International Niemann–Pick Disease Alliance
The Wylder Nation Foundation, which seeks treatment for acid sphingomyelinase deficiency (ASMD) NPD type A, has partnered with biotech company Perlara:
Perlara PBC, a company with a focus on ‘finding treatments for rare diseases previously believed to be too rare to cure’ has joined with Wylder Nation, in order to back research which could lead to positive potential treatments in the future. The Wylder Nation Foundation was set up in 2012 by parents Steven and Shannon Laffoon, after their son Wylder passed away at just three years old. Wylder left behind lessons of unconditional love and the power of hope – something which provided his parents with perspective on how to make a lasting impact on the world by paving the way to help others who may be struggling in the same situation. By taking lessons learned from Wylder’s life, they have used this as a catalyst for positive change, and this is exactly what fuels their foundation’s mission…and makes them so passionate and driven when it comes to accelerating the discovery of treatment options for other children with Lysosomal Storage Disorders (LSDs).
Last year, Perlara PBC announced its first commercial deal, a research collaboration with and investment by Novartis for three lysosomal storage disorders, starting with Niemann-Pick Type C, so this further partnership shows how research in this area is continually expanding. Steven Laffoon, Co-Founder and President of Wylder Nation Foundation, had this to say:
“Perlara’s unique drug discovery platform and business model is a perfect fit for advancing our efforts in discovering existing compounds that can be repurposed, and new novel compounds that can be optimised for treating Niemann-Pick Type A”.
After announcing their groundbreaking partnership, Wylder Nation have subsequently signed a “PerlQuest”, a title coined by Perlara to describe their research phases which is divided into three separate stages. The first stage will consist of a natural history study of ASMD NPD Type A using model organisms. The second involves screening an extensive library of known bioactive and approved compounds in a bid to validate the models, and finally the last phase entails a screening which seeks a novel lead compound or series of compounds that can be tested in mice.
Perlara CEO Ethan Perlstein argues that Perlara’s unique approach offers an innovative and efficient way for patient groups to expedite drug development for diseases that are often overlooked due to their rarity. Perlstein acknowledges the challenge that research into this area holds. However despite this he remains confident: “I still believe if you de-risk the compound enough there will be an ultra-rare company or a pharma firm out there to license it — The goal of the PerlQuest model is to democratise venture philanthropy and make it possible for any patient advocacy group anywhere in the world to be equal partners in their own personalized drug discovery journey”.
