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 ...

Gene regulation refers to the mechanisms that act to induce or repress the expression of a gene. These include structural and chemical changes to the genetic material, binding of proteins to ...

The company raised $200 million after early findings hinted its therapy could be more potent than others like it, including Sarepta Therapeutics’ Elevidys.

After hours: February 21 at 4:53:49 PM EST Loading Chart for ALNY ...

Gene expression is the generation of a functional gene product from the information encoded by a gene, through the processes of transcription and translation. Gene products are often proteins ...

The FDA has approved Alnylam’s gene silencing drug Oxlumo ... Alnylam’s Givlaari, FDA-approved a year ago for acute hepatic porphyria, costs $575,000 per year at full price in the US, although ...

Danish drugmaker Novo Nordisk must like what it has seen in its two-year-old alliance with gene-silencing specialist ... for acute hepatic porphyria (AHP) and Oxlumo (lumasiran) for primary ...

Ongoing launch of CASGEVY ® continues to gain momentum; new cell patient collection initiations expected to significantly grow in 2025- -More than 50 authorized treatment centers (ATCs) activated ...

Feb. 7, 2025 — A research team has made a major discovery on how the CUL5 gene affects CAR-T cell therapy, an advanced cancer therapy. The study reveals that reducing the activity of the CUL5 ...

Citi analysts adjusted their outlook on CRISPR Therapeutics (NASDAQ:CRSP) stock, reducing the price target to $82 from $89 while maintaining a Buy rating. The stock, which has shown significant ...