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Immitra Bio

This company develops an in-vivo gene editing platform to provide off-the-shelf curative treatments for patients with rare genetic diseases.

Alia Therapeutics

The company develops gene editing medicines for patients with genetic diseases.

Code Bio

This company develops non-viral genetic medicine delivery platforms and therapies for patients with rare and prevalent genetic diseases.

Solid Biosciences

This life science company develops gene therapy candidates for patients living with rare neuromuscular and cardiac diseases.

Ultragenyx

This biopharmaceutical company develops and commercialises therapies for patients with serious rare and ultra-rare genetic diseases.

EDITAS Medicine

The company develops CRISPR-based genome editing medicines to treat patients with serious genetic diseases, including blood disorders and inherited eye conditions.

Rocket Pharma

This company develops gene therapies for patients with rare and devastating genetic cardiovascular and haematological diseases.

Stridebio

This company develops engineered viral vectors and gene therapies for patients with rare genetic diseases.