genetic diseases

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Biotech

Rocket's gene therapy sets new efficacy bar in ph. 1 heart study

While William Blair analysts said the safety data are “highly encouraging,” they noted that the therapy uses the same vector as Sarepta’s Elevidys.
Gabrielle Masson May 16, 2025 10:36am
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Atalanta’s RNA epilepsy asset suppresses seizures in mice

Apr 10, 2025 5:30am
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Gene therapy biotech secures $150M for eye disease candidates

Apr 2, 2025 7:30am
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UPDATE: Another NIH leader is out amid Trump admin shake-up

Mar 21, 2025 10:44am
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Arbor sails into rocky gene editing waters with $74M

Mar 18, 2025 6:30am
Gene editing CRISPR gene therapy

Beam gene therapy passes early safety test in AATD

Mar 10, 2025 12:22pm