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Top related persons:
IX - 1 hemophilia - 1 Adam Cuker - 1 Cuker - 1
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U.S Australia
Top related orgs:
Drug Administration - 1 Pfizer - 1 U.S - 1 Penn's - 1 Spark Therapeutics - 1 hemophilia - 1

Search resuls for: "Spark Therapeutics"


3 mentions found


☆ FDA approves Pfizer’s first gene therapy for rare inherited bleeding disorder
  + stars: | 2024-04-26 | by ( Annika Kim Constantino | ) www.cnbc.com   time to read: +3 min
The Food and Drug Administration on Friday approved Pfizer 's treatment for a rare genetic bleeding disorder, making it the company's first-ever gene therapy to win clearance in the U.S. The agency greenlit the drug, which will be marketed as Beqvez, for adults with moderate to severe hemophilia B who meet certain requirements. Without that protein, called factor IX, patients with hemophilia B bruise easily and bleed more frequently and for longer periods of time. The gene therapy will compete with Australia-based CSL Behring's Hemgenix, a similar treatment that won FDA approval for hemophilia B in 2022. The company is also developing a gene therapy for Duchenne muscular dystrophy, a genetic disorder that causes muscles to weaken gradually.
Persons: IX, hemophilia, Adam Cuker, Cuker Organizations: Drug Administration, Pfizer, U.S, Penn's, Spark Therapeutics, hemophilia Locations: U.S, Australia
☆ Pfizer's hemophilia B gene therapy succeeds in late-stage study
  + stars: | 2022-12-29 | by ( ) www.reuters.com   time to read: +1 min
Dec 29 (Reuters) - U.S. drugmaker Pfizer Inc (PFE.N) said on Thursday its experimental gene therapy for the treatment of hemophilia B, a rare inherited blood disorder, met its main goal in a late-stage study. The drugmaker licensed its hemophilia B gene therapy from Roche's (ROG.S) Spark Therapeutics unit in 2014 for a $20-million upfront payment. Pfizer plans to discuss the late-stage data with regulatory authorities in Europe and the United States and share additional data for the experimental therapy at a scientific conference in early 2023. According to government data, the estimated prevalence of hemophilia in the United States is 12 cases per 100,000 males for hemophilia A and 3.7 cases per 100,000 males for hemophilia B. In November, the U.S. health regulator approved the first gene therapy, CSL Ltd and uniQure's Hemgenix, to treat hemophilia B.Pfizer is also testing other experimental gene therapies in late-stage trials as potential treatments for the bleeding disorder hemophilia A and muscular disorder Duchenne muscular dystrophy.
☆ See the 20-slide presentation this biotech startup used to raise $75 million for a way to treat blindness with CRISPR
  + stars: | 2022-09-14 | by ( Andrew Dunn | ) www.businessinsider.com   time to read: +2 min
About a year ago, the startup secured an exclusive deal with CRISPR gene-editing leader Intellia. SparingVision, a Paris-based startup, just raised $75 million to advance a suite of gene-therapy and gene-editing programs to treat eye diseases. The company plans to use the funds to enter the clinic with its first gene-therapy program. The cash will also help advance SPVN50, SparingVision's first gene-editing program. The program came out of a research collaboration SparingVision struck with Intellia Therapeutics, a CRISPR gene-editing leader, last year.
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