Biomarin Pharmaceutical
- Country
- 🇨🇦Canada
- Ownership
- -
- Established
- 1997-01-01
- Employees
- -
- Market Cap
- $17.2B
- Website
- http://www.biomarin.com
Spruce Biosciences Acquires Tralesinidase Alfa for Sanfilippo Syndrome Type B Treatment with BLA Submission Planned for 2026
Spruce Biosciences has acquired tralesinidase alfa enzyme replacement therapy (TA-ERT) for the treatment of Sanfilippo Syndrome Type B, a neurodegenerative and fatal genetic disease with no FDA-approved treatments.
Phenylketonuria Pipeline Shows Robust Growth with 25+ Therapies in Development
DelveInsight's latest report reveals a robust phenylketonuria (PKU) pipeline with over 20 companies developing 25+ therapeutic candidates, signaling significant advancement in treatment options for this rare metabolic disorder.
Major Advances in Angioedema Pipeline: Intellia's Gene Therapy Enters Phase 3 as Multiple Companies Race for Novel Treatments
Intellia Therapeutics has initiated Phase 3 trials for NTLA-2002, a groundbreaking CRISPR-based gene therapy for hereditary angioedema, with potential U.S. launch targeted for 2027.
AAV Gene Therapy Market Projected to Reach $107.2 Billion by 2035, Growing at 40% CAGR
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.