Atsena reels in $150M to advance work on ocular gene therapies
Title
Atsena Therapeutics Secures $150M Series C Funding to Drive Ocular Gene Therapy Advancements
Keywords
- Atsena Therapeutics
- Ocular gene therapy
- Series C funding
- X-linked retinoschisis (XLRS)
- Leber congenital amaurosis type 1 (LCA1)
- FDA designations
- Adeno-associated virus (AAV) vectors
- Inherited retinal diseases
Key Facts
- Company Overview:
Atsena Therapeutics, a clinical-stage gene therapy company, focuses on genetic-based treatments to address rare inherited retinal diseases that cause vision loss and blindness.
- Lead Programs:
* - ATSN-201:*
A gene therapy targeting X-linked retinoschisis (XLRS), a rare childhood-onset retinal disease leading to blindness. Currently in a Phase I/II trial, this therapy utilizes novel adeno-associated virus (AAV) vectors with enhanced lateral spreading capabilities.
* - ATSN-101:*
Investigational therapy for Leber congenital amaurosis type 1 (LCA1) caused by GUCY2D mutations. Positive results were observed in a completed Phase I/II trial, with plans for global trials in collaboration with Nippon Shinyaku.
- Funding Details:
- Atsena raised $150 million in an oversubscribed Series C financing round led by Bain Capital. Key investors included Wellington Management, Lightstone Ventures, Foundation Fighting Blindness, and others.
- Proceeds will fuel the clinical advancement of ATSN-201 and ATSN-101, alongside expanding its preclinical pipeline and the use of its innovative AAV.SPR capsid technology.
- FDA Designations:
ATSN-201 has achieved FDA Fast Track, Orphan Drug, and Rare Pediatric Disease designations, reflecting its potential impact on presently untreated conditions.
- Technology:
The company employs advanced AAV vectors for efficient gene delivery, capable of spreading beyond the injection site to treat fragile areas of the retina effectively.
- Leadership and Partnerships:
- The funding round facilitated the addition of Norbert Riedel, a seasoned biopharmaceutical executive, to Atsena's Board of Directors.
- Strategic collaboration with Japanese pharmaceutical company Nippon Shinyaku to advance ATSN-101 globally.
- Significance:
Gene therapy in ophthalmology represents a promising frontier, with Atsena's progress contributing to potential cures for inherited retinal diseases rather than merely managing symptoms.