Simoladagene autotemcel

Unassigned

New Medicines

Adenosine deaminase deficiency (ADA) in patients with severe combined immunodeficiency due to ADA

Information

Advanced therapy medicinal product (ATMP)
Orchard Therapeutics
Orchard Therapeutics

Development and Regulatory status

Phase II Clinical Trials
None
Phase II Clinical Trials
Yes
Yes

Category

ADA SCID ex-vivo lentiviral gene therapy, uses stem cells from the patients and corrects them genetically outside the body (ex-vivo) with a lentiviral vector carrying a functioning copy of the missing or faulty gene. Single dose infusion.
ADA deficiency is very rare and is estimated to occur in approximately 1 in 200,000 to 1,000,000 newborns worldwide. Most individuals are diagnosed in the first 6 months of life. Without treatment, they do not usually survive past age 2 years. In 10 to 15% of cases, onset of immune deficiency occurs between 6 and 24 months of age (delayed onset) or in adulthood (late onset) [1].
Adenosine deaminase deficiency (ADA) in patients with severe combined immunodeficiency due to ADA
Intravenous infusion