Coronavirus: Young Canadians with rare diseases feeling impact of pandemic

“It’s bittersweet because he’s taking a big brother role, even though he’s the little brother,”...

Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis)

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 03, 2021 (GLOBE NEWSWIRE) — Sio Gene...

Taysha Gene Therapies Announces Queen’s University’s Receipt of Clinical Trial Application Approval from Health Canada for Phase 1/2 Clinical Trial of TSHA-101 for the Treatment of Infantile GM2 Gangliosidosis

DALLAS–(BUSINESS WIRE)–Dec. 21, 2020– Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company...

Health Canada approves $2.8M treatment for spinal muscular atrophy

Health Canada has approved Zolgensma, which is bringing renewed hope to Alberta families that have...

Sio Gene Therapies Announces Positive Six-Month Follow-Up Data from Low-Dose Cohort of Phase 1/2 Trial of AXO-AAV-GM1 for GM1 Gangliosidosis

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Dec. 15, 2020 (GLOBE NEWSWIRE) — Sio Gene...

Ottawa baby a pioneer of gene therapy for rare disease

“We are just so blessed that we live in the right place and that Aidan...

Sio Gene Therapies Announces First Patient Dosed in High-Dose Cohort of AXO-AAV-GM1 Clinical Trial in Patients with GM1 Gangliosidosis

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Dec. 02, 2020 (GLOBE NEWSWIRE) — Sio Gene...

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

AXO-AAV-GM2 is the first investigational gene therapy to receive IND clearance in Tay-Sachs and Sandhoff...

Nobel Prize in Chemistry 2020: Genetic scissors: a tool for rewriting the code of life

Emmanuelle Charpentier and Jennifer Doudna are awarded the Nobel Prize in Chemistry 2020 for discovering...

Axovant and Viralgen Sign Strategic Gene Therapy Development and Manufacturing Partnership

Partnership secures access to cGMP capacity and resources at Viralgen to support the development and...