No mutation left behind – that’s the concept of Perlstein Lab, a Bay Area biotech and public benefit corporation that explores precision orphan drug delivery.
Its thesis is that though 1 in 12 people have some variety of orphan disease, the vast majority – some 95 percent – have no treatment. Because each orphan condition is caused by a “spectrum of genetic mutations,” the company said it studies each mutation in low-cost, easily malleable model organisms like yeast, worms and flies to ultimately develop personalized medicine.
Perlstein Lab raised $2 million in seed funding recently. CEO Ethan Perlstein participated in a Q&A with Total Orphan Drugs, describing his experience of “Generation Postdocalypse” influencing the choice to enter the biotech startup arena – and why the largely underserved orphan disease sector is a worthwhile path to pursue. Check it out:
With the Rise of AI, What IP Disputes in Healthcare Are Likely to Emerge?
Munck Wilson Mandala Partner Greg Howison shared his perspective on some of the legal ramifications around AI, IP, connected devices and the data they generate, in response to emailed questions.
How to Start a Biotech: Fundraising and the Future of the Orphan Drug Industry
His approach to fundraising:
Perlstein Lab has an unusual coalition of strategic and angel investors. The round was co-led by the public orphan drug company Retrophin and the Bay Area Wilsey family, which is personally affected by an ultra-orphan disease called NGLY1 deficiency. Twitter played an out-sized role in Perlstein Lab’s fundraising success. I met the founder of Retrophin, Martin Shkreli, on Twitter. And I was introduced to Matt Wilsey by my “tweep” Molly Lindquist, a patient advocate and founder of the nonprofit biomedical research crowdfunding platform called Consano. I also posted fundraising updates and press on Perlstein Lab’s AngelList page to raise our profile.
His thoughts on the future of the orphan drug industry:
A Personalized Approach to Medication Nonadherence
At the Abarca Forward conference earlier this year, George Van Antwerp, managing director at Deloitte, discussed how social determinants of health and a personalized member experience can improve medication adherence and health outcomes.
Thanks to the Internet, patients and advocates are becoming more scientifically empowered and financially savvy such that they now can start their own biotech companies or nonprofit drug discovery and development ventures. At the same time, scientist-driven biotech startups are springing up as R&D dries up in larger biopharma companies that favor acquisition. I predict that the resulting upswell in innovation and productivity will force FDA to adapt and accommodate radically over the next decade.
