Two complete response letters for group members dispatched this month by the FDA was enough to contribute to a flat performance in October for the BioWorld Neurological Diseases index. Overall, the price-weighted index has remained underwater for most of the year with its value down about 5% during this period.

Intellia Therapeutics Inc. is looking to disrupt the transthyretin (TTR) amyloidosis (ATTR) market with NTLA-2001, its CRISPR-based treatment designed to be a potential cure for the disease. The drug, which is delivered via a lipid nanoparticle, edits the patient's DNA in vivo to create a stop codon and eliminate the expression of TTR, the protein that aggregates in ATTR patients' nervous systems and hearts, disrupting their functions.

The pace at which companies are integrating the sophisticated tools of artificial intelligence (AI) and machine learning (ML) into their early stage drug discovery and development programs is accelerating, as evidenced by the creation of new dedicated AI-focused biopharma companies and the eagerness of drug developers to partner with AI firms. A panel at the BIO Investment Forum delved into the impact of AI on the sector and its ability to assist scientists in uncovering the intricacies of disease mechanisms and lead them toward novel drug targets.