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 ...

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 ...

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 ...

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 ...

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 ...

Adenosine deaminase (ADA) deficiency is a rare autosomal recessive disorder that underpins a severe form of combined immunodeficiency (SCID), resulting in the accumulation of toxic metabolites that ...

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 ...

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 ...

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 ...

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 ...