NGNE vs CAPR Comparison

Neurogene Inc is a clinical-stage biotechnology company committed to overcoming the limitations of neurological diseases into treatable conditions, by harnessing its proprietary transgene regulation technology, EXACTTM (Expression Attenuation via Construct Tuning). It is building a differentiated product portfolio of genetic medicines for rare neurological diseases with high unmet needs. The drug candidates in the company's product pipeline include NGN-401, which is in development for the treatment of Rett syndrome, using its EXACT platform, and NGN-101, a non-EXACT, conventional gene therapy development candidate for the treatment of CLN5 Batten disease, designed to deliver the CLN5 gene and packaged in an AAV9 capsid.

Capricor Therapeutics Inc is a clinical-stage biotechnology company focused on the development of transformative cell and exosome-based therapeutics for treating Duchenne muscular dystrophy (DMD), a rare form of muscular dystrophy which results in muscle degeneration and premature death, and other diseases with high unmet medical needs. The company's product pipeline includes CAP-1002, deramiocel an allogeneic cardiac-derived cell therapy. Deramiocel is currently advancing through Phase 3 clinical development for the treatment of Duchenne muscular dystrophy. Through its proprietary StealthXTM platform, the group uses its exosome technology to conduct preclinical research focused on vaccinology, delivering oligonucleotides, proteins, and small molecules to treat.