  • Furthermore, these designations also highlight the FDAs recognition that GM2 Gangliosidosis is a devastating rare disease, and we believe this is an important milestone for the GM2 Gangliosidosis community.
  • GM2 Gangliosidosis, also known as Tay-Sachs disease and Sandhoff disease, is a rare, neurodegenerative disease that causes progressive dysfunction of the central nervous system.