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 ...

Gene therapy treatments for rare diseases are being developed, but getting them out of the lab has proved challenging.

X-linked severe combined immunodeficiency disease (X-SCID) is a rare genetic disorder characterized by profound defects in T-cell, B-cell, and natural killer (NK) cell function, caused by mutations in ...

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 ...

An investigational gene therapy showed sustained clinical efficacy for children born with ADA-SCID. In a study of 62 children, overall survival after treatment was 100% and event-free survival was 95% ...

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 ...

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 ...

Infants with severe combined immune deficiency (SCID) who are diagnosed and receive hematopoietic cell transplant at age 3.5 months or earlier have a 94% probability of survival, according to a study ...

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 ...

For children born with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID), an investigational gene therapy showed sustained clinical efficacy and safety at a median ...

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 ...