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Long Island has a ‘high burden’ for a common blood disorder: new study
It's caused by a mutation in the gene responsible for making hemoglobin, a protein in red blood cells.
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LEO Pharma to acquire gene therapy company Replay
LEO Pharma has signed a definitive agreement for the acquisition of Replay, which focuses on treating rare genetic dermatological conditions.
2026 Kentucky Derby picks, odds, predictions, favorites: Best bets by top expert who had $1,045 win last year
Gene Menez revealed his Kentucky Derby 2026 picks for the Run for the Roses on Saturday, May 2 at Churchill …
2026 Kentucky Derby picks, odds, predictions, favorites: Best bets via top expert who had $1,045 win last year
Gene Menez revealed his Kentucky Derby 2026 picks for the Run for the Roses on Saturday, May 2 at Churchill …
2026 Kentucky Derby picks, odds, predictions, favorites: Best bets via expert who had $1,045 win last year
Gene Menez revealed his Kentucky Derby 2026 picks for the Run for the Roses on Saturday, May 2 at Churchill …
Ray Therapeutics receives EMA PRIME status for gene therapy
Ray Therapeutics has received PRIME designation from the EMA for its RTx-015 gene therapy to treat retinitis pigmentosa (RP).
2026 Kentucky Derby picks, odds, predictions, favorites: Best bets by expert who had $1,045 win last year
Gene Menez revealed his Kentucky Derby 2026 picks for the Run for the Roses on Saturday, May 2 at Churchill …
2026 Kentucky Derby picks, odds, predictions, favorites: Best bets from expert who had $1,045 win last year
Gene Menez revealed his Kentucky Derby 2026 picks for the Run for the Roses on Saturday, May 2 at Churchill …
2026 Kentucky Derby picks, odds, predictions, favorites: Best bets from expert who had $1,045 payout last year
Gene Menez revealed his Kentucky Derby 2026 picks for the Run for the Roses on Saturday, May 2 at Churchill …
Rocket to sell PRV for $180m to advance gene therapy pipeline
Rocket Pharmaceuticals has signed an agreement to sell its rare paediatric disease PRV for $180m to advance its gene therapy …
Eli Lilly bets on recombinases in $2.25bn Profluent R&D pact
Eli Lilly and Profluent will harness AI to discover and develop recombinases for precise, large-scale gene editing.
Rocket sells priority review voucher for $180M after Kresladi gene therapy approval
Rocket Pharmaceuticals has offloaded its lucrative FDA rare pediatric disease priority review voucher, striking a $180 million sale that reflects …
2026 Kentucky Derby picks, odds, predictions, favorites: Best bets by expert who had $1,045 payout last year
Gene Menez revealed his Kentucky Derby 2026 picks for the Run for the Roses on Saturday, May 2 at Churchill …
Milestone for CRISPR: First-of-Its-Kind Gene Editing Treatment Successfully Passes Clinical Trial
A biotech company is looking to get approval next after their recent discovery.
STAT+: Eli Lilly enlists AI startup for next-generation gene editors
Eli Lilly's deal with Profluent aims to go beyond CRISPR by using AI-designed enzymes to insert entire genes. It could …
Intellia’s Data Reveal Tees Up FDA Filing for CRISPR-Based In Vivo Gene-Editing Med
Intellia Therapeutics’ lonvo-z uses CRISPR to inactivate a gene in the liver to reduce levels of a protein key to …
Intellia’s in vivo gene therapy victorious in Phase III HAE trial
Intellia’s CEO said that the trial marks the first positive Phase III readout for an in vivo CRISPR gene therapy. …
STAT+: Astellas retries XLMTM gene therapy after deaths
Veradermics' hair loss pill late-stage trial success, FDA fast-tracks reviews for psychedelics, and more biotech news
STAT+: Pharmalittle: We’re reading about a Lilly deal, an Intellia CRISPR rare-disease treatment, and more
Intellia disclosed that a single dose of its gene editing treatment dramatically reduced swelling attacks in patients with hereditary angioedema
FDA Approves First-Ever Gene Therapy for Treatment of Genetic Hearing Loss
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