News

The U.S. Food and Drug Administration (FDA) has refused to approve the initiation of Abeona Therapeutics‘s Phase 3 clinical trial evaluating its gene-corrected cell therapy EB-101 for recessive dystrophic epidermolysis bullosa (RDEB). The clinical hold, the company explained, is…

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…

A novel gene editing technique was able to correct mutations in COL7A1 in cells taken from people with recessive dystrophic epidermolysis bullosa (RDEB), providing a proof of concept for using the editing technique in the disease. The finding was published in the Journal of Investigative Dermatology in a study titled,…

A type of wound dressing, called dialkylcarbamoylchloride (DAAC)‐coated cotton acetate, closes infected wounds related to epidermolysis bullosa (EB) faster than the standard saline dressing plus a topic antibiotic, and with similar antibacterial effects, a study found. The study, “Clinical efficacy of dialkyl-carbamoyl chloride‐coated cotton acetate dressing…

Cell therapy with genetically engineered skin grafts requires that collagen type VII be “corrected” in two types of skin cells — keratinocytes and fibroblasts — to be effective against recessive dystrophic epidermolysis bullosa (RDEB), a study in mice has found. Researchers saw that only skin models made of collagen…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

RegeneRx Biopharmaceuticals announced the first patient enrolled in a Phase 2 clinical trial of RGN-137, the company’s investigational topical gel for treating epidermolysis bullosa (EB), achieved complete wound healing. RGN-137 is a gel form medication intended to accelerate wound healing when applied directly on skin lesions. It is…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

Injection of mesenchymal stromal cells (MSCs) — cells that can be isolated from several tissues and may grow into different cell types when cultured in a lab dish — may prevent localized skin blistering sometimes seen in recessive dystrophic epidermolysis bullosa (RDEB), a study says. Results from the study,…

Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people who’ve made it to adulthood. Neena Nizar doesn’t have to imagine. The 41-year-old English professor at Metro Community College in Elkhorn,…

Amryt Pharma is conducting a Phase 3 clinical trial evaluating the effectiveness and long-term safety of Oleogel-S10 (AP101), a topical formulation of birch bark extract, for accelerating wound healing in patients with epidermolysis bullosa (EB). EASE is a pivotal study designed to support regulatory submissions seeking approval…

With its lead therapy candidate AGLE-103 set to begin clinical testing in the coming months, Aegle Therapeutics is being honored with a Partners in Progress Award by the Dystrophic Epidermolysis Bullosa Research Association of America (Debra of America). Aegle is among three companies being recognized for its efforts to…