Bloomsbury Genetic Therapies

Neurogene Inc

This company develops genetic medicines and gene therapies to treat patients and families affected by rare neurological diseases including Rett syndrome and Batten disease.

Genespire

This company develops off-the-shelf gene therapies for children and adults affected by inherited metabolic diseases and primary immunodeficiencies.

Mahzi Therapeutics

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

Comet Therapeutics

This biotechnology company develops coenzyme A metabolism therapies to treat patients with inherited metabolic diseases and orphan neurological disorders.

Minoryx

This biotechnology company develops orphan drugs and therapies for patients suffering from rare, life-threatening genetic diseases of the central nervous system.

Uncommon Therapeutics

This company develops gene-based medicines for patients with rare neurodevelopmental disorders, with an initial focus on treating Rett Syndrome.

Orchard Therapeutics

Orchard Therapeutics develops haematopoietic stem cell gene therapies for patients living with severe genetic diseases.

Bluebird Bio

This company develops and manufactures gene therapies for patients with severe genetic diseases, including sickle cell disease and beta-thalassaemia.