Gene therapy is superior to haploidentical hematopoietic stem cell transplantation (HSCT) to treat X-linked severe combined immunodeficiency (SCID-X1), according to a report published online April 13 ...
Overall and event-free survival of 100 and 95%, respectively, seen with autologous CD34+ hematopoietic stem-cell lentiviral gene therapy. HealthDay News — Autologous CD34+ hematopoietic stem-cell ...
Current GT strategies employ viral vectors (eg, murine g-retroviruses or lentiviruses) to introduce genetic material into isolated HSCs. However, gene editing (eg, CRISPR-based GT) is under active ...
Kohn, MD will speak about gene therapy for Inborn Errors of Immunity NEW YORK, Feb. 11, 2026 /PRNewswire/ -- On March 25, 2026 the Jeffrey Modell Foundation (JMF) will host Donald B. Kohn, MD, a ...
A blood stem cell gene therapy co-developed by UCLA’s Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare and fatal immune disorder, with no serious complications ...
Rarity PBC, a Public Benefit Corporation dedicated to expanding the availability of transformative gene therapies for rare diseases, today announced the closing of a $4.6 million seed financing led by ...
Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune cells, provide nonspecific immunity for infants early in life. Patients with ...
Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by ...
Skin cancer is typically caused by damage to the skin's cells from ultraviolet radiation. But a recent case study has just shed light on another potential cause: human papillomavirus. Early results ...
AGC Biologics Partners with Rarity PBC to Advance Life-Saving Gene Therapy for "Bubble Baby Disease"
Collaboration will leverage AGC Biologics’ commercial manufacturing experience and proven platforms to seek regulatory approval for a therapy treating the rare immunodeficiency disorder, ADA-SCID.
DUBLIN – Bidding to become the second European firm to win a gene therapy approval, Glaxosmithkline plc filed a marketing authorization application (MAA) with the EMA for GSK2696273, a treatment for ...
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