Week of July 11, 2022

This week we highlight innovative treatments for heart disease and depression. We also look at a decision by the Food and Drug Administration to allow pharmacists to prescribe a COVID-19 drug, as well as the use of artificial intelligence in the fight against ALS. Finally, we touch on Vertex’s acquisition of a competitor in the diabetes space.

Verve Therapeutics, a Cambridge, Mass., biotech company, has commenced a clinical trial using a gene editing technology, called CRISPR, to treat heart disease. This is the first time such a treatment has been administered in a human trial. The company hopes to enroll up to 40 patients in this phase 1 trial and expects to report results in 2023.

The FDA granted breakthrough device designation for an Abbot product used to combat treatment-resistant depression. Abbott has had previous success using this Deep Brain Stimulation (DBS) system to treat Parkinson’s disease but has recently identified that this system can implant electrodes in the brain that can be used to regulate moods, reducing depression symptoms.

A recent decision by the FDA allows pharmacists to prescribe Paxlovid, Pfizer’s oral anti-viral drug, to individuals exhibiting COVID-19 symptoms. The decision is aimed at increasing accessibility of the drug.  Overall demand for the therapeutic has been significantly lower than expected since its initial release.  The American Medical Association opposes the FDA decision, stating that the drug should be prescribed by physicians with knowledge of patients’ medical history and the ability to monitor progress and address potential side-effects.

Insilico Medicine used a proprietary AI platform, PandaOmics, to identify 28 potential targets within the central nervous system that could lead to progress on treatment of amyotrophic lateral sclerosis (ALS). Insilico, an AI-focused biotech company, is collaborating on ALS research with Answer ALS, a global research project that is compiling patient data. Insilico intends to pursue these targets, 17 of which are deemed high confidence, with the goal of advancing to human trials.

Vertex, which has been developing stem-cell based diabetes treatments, acquired competitor ViaCyte for $320 million. ViaCyte has also made progress in the development of a stem-cell based diabetes therapeutic. ViaCyte’s treatment converts stem cells into pancreatic cells which monitor glucose levels once they are implanted into patients.

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