Vertex Pharmaceuticals is developing a cell therapy for type 1 diabetes to replace insulin-producing cells lost to chronic disease.$320 million paid Acquisition of ViaCyteVertex is approaching the first company to demonstrate this feat in clinical testing.
Boston-based Vertex complemented its own research efforts with the deal announced Monday. Privately held ViaCyte brings additional tools, technology and assets, Vertex CEO Reshma Kewalramani said in a prepared statement.
In type 1 diabetes, the immune system destroys the insulin-producing islet cells in the pancreas. Patients require regular doses of insulin, either by injection or from an insulin pump, to maintain levels of glucose-regulating hormones.
Vertex’s cell therapy candidate for type 1 diabetes, VX-880, consists of pancreatic islet cells grown in the laboratory. These cells are infused into patients along with immunosuppressive therapy to prevent the immune system from rejecting them. At last month’s American Diabetes Association annual meeting, Vertex presented data from two patients who received half the target dose of cells. Blood glucose changes were seen in both patients. One patient achieved insulin dependence at 270 days after dosing, while another patient had a 30% reduction in insulin infusions or injections at day 150.
Vertex adds VX-880 to its pipeline in 2019, buys startup Semma Therapeutics for $950M upfront. At the time of the transaction, Semma was in the preclinical stage. The ongoing Phase 1/2 study aims to enroll 17 patients.it is allowed to resume A resolution passed last week clinical hold Attended the study in May; the FDA said at the time that there was insufficient information to support a dose increase.
San Diego-based ViaCyte is developing three programs targeting type 1 diabetes. The most advanced is VC-02, a stem cell-derived islet cell packaged in an implantable pouch. The pouch is connected to the patient’s blood vessels, allowing them to interact with the transplanted cells. ViaCyte, June 2021 Nine-month clinical data showed that the transplanted cells produced insulin in the first patient treated with this approach.
Like Vertex therapy, VC-02 requires immunosuppressive drugs to prevent rejection. The second ViaCyte program, VC-01, can reduce this risk by designing a pouch that prevents the patient’s immune cells from coming into direct contact with the transplanted islet cells. ViaCyte presented additional data for both programs at the American Diabetes Association meeting in June. According to William Blair analyst Raju Prasad, these results are important for showing the potential of cell therapy to treat type 1 diabetes.
“Taken together, the data demonstrate for the first time in humans that engrafted pancreatic progenitor cells can functionally differentiate into mature, glucose-responsive cells,” Prasad wrote in a note to investors Monday morning. , (endogenous) insulin-producing islet cells.”
Vertex’s main interest in ViaCyte may be its third project, which could eliminate the need for immunosuppressive therapy. The cells in this experimental therapy, VCTX210, were edited using CRISPR technology to evade detection by the immune system. ViaCyte is developing the VCTX210 Partnering with CRISPR Therapeutics. The Switzerland-based gene-editing company is already a Vertex partner.The two companies are Collaborative Gene Editing Therapy for Two Blood Disorders, Sickle Cell Disease, Sickle Cell Disease and Beta ThalassemiaThe collaboration also extends to two muscle diseases, Duchenne muscular dystrophy and type 1 myotonic dystrophy.
Prasad sees synergy between Vertex and ViaCyte’s approach to type 1 diabetes due to the similarities in technology, development and manufacturing of the two cell therapies. The similarities, he said, would benefit CRISPR Therapeutics and Vertex in developing a CRISPR-edited therapy that reduces the risk of triggering an immune response.
However, the acquisition of ViaCyte is still subject to antitrust review. The FTC has raised concerns about the anticompetitive effects of some pharmaceutical mergers, a topic that was discussed at a recent meeting. virtual workshop last month. There are other companies developing innovative treatments for type 1 diabetes. Beta Bionics have also been clinically tested with medical devices Designed to take on the role of the patient’s pancreas.One Provention Bio’s drug candidates Currently under FDA review. Possibly the closest technology to ViaCyte is the cell bag being developed by Sernova in Canada. Designed to hold therapeutic cells, the bag could provide an alternative to daily dosing and is currently in early clinical development for type 1 diabetes.
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