Gene therapy has had its commercial struggles in the past year. The cost to patients is in the millions and fewer are stepping forward for treatment than companies would like. While development continues in this game-changing field, some have struggled with regulatory authorities during development while others have just stepped away altogether.
Only a couple of years since the first sickle cell disease (SCD) gene therapies gained U.S. FDA approval, researchers are working to expand access for younger children, and to improve manufacturing and commercialization to reach patients faster.
The BioWorld Biopharmaceutical Index extended its rebound through August and September, closing the third quarter (Q3) slightly in positive territory after losing ground earlier in the year.
Enlaza Therapeutics Inc. will take charge of research through nominating candidates in its potentially $2 billion-plus deal with Vertex Pharmaceuticals Inc. For its efforts, Enlaza is getting $45 million in an up-front payment and equity investment, plus the opportunity to bring in more than $2 billion in research, development, regulatory and commercial milestones and tiered royalties on net sales.
Enlaza Therapeutics Inc. will take charge of research through nominating candidates in its potentially $2 billion-plus deal with Vertex Pharmaceuticals Inc. For its efforts, Enlaza is getting $45 million in an up-front payment and equity investment, plus the opportunity to bring in more than $2 billion in research, development, regulatory and commercial milestones and tiered royalties on net sales.
Vertex Pharmaceuticals Inc.’s bad news from a phase II pain study and separately on the regulatory front caused shares (NASDAQ:VRTX) to close Aug. 5 at $393.83, down $78.44, or 16%. Vertex rolled out top-line results from the phase II dose-ranging study to test the safety and efficacy of its NaV1.8 pain signal inhibitor, VX-993, in tackling acute pain after bunionectomy.
A total of 33 states, plus the District of Columbia and Puerto Rico, agreed to participate in the U.S. CMS’ voluntary, outcomes-based program aimed at helping state Medicaid programs cover high-priced cell and gene therapies, starting with therapies for sickle cell disease.
Vertex Pharmaceuticals Inc. has described polycystin-1 (PKD1) (mutant) correctors reported to be useful for the treatment of autosomal dominant polycystic kidney disease.
Vertex Pharmaceuticals Inc. has synthesized sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain.
The BioWorld Biopharmaceutical Index (BBI) ended 2024 down 2.24%, despite hitting a peak of 25.19% in late August. While it remained in positive territory through November, up 3.58%, the steady decline through the later months of the year pushed the index into the red by December’s close.
