Specifically, Pfizer will expand an 11,000-square-foot plant in Sanford, North Carolina that it acquired last year when it bought Bamboo Therapeutics, a biotech company that specializes in gene therapies for certain rare diseases.
Spark Therapeutics, Inc. recently released preliminary data from a phase 1/2 dose-escalation clinical trial for SPK-8011, the company’s investigational gene therapy candidate for hemophilia A.
UniQure N.V. has announced that is has “scaled up” the manufacturing process at its large facility in Lexington, MA.
BioMarin Pharmaceutical, Inc. recently provided updated interim results from a phase 1/2 clinical study of BMN 270, the company’s investigational hemophilia A gene therapy.
The program will allow bleeding disorders camp staff learn from one another and share best practices.
Alnylam reports the latest Phase 2 study showed that the safety and tolerability associated with fitusiran “remains encouraging.”
If effective, the therapy prompts the sustained production of FVIII and enhanced protection from bleeds.
Results showed a statistically significant and clinically meaningful reduction in bleed rate, reports states.
FDA approves the Biologics License Application (BLA) for REBINYN®
New therapy failed to show significant efficacy in a phase I/II clinical study.
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