CGTLive's Pillars of Progress 2023: Top News in Dermatology


Take a look at the stories that stood out as pillars of progress and success in dermatology gene and cell therapy development in 2023.

For all of 2023, the CGTLive team was following along the clinical development of targeted and novel engineered approaches to the treatment of patients with various medical disorders. These efforts included holding in-depth conversations with experts in the clinical care of these individuals, as well as in cell and gene therapy development, culminating in our coverage of each step of progress that the most exciting cellular and genetic treatments have made along the pipeline.

From major data publications and presentations to FDA decisions and major medical meetings, the team spent all year bringing the latest information to the website's front page.

Among our areas of focus in 2023 has been dermatology, a field that saw a key approval, as well as other treatments move through the pipeline. The major news items appeared among the top pieces our team produced—but sometimes smaller stories reach those heights as well because of their clinical impact, their inventive mechanisms, or otherwise. Whatever the reason for the attention these stories got, their place here helps provide an understanding of the themes in dermatology over the course of 2023.

Here, we'll highlight some of the most-read content on CGTLive's dermatology page this year. Click the buttons to read further into these stories.

1. Expert Clinician Reactions to FDA Approval of B-VEC for Dystrophic Epidermolysis Bullosa

  • Vyjuvek (B-VEC), a gene therapy by Krystal Biotech, has been FDA approved for dystrophic epidermolysis bullosa.
  • Vyjuvek's approval brings a corrective therapy for patients, impacting local care and benefiting pediatricians and dermatologists.
  • Vyjuvek uses a novel HSV-1 vector and is the first redosable gene therapy, marking a huge milestone in gene therapy reserach.

May 22, 2023 — After many years without much in terms of therapeutic options, patients with dystrophic epidermolysis bullosa (DEB) were given a new option for treatment with the FDA’s decision to approve Krystal Biotech’s beremagene geperpavec (Vyjuvek), also known as B-VEC, a topical and redosable gene therapy. The gene therapy, the first for this patient population, is indicated for patients 6 months or older.

"I think many more local primary care pediatricians and dermatologists will start to treat epidermolysis bullosa patients now that they have an easy corrective therapy they can prescribe. This will be a benefit for epidermolysis bullosa patients as well.
– M. Peter Marinkovich, MD, of Stanford

2. The Future of Dermatology? B-VEC's Impact in DEB 6 Months Post Approval

  • B-VEC gene therapy, approved by the FDA in May 2023, offers a topical and redosable treatment for DEB, a significant advancement in dermatology.
  • Clinical data from the GEM-3 study shows B-VEC's efficacy with a 67% wound healing rate at 6 months and reduced pain during dressing changes, improving the quality of life for DEB patients.
  • With B-VEC's ease of application and positive outcomes, dermatology is transitioning toward integrating gene therapy into clinical practice, offering new possibilities for treating various skin conditions.

November 3, 2023 — In a landmark event in May 2023, the FDA approved Krystal Biotech’s beremagene geperpavec (B-VEC) under the name Vyjuvek for the treatment of dystrophic epidermolysis bullosa (DEB), making it the first approved gene therapy in the dermatology field. As we reach 6 months out from the therapy’s approval, CGTLive spoke to experts in the field about their experiences and impressions of B-VEC as well as future questions and research needs that remain.

"[We need more time] before we can truly see the full impact [but] we anticipate it to be incredibly important. Some of the people that have been on this therapy for a number of years have really seen a huge positive impact on the quality of life."
-Brett Kopelan, MA. of debra of America

3. Krystal's Dystrophic Epidermolysis Bullosa Gene Therapy B-VEC Under Review in EU

  • Krystal Biotech's gene therapy, Vyjuvek, for dystrophic epidermolysis bullosa (DEB), takes another step forward with the validation of its marketing authorization application by the European Medicines Agency.
  • The application's validation follows previous designations, including orphan drug and priority medicines, positioning Vyjuvek for potential approval in the European Union.
  • Vyjuvek, already FDA-approved in the US, is a redosable gene therapy offering a corrective treatment for DEB patients, addressing an unmet medical need.

November 27, 2023 — Krystal Biotech’s marketing authorization application (MAA) for beremagene geperpavec (B-VEC, marketed as Vyjuvek in the United States), an investigational gene therapy intended to treat dystrophic epidermolysis bullosa (DEB), has been validated by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP). The CHMP is expected to give an opinion on the MAA in the second half of next year.

“The validation of our MAA for review by CHMP is an important step toward our goal to bring VYJUVEK to patients in the EU who are living with DEB.”
- Suma Krishnan, MS, MBA, of Krystal Biotech

4. Second Gene Therapy for Epidermolysis Bullosa up for Priority Review

  • Abeona Therapeutics' gene therapy Pz-cel for RDEB gets priority review, with a PDUFA date set for May 25, 2024. T
  • opline data from the VIITAL study reveals significant wound healing and pain reduction benefits.
  • Pz-cel, if approved, could complement existing gene therapies, addressing the complex challenges of RDEB.

October 20, 2023 — The FDA has accepted Abeona Therapeutics’ biologics license application (BLA) for its autologous, gene-corrected epidermal sheet therapy Pz-cel (prademagene zamikeracel; EB-101) with priority review for the potential treatment of recessive dystrophic epidermolysis bullosa (RDEB). The FDA has set a Prescription Drug User Fee Act (PDUFA) of May 25, 2024.

“The FDA’s acceptance of our BLA for priority review underscores the high unmet need in RDEB and the potential for pz-cel to provide meaningful benefit to these patients."
– Vish Seshadri, of Abeona Therapeutics

5. The Lupus Approach: CAR-T Begins the Jump from Blood Cancers to B-Cell Driven Autoimmune Disease

  • 2023 witnessed a surge in clinical trials for CAR-T therapies targeting B-cell-driven autoimmune diseases, including those with dermatologic manifestations.
  • Several companies, including Kyverna Therapeutics, Cabaletta Bio, and ImmPACT Bio, explore CAR-T therapies for lupus and other autoimmune diseases.
  • Safety concerns and the need for collaboration between oncologists and autoimmune disease specialists emerge as critical themes in cell therapy development.

December 20, 2023 — In continuation of a trend that initially got rolling at the tail-end of last year, 2023 saw a record number of investigational chimeric antigen receptor T-cell (CAR-T) therapies enter clinical trials for the treatment of autoimmune diseases. These newly initiated studies, many of which are recruiting patients with systemic lupus erythematosus (SLE) and lupus nephritis (LN), seek to push the boundaries of a therapeutic modality that originally proved itself in the field of oncology.

"Historically, our cell therapy group doesn't necessarily work with the physicians and their staff that treat autoimmune diseases: the rheumatologists, the nephrologists, dermatologists, neurologists, etc. We certainly have relationships—we know each other, and we share patients back and forth—but we don't have the same experience having a joint program.”
– David Porter, MD, of UPenn

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