Manufacturing process now in gear for producing treatment for fragile skin

A U.S.-based biotech company announced that it has reached a “significant” milestone in advancing its experimental stem cell treatment for recessive dystrophic epidermolysis bullosa (RDEB), a rare condition marked by extremely fragile skin.

Inmune Bio said it has readied its process for commercial-grade manufacturing of CORDStrom, the stem cell platform it’s using to develop the RDEB treatment. The CORDStrom platform uses mesenchymal stem cells — a type of stem cell that’s able to grow into certain types of connective tissue — that are derived from donated umbilical cord tissue.

With any therapy made using human cells, there are strict regulatory frameworks to ensure that the treatment can be produced safely and consistently, without the risk of transmitting diseases from tissue donors to therapy recipients. In a company press release, Inmune said its process “is now commercial-ready,” and will meet manufacturing standards in the U.S., the European Union, and the U.K.

“Everyone involved in the CORDStrom platform understands the urgency and criticality of developing potentially life-changing medicines [for] children living with RDEB and other rare diseases,” said Ben Weil, head of U.K. operations at Inmune. “Building a reliable, scalable, and commercial ready manufacturing platform is only possible through the commitment and expertise of all our suppliers and service partners.”

Recommended Reading

Sept. 8, 2023 News by Marisa Wexler, MS

Itch is common in RDEB; available treatments don’t help much

According to Inmune, the first batch of commercial-ready umbilical cord tissue has been processed at a manufacturing center in the U.K.

Developer to soon seek regulatory approval of RDEB treatment

The company said it now has a supply of therapeutic cells that meet regulatory standards in the U.S. and Europe. Inmune also recently received positive feedback from the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) to guide further development, per the release.

David Moss, CEO and cofounder of Inmune, noted that “commercial manufacturing is often one of the largest execution risks facing cell therapy companies.”

Moss said this “milestone demonstrates that we have successfully transitioned from clinical manufacturing toward a scalable commercial process that supports our planned regulatory filings and long-term commercialization strategy. Combined with our recent regulatory alignment from the MHRA and the evolving UK rare disease framework, we believe CORDStrom is exceptionally well positioned for the next stage of development.”

“We now have the manufacturing infrastructure required [for] … future commercial production using a process designed to satisfy both U.K. and U.S. regulatory expectations.

A genetic disorder, RDEB is marked by abnormally fragile skin that’s prone to blisters and wounds that don’t heal easily. Using its stem cell platform, Inmune is developing a cell therapy, dubbed Ebstrocel, that aims to help facilitate better wound healing.

In a Phase 1 study called MissionEB (ISRCTN14409785), treatment with this cell therapy was shown to ease pain and itching in children with RDEB.

Based on the promising early data, Inmune has announced plans to seek regulatory approval of its RDEB cell therapy in the U.S., the U.K., and the EU. All three applications are expected later this year, the company said. Inmune is also planning to launch a Phase 3 clinical trial further testing the therapy.

The new manufacturing supply aims to support a potential commercial launch and the upcoming Phase 3 trial, per the company.

“We now have the manufacturing infrastructure required to generate master cell banks that support our planned Phase 3 program and future commercial production using a process designed to satisfy both U.K. and U.S. regulatory expectations,” said Mark W. Lowdell, PhD, Inmune’s chief scientific officer. “I am hugely proud of the team for getting to this milestone so quickly.