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Latest From bluebird bio
Accelerated Approval: Gene Therapy Confirmatory Trial Timelines Can Stretch
Pink Sheet infographic looks at the nascent space of gene therapy accelerated approvals, where confirmatory trial timelines can run many years beyond approval due to disease rarity and a focus on durability of response.
Gene Therapies: US FDA Has Used Accelerated Approval Four Times Based On Intermediate Clinical Endpoints
Three of the accelerated approvals were for CAR-T products, a class that the agency considers to be gene therapies. The FDA’s September approval of bluebird bio’s Skysona for cerebral adrenoleukodystrophy was the lone non-cancer product in the group. Pink Sheet infographic tracks the required confirmatory studies and timelines for all four products.
Emerging Gene Therapy Concern: A Child Joins A Clinical Trial, But Must Be Followed Into Adulthood
The long-term follow-up requirements expected for many gene therapies could raise consent and drop-out issues, which could hinder efforts to track safety and efficacy.
No Pain, Lots To Gain: Vertex Looks For Non-Opioid Drug Breakthrough
Vertex is closing in on annual revenues of $10bn from its cystic fibrosis drugs, but non-opioid pain therapy candidate VX-548 could open up a whole new blockbuster revenue stream if Phase III studies go to plan.
- Drug Delivery
- Gene Therapy, Cell Therapy
- Large Molecule
- Other Names / Subsidiaries
- Genetix Pharmaceuticals
- Precision Genome Engineering, Inc. (Pregenen)
- Oncology Newco
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