Brett Kopelan, MA, on the Future Treatment of Epidermolysis Bullosa With Advanced Therapeutics

“I think when we are discussing being able to treat this disease effectively, we really need to look at a holistic approach to it... I can see in the near future where a patient is actually using more than one cell and gene therapy.”

Epidermolysis bullosa (EB), a collection of rare genetic connective tissue disorders, remains highly difficult to manage for patients: until very recently, treatment for these disorders was mostly limited to palliative care. Fortunately, this is already changing, with the recent approval of Krystal Biotech’s beremagene geperpavec (B-VEC, marketed as Vyjuvek), a topical and redosable gene therapy for dystrophic epidermolysis bullosa (DEB), in May 2023, and a number of still-investigational advanced therapies on the near-term horizon.¹

In observance of EB Awareness Week, which is held each year from October 25 to 31, CGTLive™ spoke with Brett Kopelan, MA, the executive director of the Dystrophic Epidermolysis Bullosa Research Association (debra) of America, about the rapidly changing landscape of care for EB and the options that may become available for patients with EB in the future. Kopelan spoke about Abeona Therapeutics’ EB-101, an investigational autologous cell therapy intended to treat recessive dystrophic epidermolysis bullosa (RDEB) and Castle Creek Biosciences’ dabocemagene autoficel (FCX-007, D-Fi), an investigational autologous ex vivo fibroblast therapeutic agent delivered by intradermal injections. Notably, EB-101 currently has a biologics license application under review by the FDA and FCX-007 is currently being evaluated in a phase 3 clinical trial (NCT04213261).^2,3 

Kopelan also mentioned that debra is working with a company in Germany on a systemic cell therapy approach to treating EB and that outside of advanced therapeutics there are several promising topical approaches to EB currently in development.

He additionally pointed out that some of these products may be better at treating large EB wounds and that others may be better filling in the gaps. Ultimately, he concluded that in the future, a combination of approaches is most likely to be successful in treating EB, rather than relying on a single therapeutic agent. As such, the continued development of novel treatments for EB is of great importance for the patient community.

REFERENCES
1. Krystal Biotech receives FDA approval for the first-ever redosable gene therapy, VYJUVEK™ (beremagene geperpavec-svdt) for the treatment of dystrophic epidermolysis bullosa. News release. Krystal Biotech. May 19, 2023. Accessed October 25, 2023. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-receives-fda-approval-first-ever-redosable-gene
2. Abeona Therapeutics submits biologics license application to U.S. FDA seeking priority review and approval of EB-101 for the treatment of patients with Recessive Dystrophic Epidermolysis Bullosa. News release. Abeona Therapeutics. September 26, 2023. https://investors.abeonatherapeutics.com/press-releases/detail/265/abeona-therapeutics-submits-biologics-license-application
3. Castle Creek Biosciences acquiresNovavita Thera to expand innovative cell and gene therapy platform. News release. Castle Creek Biosciences. January 10, 2022. https://castlecreekbio.com/castle-creek-biosciences-acquires-novavita-thera-to-expand-innovative-cell-and-gene-therapy-platform/