Sandhoff disease

Sandhoff Disease Drug Research Report 2022: Comprehensive Insights About 5+ Companies and 5+ Pipeline Drugs Featuring IntraBio, Sanofi, Polaryx Therapeutics, & Azafaros - ResearchAndMarkets.com

Retrieved on: 
Monday, October 24, 2022

The "Sandhoff disease - Pipeline Insight, 2022," report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Sandhoff disease pipeline landscape.

Key Points: 
  • The "Sandhoff disease - Pipeline Insight, 2022," report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Sandhoff disease pipeline landscape.
  • The assessment part of the report embraces, in depth Sandhoff disease commercial assessment and clinical assessment of the pipeline products under development.
  • The companies and academics are working to assess challenges and seek opportunities that could influence Sandhoff disease R&D.
  • The companies which have their Sandhoff disease drug candidates in the most advanced stage, i.e.

CANbridge-UMass Chan Medical School Gene Therapy Research to be Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

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Wednesday, May 4, 2022

Researchers will present findings from an animal study exploring a novel second-generation gene therapy for the potential treatment of spinal muscular atrophy (SMA).

Key Points: 
  • Researchers will present findings from an animal study exploring a novel second-generation gene therapy for the potential treatment of spinal muscular atrophy (SMA).
  • This is the first data to come out of the CANbridge rare disease gene therapy research collaboration with the UMass Chan Medical School, which is focused on developing gene therapy treatments for neuromuscular conditions with unmet medical needs.
  • The Horae Gene Therapy Center faculty is interdisciplinary, including members from the departments of Pediatrics, Microbiology & Physiological Systems, Biochemistry & Molecular Pharmacology, Neurology, Medicine and Ophthalmology.
  • Additionally, the University of Massachusetts Chan Medical School conducts clinical trials on site and some of these trials are conducted by the investigators at the Gene Therapy center.

Sio Gene Therapies Provides Corporate Update

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Wednesday, April 27, 2022

NEW YORK and DURHAM, N.C., April 27, 2022 (GLOBE NEWSWIRE) -- Sio Gene Therapies Inc. (NASDAQ: SIOX) today announced that it has provided the required notice to the University of Massachusetts to terminate its licensing agreement to develop and commercialize gene therapy product candidates, including AXO-AAV-GM1 and AXO-AAV-GM2, for the treatment of GM1 gangliosidosis and GM2 gangliosidosis (including Tay-Sachs disease and Sandhoff disease), respectively.

Key Points: 
  • The Company will explore options such as the potential for a company sale, merger, business combination, or other transactions designed to maximize shareholder value.
  • All forward-looking statements are based on estimates and assumptions by Sios management that, although Sio believes to be reasonable, are inherently uncertain.
  • All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Sio expected.
  • Sio undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Sio Gene Therapies Announces Corporate Updates and Fiscal Third Quarter 2021 Financial Results

Retrieved on: 
Friday, February 11, 2022

I look forward to sharing continued updates across each of these programs as they reach meaningful clinical milestones.

Key Points: 
  • I look forward to sharing continued updates across each of these programs as they reach meaningful clinical milestones.
  • General and administrative expenses were $4.1 million for the three months ended December31, 2021 and $4.2 million for the three months ended December31, 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.

Azafaros Presents Positive Clinical and Preclinical Data Supporting Development of Lead Compound AZ-3102 in Lysosomal Storage Disorders at the 18th Annual WORLDSymposium™ Conference

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Monday, February 14, 2022

Azafaros B.V. announced positive clinical data from its first-in-human Phase 1 study with AZ-3102, the companys lead program in development as a potential treatment for pediatric neurogenetic lysosomal storage disorders (LSDs).

Key Points: 
  • Azafaros B.V. announced positive clinical data from its first-in-human Phase 1 study with AZ-3102, the companys lead program in development as a potential treatment for pediatric neurogenetic lysosomal storage disorders (LSDs).
  • All four presentations were made at the 18th Annual WORLDSymposium, held February 7 11, 2022, in San Diego, CA.
  • AZ-3102 is designed to selectively inhibit two enzymes involved in glycosphingolipid metabolism, called glucosylceramide synthase (GCS) and non-lysosomal glucosylceramidase (GbA2).
  • Stefano Portolano, Chief Executive Officer of Azafaros, stated, The presentations at this years WORLDSymposium conference demonstrate the exciting potential of AZ-3102 in rare metabolic diseases.

Sio Gene Therapies to Present at the 18th Annual WORLDSymposium™ 2022

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Wednesday, February 2, 2022

NEW YORK andDURHAM, N.C., Feb. 02, 2022 (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 it will present data in an oral platform presentation and two poster presentations at the 18th Annual WORLDSymposium 2022, to be held February 7-11, 2022.

Key Points: 
  • NEW YORK andDURHAM, N.C., Feb. 02, 2022 (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 it will present data in an oral platform presentation and two poster presentations at the 18th Annual WORLDSymposium 2022, to be held February 7-11, 2022.
  • The Company is also collaborating with Invitae, who will present a poster on the Detect LSD program.
  • The gene therapy is delivered intravenously, which has the potential to achieve a broad central and peripheral biodistribution.
  • Sio Gene Therapiescombines cutting-edge science with bold imagination to develop genetic medicines that aim to radically improve the lives of patients.

Azafaros Receives FDA Orphan Drug Designation for AZ-3102 in GM2 Gangliosidosis

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Tuesday, February 1, 2022

Azafaros B.V. today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102, a novel oral small molecule, in GM2 gangliosidosis including both Sandhoff and Tay-Sachs diseases.

Key Points: 
  • Azafaros B.V. today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for AZ-3102, a novel oral small molecule, in GM2 gangliosidosis including both Sandhoff and Tay-Sachs diseases.
  • The ODD for GM2 gangliosidosis has been granted based on efficacy demonstrated in a Sandhoff mouse model, including a clear effect on animal survival.
  • Orphan Drug Designation by the US FDA provides drug developers with special status and incentives to facilitate the development of therapeutics for rare diseases affecting fewer than 200,000 people in the US.
  • Azafaros completed a first-in-human clinical trial with AZ-3102 in healthy volunteers in 2021 and received Orphan Drug Designation in GM2 Gangliosidosis from the FDA in February 2022.

Sio Gene Therapies Announces Prioritization of Lead Gene Therapy Programs in GM1 and GM2 Gangliosidosis, Extension of Cash Runway, and CEO Transition

Retrieved on: 
Monday, January 31, 2022

NEW YORK and DURHAM, N.C., Jan. 31, 2022 (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 provided a corporate update announcing the prioritization of AXO-AAV-GM1 and AXO-AAV-GM2, its clinical stage AAV gene therapy programs for GM1 and GM2 gangliosidosis (Tay-Sachs/Sandhoff disease). The portfolio prioritization extends the Company’s estimated cash runway into the second half of 2023, beyond multiple key clinical milestones for both gene therapy programs. With this prioritization, the company intends to terminate its licensing agreement with Oxford Biomedica for AXO-Lenti-PD, its lentiviral gene therapy program for Parkinson’s disease.

Key Points: 
  • The portfolio prioritization extends the Companys estimated cash runway into the second half of 2023, beyond multiple key clinical milestones for both gene therapy programs.
  • With this prioritization, the company intends to terminate its licensing agreement with Oxford Biomedica for AXO-Lenti-PD, its lentiviral gene therapy program for Parkinsons disease.
  • I believe our programs in GM1 and GM2 have the ability to transform the care for patients and deserve this heightened focus.
  • Dr. Cheruvu added, Serving as the CEO of Sio for the last four years has been an immense privilege.

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

Retrieved on: 
Thursday, January 27, 2022

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.

Azafaros Announces Multiple Presentations at 18th Annual WORLDSymposium™

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Tuesday, January 18, 2022

Azafaros B.V. today announced its participation at the 18th Annual WORLDSymposium, a research conference dedicated to lysosomal storage diseases, taking place from February 7 11, 2022, in San Diego, CA.

Key Points: 
  • Azafaros B.V. today announced its participation at the 18th Annual WORLDSymposium, a research conference dedicated to lysosomal storage diseases, taking place from February 7 11, 2022, in San Diego, CA.
  • Details on the presentations are as follows:
    Title: A first-in-human, randomized, double-blind, placebo-controlled, ascending single- and multiple dose study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of the L-ido azasugar AZ-3102 in healthy volunteers; Virtual Poster LB- 54.
  • Title: AZ-3102, a novel brain-penetrant small molecule, significantly improves survival of Sandhoff disease mice; Late-Breaking Science Platform Presentation, Poster LB-70
    The posters will be available on the Azafaros website following presentation at the WORLDSymposium conference.
  • By applying its know-how, network, and courage, the Azafaros team challenges traditional development pathways to rapidly bring new drugs to the rare disease patients who need them.