Dystrophic epidermolysis bullosa (DEB) is a condition caused by mutations in gene COL7A in which skin becomes fragile and prone to blistering. For people with DEB, any injury or friction on the skin can lead to the formation of painful blisters.
Read moreNiemann-Pick Disease Type C
Niemann-Pick disease type C (NPC) is a rare progressive genetic disorder characterized by an inability of the body to transport cholesterol and lipids inside of cells. This leads to the abnormal accumulation of these fatty substances within various tissues of the body, including brain tissue.
Read moreAs the Cell and Gene Therapy World Turns
August 2025 cell and gene therapy updates
Read moreGene Therapy and Skin Substitute Updates
Read about developments with Elevidys, the Cell and Gene Therapy Access Model, and proposed changes for skin substitutes in 2026.
Read moreELEVIDYS on Hold for Non-Ambulatory Patients
Sarepta issued press release on June 15, 2025, titled “Sarepta Provides Safety Update for ELEVIDYS and Initiates Steps to Strengthen Safety in Non-Ambulatory Individuals with Duchenne.” As it examines the data, the company has initiated several safety initiatives.
Read morePersonalized Gene Therapy
A research team from CHOP and the Perelman School of Medicine at the University of Pennsylvania, supported by the National Institutes of Health, developed a customized gene therapy by utilizing the gene editing platform CRISPR.
Read morectDNA testing and treatment of stage 3 colon cancer
Clinical trial for stage 3 colon cancer shows promise.
Read moreNo Pain, No...Addiction?
On January 30, 2025, the United States FDA approved medication Journavx (suzetrigine), a first-in-class, non-opioid analgesic to treat moderate to severe acute pain in adults.
Read moreGene Therapy Summary 2019-2024 from a Reinsurance/Stop Loss Perspective
Summit Re sees increasing number of gene therapy cases.
Read moreMedicare Drug Price Negotiation
CMS initially selected ten drugs covered under Part D for the first cycle of negotiations with drug companies. These drugs include Januvia, NovoLog, Farigia, Enbrel, Jardiance, Stelara, Xarelto, Eilquis, Entresto, and Imbruvica.
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