1. Scrip
  2. >>Advanced Therapies
  3. >>Cell Therapies

Hemophilia Seen As Good Testing Ground For Commercializing Gene Therapy

 
• By Mandy Jackson

With a well characterized genetic driver, a clear marker to show whether it's working, and available treatments that carry high annual costs, hemophilia may be a good testing ground for commercializing gene therapy, but payers still have to work out how to reimburse the one-time medicines.

3-D Rendering Red Blood Cells

Hemophilia may be one of the clearest examples of a win-win situation for the gene therapy field, because it's a disease with a well-characterized genetic cause, a clear marker of whether treatment is working, regulatory support for novel agents and the currently available therapies are incredibly expensive.

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required
Start Free Trial

Already a subscriber?

More from Cell Therapies

Cartesian Maps Out CAR-T Strategy With Simpler Logistics In Mind

 
• By Alaric DeArment

The company announced positive Phase IIb data for Descartes-08 in myasthenia gravis, with especially strong efficacy among patients without prior biologic treatment.

What Is Fuelling Cell And Gene Therapies In India?

 
• By Vibha Ravi

Bharat Biotech is the latest entrant in India’s cell and gene therapy space, joining majors like Intas Pharma, Sun Pharma, Cipla and Dr. Reddy’s. An indigenously developed CAR-T cell therapy launched last year, what is driving growing interest and success in cell and gene therapies?

Vertex Ends One Type 1 Diabetes Cell Therapy, Shifts Focus To Another

 
• By Joseph Haas

Seeking cell therapy approaches to cure type 1 diabetes, Vertex abandons a candidate encapsulated to avoid immune system detection but hopes to file another candidate for approval in 2026.

Japan T-Reg Venture RegCell Raises New Funds, Relocates To US

 
• By Lisa Takagi

Kyoto-based venture moves HQ to California to expand R&D and business outreach for its regulatory T-cell technology, as it raises around $46m in public and private funding.

More from Advanced Therapies

What Is Fuelling Cell And Gene Therapies In India?

 
• By Vibha Ravi

Bharat Biotech is the latest entrant in India’s cell and gene therapy space, joining majors like Intas Pharma, Sun Pharma, Cipla and Dr. Reddy’s. An indigenously developed CAR-T cell therapy launched last year, what is driving growing interest and success in cell and gene therapies?

Lilly Licenses Sangamo’s Capsid Technology For CNS Gene Therapy

 
• By Alaric DeArment

Deal Snapshot: Lilly is the third company to sign a licensing deal for STAC-BBB with Sangamo, which also aims to secure a deal for its Fabry disease program in the second quarter.

Atsena To Advance XLRS Gene Therapy With Latest Funding Round

 
• By Alaric DeArment

The company closed a $150m series C funding round shortly after opening the second part of its Phase I/II study of ATSN-201 and partnering with a Nippon Shinyaku on ATSN-101.