Spark Therapeutics, Inc.
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Latest From Spark Therapeutics, Inc.
Pfizer’s Gene Therapy Asset Chases UniQure/CSL’s Hemgenix After Pivotal Hemophilia B Success
Pfizer’s fidanacogene elaparvovec has hit the mark in a Phase III hemophilia B trial just a month after rival UniQure’s Hemgenix won US approval, with Pfizer’s plans for regulatory action raising the stakes for Hemgenix.
Value-Based Agreements On The Cards For UniQure/CSL’s $3.5m Hemophilia B Gene Therapy
While Hemgenix has courted criticism since its $3.5m price tag was unveiled, a drug pricing expert has told Scrip discounts and value-based agreements could make the therapy more palatable to payers.
Vision Possible: Why Eyestem Is 'Bullish' About Its Dry AMD Candidate
Eyestem founder and CEO talks to Scrip about progress made by the firm’s experimental treatment for dry age-related macular degeneration, cell and gene therapy pricing considerations and the wider competitive landscape. The company is also developing treatments for retinitis pigmentosa and idiopathic pulmonary fibrosis.
Round Two: BioMarin Resubmits Hemophilia A Gene Therapy Roctavian In US
BioMarin maintains its lead in the race to win approval for a hemophilia A gene therapy in the US by resubmitting Roctavian two years after the FDA issued a CRL in response to the original BLA.
Company Information
- Industry
-
Pharmaceuticals
- Drug Delivery
-
Drug Delivery
- Macromolecule
- Site Specific
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Biotechnology
- Gene Therapy, Cell Therapy
- Other Names / Subsidiaries
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- Genable Technologies Limited
- Roche Holding AG (RHHBY)
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