Expanding partnership aims to advance stem cell therapy to treat RDEB
Inmune working with UK charity to develop CORDStrom for wound healing
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Inmune Bio is expanding its partnership with the U.K.-based charity Anthony Nolan to advance an investigational stem cell therapy — dubbed CORDStrom — that’s primarily being developed to treat the rare skin-related disease RDEB, fully known as recessive dystrophic epidermolysis bullosa.
Under the agreement, Anthony Nolan — which specializes in recruiting and matching potential stem cell donors to patients, while advancing the development of cell and gene therapies and providing tissue banking services — will secure the long-term provision of high-quality umbilical cord tissue for use by Inmune.
That tissue serves as the source of mesenchymal stromal cells (MSCs), which are the specialized stem cells used to manufacture CORDStrom. MSCs can potentially boost wound healing in people with RDEB, according to the partners.
Inmune said the expanded collaboration will leverage the charity’s Cell Therapy and Laboratory Services procurement, screening, and quality management of donor materials to ensure that each CORDStrom product meets rigorous criteria. This will help enhance manufacturing reproducibility while also supporting regulatory compliance, the company stated in a press release announcing the “strategic expansion” of the two sides’ agreement.
“By partnering with Anthony Nolan’s Cell Therapy & Laboratory Services, we are combining the most advanced MSC cell platform with the most reliable source of cellular starting material,” said David Moss, CEO of Inmune.
Epidermolysis bullosa (EB) encompasses a group of genetic diseases marked by fragile skin that blisters and tears easily. One of its subtypes, RDEB, is caused by mutations in the COL7A1 gene that impair the function of collagen VII, a protein that helps hold the skin’s layers together.
People with RDEB can develop chronic wounds and excessive scarring. The disease is also associated with debilitating itch and pain that can worsen wounds and significantly affect quality of life.
CORDStrom uses cells from human umbilical cords
CORDStrom uses MSCs derived from human umbilical cords, which can grow into other cell types to help promote tissue regeneration. This potentially could help in healing skin wounds in people with RDEB.
The therapy is designed to be off-the-shelf, meaning it’s readily available when needed, and suitable for supply in the U.S., the U.K., and the European Union, as well as across other global regions, according to Inmune.
Unlike traditional MSC-based therapies, which can be affected by differences between donors and manufacturing batches, CORDStrom uses MSCs that are tested, screened, and pooled to generate standardized cell banks. Such an approach helps overcome limitations that have historically limited the development of MSC-based therapies, per Inmune.
“We view CORDStrom as a tremendous platform that finally solves the fundamental challenge of understanding MSCs as a drug,” Moss said. The company said it believes CORDStrom could be “a transformative treatment for this devastating skin-fragility disorder.”
CORDStrom was tested against a placebo in a clinical trial called MissionEB (ISRCTN14409785), which involved 30 children and adolescents with intermediate and severe RDEB. The results showed that the therapy significantly reduced disease activity, with participants reporting less pain and itching after treatment.
Immune planning to broad approvals of therapy to treat RDEB
Based primarily on such data, Inmune earlier this year announced plans to seek regulatory approval of CORDStrom in the U.S., the U.K., and the EU to treat RDEB.
The therapy has also been granted rare pediatric disease designation and orphan drug status by the U.S. Food and Drug Administration (FDA) for the treatment of EB. Both designations are designed to incentivize the development of treatments for rare diseases.
Beyond advancing CORDStrom as a potential treatment for RDEB, the partnership will also support the expansion of the MSC platform into additional diseases.
For example, the company is developing CORDStrom-col7a, a candidate therapy designed to address the underlying genetic defects that cause EB. In oncology, the platform is being extended through CORDStrom-TRAIL, an investigational therapy designed to target solid tumors using the platform’s immunomodulatory properties.
Inmune is also exploring applications in inflammatory and degenerative diseases, such as osteoarthritis and systemic lupus erythematosus (SLE), the most common form of lupus.
“This collaboration allows us to move beyond the limitations of traditional cell therapy and provide consistent, high-potency treatments for patients suffering from RDEB, cancer, and chronic inflammatory conditions,” Moss said.
The expanded collaboration with Anthony Nolan is intended to secure long-term access to the donor tissue used to manufacture the therapy.
“This partnership with our Cell Therapy and Laboratory Services allows our donor materials to reach their full potential within a cutting-edge manufacturing platform, facilitating the development of medicinal products that meet the highest ethical and regulatory standards for patients worldwide,” said Nicola Alderson, chief operating officer of Anthony Nolan.
Moss added: “We remain hugely grateful to the volunteer donors who provide the [umbilical cord] for our program and who consent to UK and US testing to make CORDStrom universally available.”
