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.

Gene Therapy 356

Gene Therapy Gene Trials Research 356

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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Gene Therapy Gene Trials 118

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.

Gene Therapy 82

Gene Therapy Gene Marketing 82

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.

Trials 100

Trials Gene Therapy Gene 100

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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.

Gene Therapy

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.

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Gene Therapy Gene 95

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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Gene Therapy Research Genome Genomics 52