Perhaps despite not having the cache of ‘guaranteed-weight-loss’, ‘quit smoking now’ or even a cure-all for cancer, pharmaceuticals for the treatment of rare diseases can be an attractive investment strategy.

Amicus Therapeutics is the developer of small molecule, orally administered drug called pharmacological chaperones for the treatment of human genetic diseases. The company is currently developing drugs for the treatment of Fabry disease, Gaucher disease and Pompe disease. It focuses on lysosomal storage disorders and diseases of neurodegeneration.
Active investors in Amicus include CHL Medical Partners (formerly known as Collinson Howe & Lennox), Canaan Partners, Frazier Healthcare Ventures, New Enterprise Associates and Prospect Venture Partners.

CHL Medical Partners is a premier venture capital firm that partners with entrepreneurs and inventors to create and finance innovative biotechnology, molecular diagnostic, healthcare service and medical device companies. They are known throughout the industry for identifying scientific ideas and healthcare service models and creating businesses with excellent market potential. They have invested in over 80 start-ups and are currently investing their sixth fund, the $120 million CHL Medical Partners III, L.P.

Canaan Partners, founded in 1987, is a venture capital fund that invests in all stages of development, notably Series A & B financing. Headquartered in Gurgaon, Haryona, India, the firm typically invests $1 million to $20 million in its portfolio company.

One of the country’s leading providers of venture and growth equity capital to emerging healthcare companies, Frazier Healthcare Ventures partners with outstanding entrepreneurs to build durable companies that deliver therapies and services to largely unmet healthcare needs. The firm has invested in over 100 companies in the biopharma, medical device, and healthcare service sectors since their inception in 1991.

Menlo Park, California-based New Enterprise Associates invests primarily in the information technology sector with a focus on communications, software and services as well as the electronics and healthcare industries. The venture capital group concentrates on energy technology, biopharmaceuticals, medical devices, healthcare services and healthcare information technology industries.

Prospect Venture Partners is a venture capital firm with over $1B of capital currently under management. The firm, dedicated to investing in outstanding biopharmaceutical and medical device companies, invests in companies with a wide variety of development and financing requirements including new company incubations, first and second venture financing rounds and later stage private and public companies with proven business models requiring expansion capital. They typically have representation on the board of each of its portfolio companies and serves as an active partner.