Spark Therapeutics, Inc.

AAV Gene Therapy Market Projected to Reach $107.2 Billion by 2035, Growing at 40% CAGR

3 months ago

• The adeno-associated virus (AAV) gene therapy market is expected to surge from $2.7 billion in 2024 to $107.2 billion by 2035, driven by advances in genetics, biotechnology, and personalized medicine. • Marketed AAV therapies include Luxturna for hereditary retinal dystrophy, Zolgensma for spinal muscular atrophy, and Glybera for lipoprotein lipase deficiency, with numerous promising candidates in late-stage development. • Improved diagnostic technologies, regulatory support, and significant investment from companies like Spark Therapeutics, Novartis, and BioMarin are accelerating the development of next-generation AAV therapies for previously untreatable genetic disorders.

Hemophilia A and B Pipeline Advances with Novel Therapies in Clinical Trials

8 months ago

• Several companies are actively developing novel therapies for Hemophilia A and B, with many in various phases of clinical trials, showing promise for improving treatment options. • TiumBio Co., Ltd. has submitted a Clinical Trial Application to initiate a Phase 1b investigation of TU7710, a new recombinant activated factor VII for hemophilia patients with inhibitors. • The FDA approved ALTUVIIIO (efanesoctocog alfa) in February 2023, a factor VIII replacement therapy for adults and children with Hemophilia A, for routine prophylaxis and on-demand treatment. • BioMarin received FDA approval for Roctavian in June 2023, a single-dose gene therapy for severe Hemophilia A, marking a significant advancement in treatment.