The Orphan Drug Act (ODA)
What is an Orphan Disease?
The New Technological Hope
What is Gene Therapy?
The Biohealth Capital Region Companies (BHCR)
- Vielo Bio, Inc., Gaithersburg, Maryland
- Vielo Bio is creating options to address severe inflammation and autoimmune diseases. Though they are unusually fronted with rare disease drugs, they have raised $350 million dollars from large investors since their conception in February 2018 and have big plans for more when they go public.
- American Gene Technologies., Rockville, Maryland
- American Gene Technologies uses cell and gene therapies to develop options for liver cancer, phenylketonuria (PKU), HIV, and genetic disorders. Their leading treatment candidate is aiming to cure HIV.
- REGENXBIO, Rockville, Maryland
- REGENXBIO uses gene therapy to develop treatments for retinal, neurodegenerative, and metabolic diseases. They are attempting work with adeno-associated viral vectors (AAV) which can be used for a wide range of diseases; they have the rights to 100 novel AAV vectors and 100 more in earlier stages, though only two have been approved to date.
- Cerecor, Inc. (CERC), Rockville, Maryland
- Cerecor, Inc. focuses on innovative solutions for orphan diseases, neurology, and ultra-rare pediatric diseases including through prescription medications, devices, and dietary supplements.
- Asklepion Pharmaceuticals, Baltimore, Maryland
- Asklepion Pharmaceuticals focuses exclusively on rare diseases, especially rare pediatric diseases that lack treatment options.
- Elixirgen Therapeutics Inc., Baltimore, Maryland
- Elixirgen Therapeutics, Inc., uses stem cells for diseases including rare blood disorders and chromosomal diseases.