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More Evidence Gene Therapy Might Cure Sickle Cell Disease

Drugs.com

MONDAY, June 12, 2023 -- A clinical trial that’s attempting to discover a cure for sickle cell disease has found a new gene therapy to be safe and successful in four patients. Two of the patients were treated at Cleveland Clinic Children’s in.

Therapies 264
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CAR-T for lupus: the ‘tip of the iceberg’ for cell therapy in autoimmune disease

BioPharma Drive: Drug Pricing

Since a landmark paper in 2022, drugmakers have begun nearly a dozen trials of cell therapies for lupus, with more set to start. Here’s why their efforts are worth watching.

Therapies 353
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Artiva prices $167M IPO, riding optimism for autoimmune cell therapy

BioPharma Drive: Drug Pricing

The offering comes days after the publication of a paper showing what analysts said was early proof that “off-the-shelf” cell therapies can treat inflammatory diseases.

Therapies 327
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Gene therapy gets a turbo boost

Science Daily: Pharmacology News

For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. Gene therapy, the idea of fixing faulty genes with healthy ones, has held immense promise. But a major hurdle has been finding a safe and efficient way to deliver those genes.

Therapies 297
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FDA Advisors Say New Gene Therapy for Sickle Cell Disease, Exa-cel, is Safe

Drugs.com

1, 2023 -- A new gene therapy for sickle cell disease was deemed safe by a U.S. WEDNESDAY, Nov. Food and Drug Administration advisory panel on Tuesday, paving the way for full approval by early December. The FDA had already decided that the.

Therapies 263
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New gene delivery vehicle shows promise for human brain gene therapy

Science Daily: Pharmacology News

In an important step toward more effective gene therapies for brain diseases, researchers have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein.

Therapies 315
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New gene therapy for muscular dystrophy offers hope

Science Daily: Pharmacology News

A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the future, repairing those muscles.

Therapies 303