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FDA Lifts Clinical Hold on Selecta Biosciences MMA Gene Therapy Candidate

March 11, 2022

Selecta Biosciences said that the U.S. Food and Drug Administration lifted its clinical hold on the company’s experimental gene therapy SEL-302 for methylmalonic acidemia.

Photo: Carsten Brunn, president and CEO of Selectra Biosciences

Methylmalonic acidemia (MMA) is a rare monogenic disorder in which the body cannot break down certain proteins and fats. This metabolic disease may lead to hyperammonemia and is associated with long-term complications including feeding problems, intellectual disability, chronic kidney disease, and inflammation of the pancreas. Symptoms of MMA usually appear in early infancy and vary from mild to life threatening. Without treatment, MMA can lead to coma and in some cases death.

SEL-302 is the company’s MMA-101 gene therapy plus its ImmTOR technology that is an immune tolerance platform the company expects will allow redosing of gene therapies by mitigating unwanted immune responses.

On November 23, the FDA issued a clinical hold in order to obtain additional information on the chemistry, manufacturing and controls related to the MMA-101 product candidate. There were no outstanding clinical or preclinical questions in the FDA letter. The clinical trial had not yet been initiated at the time the hold was placed.

Carsten Brunn, president and CEO of Selecta the company plans to begin a phase 1 clinical trial as quickly as possible.

Author: Rare Daily Staff

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