Mariposa Therapeutics

BioMendics

BioMendics develops liquid crystal small molecule therapeutics for patients suffering from rare genetic dermatologic disorders such as epidermolysis bullosa simplex.

Castle Creek Biosciences

This company develops cell and gene therapies for patients suffering from rare genetic skin and connective tissue diseases.

Krystal Biotech

This biotechnology company develops and commercialises redosable gene therapies for patients with rare genetic diseases, including dystrophic epidermolysis bullosa.

Orvida Pharma (Formerly Kamari)

This biotechnology company develops oral and topical small-molecule treatments for patients with rare and severe genetic skin diseases and chronic itch conditions.

Aegle Therapeutics

This company develops extracellular vesicle therapies derived from stem cells to treat patients with rare dermatological disorders and severe burns.

RougeTx

This biotechnology company develops small-molecule therapies that restore vascular stability to treat patients with hereditary haemorrhagic telangiectasia and other bleeding disorders.

Mahzi Therapeutics

This biotechnology company develops gene and antisense oligonucleotide therapies for patients with rare genetic neurodevelopmental disorders.

Imara

This biopharmaceutical company develops oral small-molecule therapies to treat patients with rare genetic blood disorders, including sickle cell disease and beta thalassemia.