Patient dies in Pfizer study of Duchenne gene therapy
Bio Pharma Dive
MAY 7, 2024
Pfizer said the patient, a young boy who was treated earlier last year, had died suddenly. The company is working with trial researchers to investigate further.
Bio Pharma Dive
MAY 7, 2024
Pfizer said the patient, a young boy who was treated earlier last year, had died suddenly. The company is working with trial researchers to investigate further.
pharmaphorum
MAY 7, 2024
Pfizer has paused dosing in a study of its Duchenne muscular dystrophy gene therapy after a sudden patient death in another trial
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Fierce Pharma
MAY 7, 2024
After a 2023 approval from the FDA, Krystal Biotech has collected more than $95 million from its launch of Vyjuvek, the first treatment for the rare skin disease dystrophic epidermolysis bullo | The topical gene therapy has pulled in nearly $100 million during its first three quarters on the U.S.
pharmaphorum
MAY 7, 2024
Two-year data from a phase 1/2 trial of eyeDNA Therapeutics' gene therapy for retinitis pigmentosa sets up pivotal trials, says the new biotech.
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As demand for advanced therapies increases, so does the need for more specialized supply chain support, as these products have strict transportation and handling requirements. It is crucial to ensure supply chain risks are mitigated and have proactive strategies in place to address unforeseen challenges before they become an issue.
Fierce Pharma
MAY 7, 2024
As of mid-April, Vertex said it had collected 5 patients' cells for eventual treatment with sickle cell disease therapy Casgevy. That puts Vertex ahead of its gene therapy rival bluebird bio, which recently said it had collected just one patient's cells for its own SCD treatment Lyfgenia.
BioPharma Reporter
MAY 7, 2024
Broken String Biosciences, a genomics company driving cell and gene therapy developing, has entered a research collaboration with the Francis Crick Institute, a biomedical discovery institute dedicated to understanding the biology underlying health and disease.
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