Following last week’s BIO fly-in to meet lawmakers on Capitol Hill, Rep. Jake Ellzey (R-TX) yesterday had a “fly-out” to tour BIO member company Nanoscope Therapeutics in Arlington, TX, located in his congressional district.
Nanoscope Therapeutics is a late-stage clinical biotech developing gene therapies to cure genetic diseases that cause vision impairment and blindness. Rep. Ellzey met CEO Sulagna Bhattacharya, a member of the BIO Board.
Nanoscope’s treatment, MCO-010, achieved the primary endpoint, “demonstrating a statistically significant improvement of best-corrected visual acuity” in people with advanced retinitis pigmentosa (RP) in a Phase 2 clinical trial, Nanoscope announced last month.
The drug received Orphan Drug and Fast Track designation by the U.S. Food and Drug Administration (FDA) for treating RP and Stargardt, two rare diseases. MCOs may address another indication: “all forms of retinal degeneration,” the company says.
What Rep. Ellzey heard: Legislative priorities include the ORPHAN Cures Act, which encourages researching second indication uses for orphan drugs, and thanking Rep. Ellzey for voting to restore the R&D tax deductions which is critical, even for companies like Nanoscope that aren’t yet earning sales revenues. He also heard about the firm’s impact on patients and the local economy.
BIO’s view: “Having Members of Congress witness first-hand the innovative research of BIO’s member companies helps us tell our story and emphasizes the work our industry is doing to tackle devastating diseases,” said Aiken Hackett, BIO VP of Federal Government Relations. "These visits are excellent opportunities to underscore how policies in Washington directly impact the development of therapies and cures, and even more so, the patients and their families.”
