Gene, Cell Therapies and Advanced Rare Disease Talk at AAD 2024

More than 700 rare genetic dermatoses are registered by national medical organizations, Joyce Teng, MD, PhD, told HCPLive. Though there a multitude of integral differences in characteristics, symptoms and mechanisms among each, a consistent notion is endorsed by researchers: a breakthrough in even just one may help to benefit them all.

In the second segment of an interview during the Society for Pediatric Dermatology (SPD) 2024 Pre-AAD Meeting in San Diego, CA, this week, Teng, professor of dermatology at Stanford University, continued her discussion on recently developed treatments for dystrophic epidermolysis bullosa (DEB). As she explained, the disease’s recently burgeoning treatment space—replete with a new gene therapy, natural topic treatment, and an investigative cell graft procedure—is emblematic of the opportunities to recycle and repurpose innovations across rare dermatoses.

Teng looks forward to that sentiment being reflected in sessions at AAD 2024.

“(We can use) some of these agents, from anti-inflammatory indicated for other diseases to the genetic space, to help with managing care of these challenging patients,” she said. “And so, in genetic diseases, there are going to be a lot of exciting sessions that are going to be presented...as well as upcoming trials for other diseases, and really encapsulate what I just described.”

Specifically in DEB, Teng is hoping discourse at AAD 2024 and beyond considers utilizing more familiar agents complementary to the new therapies on the market.

“For instance, even though we have a gene therapy for (DEB), patients that have wound healing are terribly itchy, so much so that they injure themselves,” Teng said. “And that diminishes some of the efficacy of the treatment that we're using. So can we use additional anti-itch therapy, for instance, to control and regulate those self-injury behaviors and modify skin inflammation, so we can optimize outcomes?”

Teng also expressed interest in learning more about better skin barrier reinforcement strategies for patients with DEB, and whether such an emphasis has any benefit for treatment outcomes with beremagene geperpavec (B-VEC; VYJUVEK), prademagene zamikeracel (pz-cel), or the like.

“I am looking for a lot of the new information coming out, (including) new imaging modalities, and how even in the genetic skin diseases space: how do we track our clinical improvement and modulate wound closure, for instance?” Teng said. “And how do we assess a patient's improvement from these exciting advanced therapies that we developed is another really hot topic.”