Shire plc (LSE: SHP, NASDAQ: SHPG) and the US Foundation Fighting Blindness (FFB) are set to collaborate on a new initiative to accelerate the development of therapies that could treat autosomal dominant retinitis pigmentosa (adRP). The company has demonstrated a significant expansion of its business interests in the ophthalmic market in recent years, now under-scored with a strategic partnership capable of providing valuable expert knowledge in the field of inherited retinopathies. Under the agreement, FFB and its “research partners” are understood to provide Shire with knowledge and expertise in adRP, while the foundation are aiming to benefit from an accelerated development of novel therapies for the treatment of the retinal disorders.
Retinitis pigmentosa (RP) may be inherited through a variety of mechanisms including dominant, recessive, X-linked, digenic and mitochondrial mutations. A little under half of all RP patients suffer from the dominant form of the disease, variously estimated at between 75,000 and 150,000 individuals in the developed world. Approximately a dozen genes or more are mutated in a dominant fashion to cause RP, a prime example being mutations in the rhodopsin gene, the protein product of which initiates the visual transduction cascade. Clinically, adRP is characterized by the gradual onset of night blindness in late childhood or teenage years, followed by a progressive constriction of the visual field and a loss of peripheral vision, often leading to legal or complete blindness. Several companies worldwide are in the process of developing gene therapies, and other therapeutic approaches, for the treatment of the disease.
A press release from the company and FFB outlined the new agreement and how FFB’s scientific advisory board may provide input and advice to Shire on pre-clinical and/or clinical development of Shire compounds for the treatment of adRP. Commenting on the announcement, Patricia Zilliox, Chief Drug Development Officer at FFB’s Clinical Research Institute stated, “Autosomal dominant retinitis pigmentosa is a rare, inherited disease that has a significant impact on patients and their families as they navigate the challenges associated with severe vision loss. Through our agreement with Shire, we are excited to drive research and development and extend our expertise to dedicated partners actively working to accelerate treatments for those living with retinal diseases.” The announcement was additionally welcomed by Shire’s Senior Vice President and Head of Global Research and Nonclinical Development, Albert Seymour, stating, “this agreement is an example of the creative approaches in early-stage rare disease research and development that we’re taking at Shire. Both organizations have a strong shared commitment to unearthing and developing innovative treatment opportunities in areas of significant, unmet medical need.”