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Serious Side Effect Sidelines Gene-Edited Sickle Cell Therapy from Graphite Bio

MedCity News

Graphite Bio voluntarily paused a Phase 1/2 test of its gene-edited therapy for sickle cell disease after the first patient in the study developed a serious blood complication. The setback will also delay plans to reach the clinic with another genetic medicine in its pipeline.

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AbbVie Expands Psychiatric Drug Prospects, Inking R&D Pact With Neuroscience Startup

MedCity News

The companies aim to bring patients drugs that offer the benefit of psychedelics but with a better side effect profile. AbbVie’s alliance with Gilgamesh Pharmaceuticals is pursuing next-generation psychiatric medications.

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FDA Nod in Duchenne Helps Wider Swath of Patients With the Rare Muscle Disease

MedCity News

Corticosteroids are a standard first-line treatment for Duchenne muscular dystrophy, but these drugs have many side effects. FDA approval of Italfarmaco’s Duvyzat is the first nonsteroidal drug to pass the agency’s regulatory bar for treating this rare disease.

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MHRA approves first gene therapy for infusion into the brain

European Pharmaceutical Review

The product, produced by biopharma company PTC Therapeutics, is approved for patients 18 months and over. The most common side effects observed were initial insomnia, irritability and dyskinesia. It has been granted marketing authorisation by the Medicines and Healthcare Products Regulatory Agency (MHRA) in Great Britain.

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Merck’s $1.3B Imago Buyout Brings Blood Cancer Drug to Rival BMS, Incyte & GSK

MedCity News

The deal gives the pharmaceutical giant a small molecule that could provide an alternative to a class of drugs that introduce dangerous side effects. Merck is acquiring Imago BioSciences, a clinical-stage biotech whose lead drug is in development for myelofibrosis and other blood cancers.

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GSK to Gain Chronic Cough Drug With $2B Buyout of Bellus Health

MedCity News

Acquiring Bellus Health gives GSK a drug for recurrent chronic cough that could offer a better side effect profile compared to a Merck drug that addresses the same target. Bayer was also pursuing the target, but stopped work on its drug candidate last year.

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Novartis warns of two patients deaths after dosing with gene therapy Zolgensma

MedCity News

Liver complications are a known side effect risk of Zolgensma and other genetic medicines that are delivered by engineered viruses. The two deaths reported following treatment with Novartis’s Zolgensma mark the first fatal cases of acute liver failure associated with the gene therapy.