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SEL-302

Development stage: Phase 2
Lead developer: Selecta Biosciences
Modality: Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, Small Molecules, Nanoparticles → Drug Delivery Systems
Administration: Intravenous

Overview

SEL-302 is an investigational gene therapy developed for the treatment of methylmalonic acidemia (MMA), a rare inherited metabolic disorder. It consists of two components: MMA-101, a recombinant adeno-associated virus serotype 8 (AAV8) vector delivering the human methylmalonyl-CoA mutase (MMUT) gene to the liver, and ImmTOR (also referred to as SEL-110), a nanoencapsulated form of sirolimus designed to induce immune tolerance and enable redosing by mitigating unwanted immune responses against the viral vector or transgene[1][2][3][4][5][7]. The therapy aims to provide a potentially durable and lifelong correction for patients with MMA by restoring MMUT enzyme activity.

Other names: MMA-101 plus ImmTORMMA101 plus ImmTORMMA 101 plus ImmTOR

Targets

MMUT (Methylmalonyl-coenzyme A mutase)Mechanistic target of rapamycin complex 1

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