Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics AG (Nasdaq: CRSP) have announced positive results from pivotal trials for their investigational treatment, exagamglogene autotemcel (exa-cel), in patients with transfusion-dependent beta thalassemia (TDT) or severe sickle cell disease (SCD). 

Both trials, CLIMB-111 and CLIMB-121, met their primary and key secondary endpoints at pre-specified interim analyses, demonstrating the efficacy and safety of exa-cel. 

The companies also revealed yesterday that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Applications (BLAs) for exa-cel in SCD and TDT, with priority review granted for SCD and standard review for TDT. The FDA has set target action dates for December 8, 2023, and March 30, 2024, respectively. 

Exa-cel is a potential gene-edited cell therapy that aims to alleviate symptoms and reduce the need for transfusions in patients with SCD and TDT.

In the pre-market and following the announcement, $VRTX is trading at $336.06, up $3.48 (+1.05%), while $CRSP is trading at $66.47, reflecting an increase of $3.88 (+6.20%).

In another development, Sonoma Pharmaceuticals, Inc. (Nasdaq: SNOA) has announced a new application for intraoperative pulse lavage irrigation treatment, offering a safe and effective alternative to commonly used IV bags in various surgical procedures. 

The company's pulse lavage container, used with a pulse lavage irrigation device, replaces non-antimicrobial saline and aggressive rinsing solutions with Microcyn Technology, which reduces microorganisms and promotes wound healing. 

This solution is cost-competitive with IV bags and is expected to be commercially available in Europe by September 2023, followed by a launch in the U.S. in 2024.

$SNOA is trading at $1.28, up $0.31 (+31.96%) in pre-market.

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