RARECast is a Global Genes podcast hosted by award-winning journalist Daniel Levine. It focuses on the intersection of rare disease with business, science, and policy.
News last month of the Cystic Fibrosis Foundation’s $3.3 billion sale of royalty rights to Kalydeco and other Vertex Pharmaceuticals drugs it helped fund served as a bold statement to the growing prominence and power of venture philanthropy. We spoke to Margaret Anderson, executive director of FasterCures, about the transaction, the role new funding and collaboration models are playing in accelerating drug development, and what traditional investors and disease groups are learning from each other.
Prosensa, a biotechnology company developing a breakthrough therapy for the rare, fatal, genetic disease Duchenne muscular dystrophy suffered a big setback when disappointing, late-stage trial results of its lead therapeutic led its partner, GlaxoSmithKline, to return rights to the experimental drug in January 2014. Prosensa’s stock plummeted and the future of the company was uncertain. CureDuchenne, which had provided early funding for the company, stepped in once again. This time it made a $7 million commitment to restore the clinical programs and move the company forward. We spoke to Debra Miller, president and CEO of CureDuchenne about her experience with Prosensa, the recent news that BioMarin would acquire the company for up to $840 million, and the role venture philanthropists like her can play in accelerating the development of life saving drugs.
Ilan Ganot is not your typical biotech entrepreneur. When his son Eytani was diagnosed with Duchenne muscular dystrophy, he quit his job as a hedge fund manager with JPMorgan in London, relocated his family to Boston, and launched a company focused exclusively on finding treatments for Duchenne. We spoke to Ganot, CEO and founder of Solid Ventures, about his company, the thinking behind his business model, and whether his approach is transferable to other rare diseases.
Crowdfunding, once thought of as little more than a way to raise money for small scientific projects, is increasingly being used to raise significant capital to fund startups. Perlstein Lab, a San Francisco-based starup focused on finding new drugs to treat rare diseases, raised more than $2 million using crowdfunding. We spoke to Ethan Perlstein, founder and CEO of Perlstein Lab, about his company, the potential of crowdfunding, and the unusual role social media has played in his efforts to raise capital.
The combination of high cost, long development times, and small patient populations for rare diseases is driving new models to accelerate drug development. The story of the newly formed rare disease company Vtesse is a compelling example. Driven by parents, advanced to the clinic by the National Institutes of Health, and licensed by a new company formed out of an orphan drug accelerator, Vtesse is advancing an experimental drug to treat Niemann Pick Disease Type C and other lysosomal storage disorders. We spoke to Ben Machielse, CEO of Vtesse, about his company, the unusual collaborations that led to its formation, and what it says about how patients, government, investors, and industry can work together to speed the development of new therapies for rare diseases.
At the age of 8, Bill Elder, Jr. was diagnosed with cystic fibrosis, a rare, genetic disease expected to cut his life short. Now a third year medical student, Elder has been the beneficiary of Vertex Pharmaceutical’s Kalydeco, an example of the transformative effects personalized therapies can have on patients. As President Barack Obama announced plans for a precision medicine initiative during his State of the Union address, Elder sat alongside First Lady Michelle Obama as a living and breathing testament to the promise of precision medicine. We spoke to Elder about his recent visit to Washington, his experience with cystic fibrosis, and the difference Kalydeco has made in his life.
World Rare Disease Day, an annual observance held on the last day of February, seeks to raise awareness for rare diseases. On that day, hundreds of patient organizations from more than 80 countries will be participating in activities to call attention to thousands of ailments, many of which are without treatments or even names. We spoke to Hudson Freeze, director of the Human Genetics Program at Sanford Burham Medical Research Institute, about World Rare Disease Day, the importance of raising awareness, and how research into rare diseases can shed light into more common ailments.
The search for cures for diseases is limited by the ability of scientists to consume and understand the rapidly expanding volumes of biomedical literature produced each year. Andrew Su, associate professor in the department of Molecular and Experimental Medicine at The Scripps Research Institute, has a solution. He wants to enlist members of the rare disease community and general public to become citizen scientists and pour through biomedical journals to help find connections and identify research in one area that may shed light on another. We spoke to Su about his project Mark2Cure, the role crowdsourcing can play in accelerating rare disease research, and why members of the rare disease community may better suited for elucidating important findings than supercomputers.
Noah Coughlan will set out on a 3,100 mile run across America from the Statue of Liberty to San Diego’s Ocean Beach in the hope of raising awareness and support in the fight against rare diseases. It will be the third run across the country for the founder of the Run4Rare Foundation and begin on February 28, World Rare Disease Day. We spoke to Coughlan about his run, what motivated him to do this, and what he hopes to accomplish.
Recursion Pharmaceuticals has set the audacious goal for itself of developing 100 drugs in 10 years for rare diseases. The company, focused on repurposing compounds abandoned in clinical development by other drugmakers, thinks it can achieve this by a fundamentally different approach to drug development than the industry’s traditional process of screening compounds against potential disease targets. We spoke to Chris Gibson, founder and CEO of Recursion, about the approach his company is taking, whether rare diseases particularly lend themselves to it, and whether it has implications more broadly for drug development.
