Following a strategic transaction with Graphite Bio Inc., Kamau Therapeutics is emerging from stealth with sickle cell treatment nulabeglogene autogedtemcel (nula-cel). Kamau received an option to acquire all of Graphite’s genome editing assets, including a platform technology that integrates precision DNA repair using homology directed repair and CRISPR/Cas9, as well as the autologous CRISPR/Cas9 gene corrected CD34+ cell product nula-cel, which offers a potential cure for sickle cell disease derived from the patient's cells.

Injecting a combination of cisplatin and vinblastine – engineered by way of the company’s Dfuserx platform – turned out to be just the ticket for early stage breast cancer in the phase II study called Invincible conducted by Intensity Therapeutics Inc. Shares of the Shelton, Conn.-based firm (NASDAQ:INTS) closed Dec. 8 at $6.88, up $2.68, or 63%, having traded as high as $11.44, thanks to Wall Street’s satisfaction with the data testing INT230-6, which emerged during the San Antonio Breast Cancer Symposium.

The COVID-19 pandemic took a huge bite out of the U.S. FDA’s ability to conduct inspections in a timely manner, but the FDA’s Douglas Stearn said the agency has nonetheless ramped up these activities.

Researchers have defined an amyotrophic lateral sclerosis (ALS) reversal phenotype as having an initial diagnosis of ALS but subsequently showing a progressive and sustained clinical improvement, based on an unusual case they found.

At the recent American College of Neuropsychopharmacology meeting, Trevena Inc. presented preclinical data for the novel selective sphingosine 1-phosphate S1P1 receptor modulator TRV-045, being developed for the treatment of chemotherapy-induced peripheral neuropathy (CIPN).

While early stage and involving a relatively small patient population, the interim phase Ib readout from the combination cohort testing estrogen receptor (ER)-targeting candidate vepdegestrant in combination with CDK4/6 inhibitor Ibrance (palbociclib) in heavily pretreated patients with ER-positive/HER2-negative breast cancer was impressive enough to prompt partners Arvinas Inc. and Pfizer Inc. to expand development work on the program. The results also struck a chord on the Street, with shares of Arvinas (NASDAQ:ARVN) gaining 31% on the day.