SEL-302 for Methylmalonic Acidemia (MMA)
Our lead gene therapy product candidate is intended to treat MMA, an inborn error of metabolism that affects approximately one in 25,000-48,000 newborns in the United States. MMA patients have a deficiency in an enzyme known as methylmalonyl-CoA mutase (MUT) and are therefore unable to process certain proteins and fats, leading to accumulation of toxic metabolites. Symptoms start to develop in early childhood and those afflicted suffer from a wide range of severe disease-related complications despite strict diet.
SEL-302 combines an in silico-designed gene therapy vector known as Anc80 that Selecta has in-licensed from Massachusetts Eye and Ear (MEE) with transgenes discovered in the Venditti laboratory at the National Human Genome Research Institute (NHGRI) and Selecta’s proprietary SVP-Rapamycin (SEL-110). This gene therapy product candidate is designed to enable the treatment of patients with pre-existing antibodies; the ability to administer multiple doses to achieve and maintain sufficient levels of MUT over the course of the patient’s lifetime; and the prevention of cellular immune responses that often reduce the expression levels of gene therapies and cause liver toxicity.
To advance the MMA program, Selecta has entered into a Collaborative Research and Development Agreement (CRADA) with MEE and NHGRI.