New data show that a high dose of Intellia’s in vivo CRISPR-Cas9 based therapy is safe and effective for up to six months, which could unlock a new era in genetic disease treatment.
Intellia looks to have a potentially curative ATTR amyloidosis gene therapy on its hands, but must replicate the early results in a pivotal study. • Source: Alamy
Updated results from Intellia Therapeutics and Regeneron’s CRISPR Cas9-based in vivo gene-editing candidate NTLA-2001 have boosted confidence that it could be a major breakthrough in treating genetic diseases.
NTLA-2001 is the first CRISPR-based therapy candidate to be administered systemically to patients to permanently edit out a disease-causing gene...
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The Swiss pharma company is rebalancing the portfolio, emphasizing best-in-class drugs and moving into new areas like cardiovascular and metabolic disease.
In this week's episode: Trump’s UK drug pricing win; Novo explains semaglutide Alzheimer's rationale; J&J and Novartis on this year’s biggest M&A deals; a look at the mixed performance of CAR-Ts; and Akeso’s goals beyond ivonescimab.
Pipelines in bioconjugates continue to develop, from ADCs to XDCs, with radionuclide drug conjugates (RDCs) and antibody-oligonucleotide conjugates (AOCs) leading the expansion.
Lilly's first Phase III triplet win opens the door to a label expansion in BTK-pretreated CLL, with analysts suggesting Jaypica could attain blockbuster status. However, the window to capitalize may be brief as next-generation BTK degraders close in with head-to-head trials.