Beam Therapeutics reported promising Phase 1/2 data for BEAM-302, demonstrating durable gene correction for AATD. See why I ...
Gene therapy, which promises a possible cure for rare diseases like sickle cell, is losing early investors to higher-reward ...
In the last year, some drugmakers have pulled back from the sector, including Pfizer, which recently stopped selling its gene ...
A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
The fall in CRSP stock can be attributed to a lack of pipeline updates. Despite securing approval for its first marketed product in early 2024, it is yet to record product sales.
Zynext Ventures is investing in Illexcor Therapeutics to advance the clinical development of ILX002, its lead candidate for ...
The gene therapy world is in turmoil, but Arbor, armed with more than a billion dollars in partnerships and raises, is going forward.
Sebastien Beauzile, 21, has been cured of sickle cell anemia, according to his doctors at Cohen Children’s Medical Center.
Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...
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New Gene Therapy Cures Man's Sickle Cell AnemiaA 21-year-old man has become the first person in New York—and one of the first in the world—to be cured of sickle cell anemia ...
From STAT’s Andrew Joseph: AstraZeneca said this morning it was buying the Belgian cell therapy company EsoBiotec for $425 million upfront, building out another cancer-targeting approach.
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