Vertex Pharmaceuticals Seeks Solutions to Cure T1D

Dr. Fredrick Banting himself said, “Insulin is not a cure,” and while T1D technology is advancing at lightning-bolt speed, people still struggle to afford insulin, and keeping glucose levels in check is an arduous twenty-four-hour job even with all of the fabulous looping devices available.

The Juvenile Diabetes Cure Alliance defines a T1D practical cure as any solution that delivers a near-normal lifestyle for people living with established type 1 diabetes. This includes low A1Cs under 7%, non-invasive wearable devices with minimal monitoring, no hypo episodes, and an unrestricted diet.

Fortunately, Vertex Pharmaceuticals, a global biotech corporation based in Boston, Massachusetts, with international headquarters in London, United Kingdom, is developing cell therapies to treat and cure type 1 diabetes. The biopharmaceutical company has research and development sites worldwide.

About Vertex Pharmaceuticals

Founded in 1989, Vertex’s mission is to invest in scientific innovation to create transformative medicines for people with serious diseases. In addition to T1D, these disorders include cystic fibrosis (CF), sickle cell disease, and HIV/AIDS.

Vertex scientists saw the need to research investigational stem cell therapies to replace the insulin-producing beta cells (the cells lost in T1D). These researchers are dedicated to developing a cell therapy that may potentially replace the destroyed insulin-producing cells in people with T1D.

Their investigational approach is to produce billions of insulin-producing cells in the laboratory and use various methods to transfer them to patients.

They are studying two ways to do this:

1. ‍The patient receives an infusion of insulin-producing cells and, at the same time, immunosuppressant drugs to stop the body from attacking the cells.
2. ‍The cells are packaged in a small, protected device implanted into the patient during a surgical procedure.

The goal in both cases is for the cells to produce insulin independently, automatically, and on demand—normalizing blood sugar and replicating what the cells in the pancreas should do.

Here are a few promising Vertex innovations on the horizon.

Vertex Mergers and Clinical T1D Trials

VX-880

Vertex’s VX-880 therapy is designed to help patients avoid serious blood glucose levels and maintain A1Cs below 7%. In the Phase 1 / 2 study of VX-880, this insulin-producing islet cell therapy is used with standard immunosuppression medicine.

The efficacy as of the last data cut was that all 14 patients dosed with VX-880 therapy demonstrated islet cell engraftment and production of endogenous insulin. All Part A and B patients, except for one who withdrew consent, demonstrated glycemic control with ADA-recommended levels (A1C <7%) and no longer required insulin.

Most importantly, this is the first laboratory-developed stem cell that has proven effective in humans. And even though this trial requires patients to take immunosuppressant drugs to stop the body from attacking the insulin-producing beta cells, it’s still in very promising stages.

This investigational stem-cell-derived therapy could save the lives of individuals with hypoglycemic unawareness and severe hypoglycemia. Vertex presented results at the American Diabetes Association’s 83^rd Scientific Session. Positive results in future trials are happening in the US and internationally with similar outcomes.

VX-264

VX-264 is the investigational approach mentioned above, where the patient receives encapsulated cells in a protective device to be surgically implanted in the body. This procedure is gauged without using immunosuppressive therapy as the devices shield the cells from the body’s immune system attack.

What’s more exciting is that Vertex received FDA clearance for the new drug application for VX-264. The novel encapsulated cell therapy is designed and approved for treatment of type 1 diabetes individuals. This innovative encapsulation device promises to protect implanted cells from attack.

To learn more about the studies, interested parties can contact Vertex’s medical team at medicalinfo@vrtx.com or call 617-341-6777.

Semma Therapeutics

Before the discoveries mentioned earlier, in 2019, the company purchased Semma Therapeutics, the world’s most advanced stem cell company. This acquisition marked Vertex’s goal of developing cell-based treatments for T1D. Semma had already created a “small, implantable device that holds millions of replacement beta cells, letting glucose and insulin through but keeping immune cells out.”

ViaCyte

Another important merger of Vertex involved the biotech partner ViaCyte. In July 2022, Vertex purchased ViaCyte for $320 million and, in turn, eliminated its main competition. Taking this major player out of the game, Vertex gained rights to PEC-Direct, an implantable device infused with progenitor cells, developed inside the body, and PEC-Encap, a device in which the developed cells are protected from immune rejection by a semi-permeable membrane.

“VX-880 has successfully demonstrated clinical proof of concept in T1D, and the acquisition of ViaCyte will accelerate our goal of transforming, if not curing, T1D by expanding our capabilities and bringing additional tools, technologies and assets to our current stem cell-based programs,” said Reshma Kewalramani, MD, Chief Executive Officer, and President of Vertex.

CRISPR Therapeutics

Vertex and CRISPR Therapeutics have worked together to treat sickle cell disease. The new collaboration involved developing treatments for T1D. Last March 2023, Vertex announced a $100 million agreement with CRISPR to develop cell therapies that produce insulin without the autoimmune triggering effect.

CRISPR has an investigational therapy, CTX211, for T1D, in which the treatment uses gene edits to enhance cell fitness, allowing patients to produce their insulin in response to glucose. This new arrangement allows Vertex to use CRISPR Therapeutics’ therapy that focuses on gene-editing technology, working to evade the immune system, another treatment with built-in protection from autoimmune attacks.

Vertex-Lonza Manufacturing Facility for T1D Cell Therapies

Finally, another impressive bit of Vertex news in 2023 was the deal with Lonza Bioscience, a global pharmaceutical biotech partner, to build a stem cell supply factory. The endeavor hopes to optimize an ample supply of stem cells at a commercial level. This powerhouse initiative to produce insulin-producing cells began construction late last year and will be solely for developing cells tested in Vertex’s VX-264 and VX-880 clinical trials.

Operated by Lonza, the manufacturing building is expected to span over 130,000 square feet upon completion. It is located in Portsmouth, New Hampshire, on Lonza’s preexisting campus. The facility is anticipated to create up to 300 new jobs.

“Vertex’s allogeneic, fully differentiated, insulin-producing islet cells and the results from the Phase 1/2 VX-880 program represent a scientific breakthrough that offers the potential to transform the treatment of T1D,” said Reshma Kewalramani, MD, Chief Executive Officer and President of Vertex. “Establishing this strategic partnership with Lonza, a world-class manufacturing organization, is a critical milestone in this journey and underscores our long-term commitment to patients with   T1D.”