ProQR Therapeutics

Tay Therapeutics

This biotech company develops small molecule oral gene therapies for patients with rare genetic disorders and inflammatory conditions through licensing and strategic partnerships.

Shape Therapeutics

The company develops programmable RNA medicines and delivery systems for patients with genetic disorders and other hard-to-treat diseases.

ARTHEx Biotech

This clinical-stage biotechnology company develops RNA-based treatments for patients with rare neuromuscular genetic diseases, including myotonic dystrophy type 1.

Aera Therapeutics

This biotechnology company develops delivery platforms for genetic medicines to treat various diseases for biopharmaceutical partners and patients.

Strategic Partners

This company develops drug candidates for serious genetic disorders to serve patients with rare diseases.

Alia Therapeutics

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

Averna Therapeutics

This biotechnology company develops genomic medicines using RNA-based technology to insert therapeutic genes into safe genomic locations for patients with various diseases.

Scenic Biotech

This company develops disease-modifying small molecule therapeutics for patients with rare genetic and neurodegenerative disorders using its proprietary genetic modifier discovery platform.