Sio Gene Therapies Announces Four Upcoming Oral Presentations at the 24th Annual Meeting of the American Society of Gene and Cell Therapy streetinsider.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from streetinsider.com Daily Mail and Mail on Sunday newspapers.
Published: Mar 01, 2021 By Heather McKenzie
February 28 marked Rare Disease Day, and biotech companies gathered together to raise awareness, advocate for improved access to treatment and let patients know how they are pivoting to improve patient care and access during the COVID-19 pandemic and beyond.
Rezolute Inc., a clinical-stage biopharmaceutical company developing novel therapeutics for patients with metabolic and orphan diseases, was just beginning a Phase IIb study of its lead candidate, RZ358, for Congenital hyperinsulinism (HI), when the chaos struck.
“We have hospitals around the world where the COVID ward was right next to the research ward. We had hospitals that for six months shut down entirely,” shared Erin O’Boyle, Head of Clinical Operations at Rezolute. “We had a lot of overnight stays, but then, right when we were kicking off the study and going into enrollment and starting to activate our sites, COVID hit, and it really kind of changed the
Published: Feb 03, 2021
- First potentially disease-modifying gene therapy for GM2 gangliosidosis to enter clinical studies
- Expect to continue patient identification, screening, and enrollment in Stage 1 of the study throughout 2021
NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 03, 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 first patient with infantile Tay-Sachs disease has been dosed in a Phase 1/2 trial evaluating AXO-AAV-GM2, an investigational gene therapy for the treatment of GM2 gangliosidosis, also known as Tay-Sachs or Sandhoff disease.
Press release content from Globe Newswire. The AP news staff was not involved in its creation.
Sio Gene Therapies Announces Positive Six-Month Follow-Up Data from Low-Dose Cohort of Phase .
Sio Gene TherapiesDecember 15, 2020 GMT
- Generally well-tolerated with a favorable safety profile in five patients
- Serum beta-galactosidase enzyme activity increased in all patients at all timepoints between Day 7 and Month 6, representing an approximate doubling in enzyme activity after gene transfer
- At Month 6, enzyme activity restored to 23-57% (mean: 38%) of normal reference levels
- All five children demonstrated signs of clinical disease stability as assessed by Vineland-3 Growth Scale Value, Upright and Floor Mobility, and Clinical Global Impression (CGI) scales