In the next six months, the FDA expects to issue decisions on three novel therapies.
Read moreNew Gene Therapy Approved for Genetic Hearing Loss
On April 23, 2026, the US Food and Drug Administration approved gene therapy Otarmeni (lunsotogene parvec-cwha) for the treatment of genetic hearing loss.
Read moreRyoncil Update
On December 18, 2024, the FDA approved Ryoncil (Remestemcel-L-rknd) as the first and only off-the-shelf allogeneic bone marrow-derived mesenchymal stromal cell therapy for children who are two months old to adolescents with steroid-refractory acute graft-versus-host disease (SR-aGVHD).
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 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 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 moreGene Therapy Summary 2019-2024 from a Reinsurance/Stop Loss Perspective
Summit Re sees increasing number of gene therapy cases.
Read moreGene and Cell Therapy Update
November ushered in two new therapies, AUCATZYL® (obecabtagene autoleucel), a CAR-T therapy which was approved on 11/8/2024, and Kebilidi™ (eladocagene exuparvovec-tneq), a gene therapy which was approved on 11/13/2024.
Read moreNew Treatment for Uveal Melanoma: HEPZATO
Uveal melanoma is the most common primary intraocular malignancy, and while several local therapies can be offered for primary disease control such as plaque brachytherapy, proton therapy and enucleation, effective therapies to control distant disease have been limited.
Read morePfizer Voluntarily Withdraws All Lots of Sickle Cell Disease Treatment OXBRYTA® (voxelotor) from Worldwide Markets
Pfizer announced on September 25, 2024, that they are voluntarily withdrawing all lots of OXBRYTA (voxelotor) and discontinuing all active clinical trials and expanded access programs worldwide.
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