Rare disease drug development has a math problem.
One drug. One disease. $1B+. 10+ years.
That math doesn't work for 1500+ rare metabolic diseases. The proof: 80% still have no treatment.
At @PerlaraPBC & Cure SRD5A3 I’ve spent 6 years discovering and developing medicines for rare diseases. The TradBio model doesn't work for rare.
My takeaway: many rare metabolic diseases break down at the same hubs in metabolism.
Target the hub. Treat many diseases with a single medicine.
Today I'm announcing @Metabolisbio. A biotech built with my co-founder @eperlste to develop keystone medicines: a new class of treatments for rare metabolic diseases.
We discover naturally occurring metabolites and develop them into medicines that can treat many rare diseases.
We also run AI-powered at-home patient studies. Faster data. Lower cost. No traveling to a trial site.
Our first program is NMN for DHDDS-CDG. A pediatric neurodegenerative disease.
NMN has already shown it can slow disease progression in patients. Read the preprint: https://t.co/KVD7uAq1DQ
We're raising a pre-seed round. If you believe rare disease is the proving ground for the next generation of metabolic medicines let's talk.
https://t.co/oh4bvl7YcI
We're raising a pre-seed round.
If you believe rare disease is the proving ground for the next generation of metabolic medicines, let's talk.
https://t.co/vfnJD9cEDB
Our first program is NMN for DHDDS-CDG. A pediatric neurodegenerative disease.
NMN has already shown it can slow disease progression in patients.
Read the preprint: https://t.co/rj5I9mJO5O
Today I'm announcing @Metabolisbio. Abiotech built with my co-founder @eperlste to develop keystone medicines: a new class of treatments for rare metabolic diseases.
Today is World Congenital Disorders of #Glycosylation Awareness Day #WorldCDGDay
To raise awareness & celebrate our wonderful community - we put together a report on SRD5A3-CDG & the ongoing research at Cure SRD5A3!
Read it here
https://t.co/beFD6jpLhQ
#StandUnited4CDG
@Mom2EmAndTy Laura, I am so very sorry for your loss. Emily was such a bright light I will always remember her laughter, hugs and wonderful spirit. Sending you and your family love during this difficult time
Inspiring profile of #NGLY1 super-advocate @mattmight and all that he’s done for his family, the #NGLY1 community, and the rare community writ large.
https://t.co/VofGJZpPSz