Rare Disease Collaboration: NORD And Aldeyra Therapeutics
Aldeyra Therapeutics, a Burlington, Massachusetts-based biotechnology company developing treatments for rare skin and eye diseases, announces December 4th, a collaboration with NORD (National Organization for Rare Disorders), a non-profit committed to helping people with rare diseases. The partnership’s mission is to increase the awareness of the rare disease, Sjögren-Larsson Syndrome (SLS), to give patients the opportunity to connect with each other, and to access information about the disease, resources, and treatments.
Patients with SLS exhibit multiple medical conditions: retinal dysfunction, severe dry skin, spasticity, and mental delay. In order to inherit SLS, both parents each carry one copy of the mutated gene. SLS affects about 1,000 people in the U.S. and approximately 2,000 people in Europe.
Under the agreement:
“NORD will develop a multi-phased approach to enhance the ability of the SLS population to communicate and receive optimal care through development of a patient contact registry and other tools, such as social media. This will be conducted through RareLaunch™, NORD’s capacity-building and mentor program, through which it works with rare disease patients to develop or evolve disease-specific communities for the purpose of sharing information, accessing resources and learning from medical experts.”
Aldeyra Therapeutics, founded in 2004, is developing drug candidates that are designed to trap aldehydes (by binding to and allowing for the disposal of free aldehydes) :
“Free aldehydes are naturally occurring chemical species that, among other things, promote inflammation. At high levels, aldehydes are toxic, binding proteins, lipids, carbohydrates, and DNA … A variety of diseases are thought to be related to free aldehydes, including autoimmune diseases, inflammatory diseases, neurological diseases, cardiovascular diseases, and endocrinologic disease.”
Aldeyra Therapeutics’ lead drug candidate, NS2, is a topical formulation for the treatment of SLS. A NS2 eye drop formulation for the treatment of Acute Anterior Uveitis (inflammatory eye disease) is also being developed. The company plans on filing FDA Investigational New Drug (IND) applications for clinical testing of NS2 in 2014 for both SLS and acute anterior uveitis.