Navigation Links
Perlstein Lab Raises $2 Million For Orphan Drug Discovery
Date:8/5/2014

SAN FRANCISCO, Aug. 5, 2014 /PRNewswire/ -- Perlstein Lab PBC, a San Francisco-based public benefit corporation whose mission is precision orphan drug discovery, just closed a $2 million seed round. The round was co-led by the clinical-stage orphan drug developer Retrophin, Inc. and the Wilsey family, which is personally affected by an orphan disease.

One in 12 people suffer from an orphan disease, yet 95% of orphan diseases have no FDA-approved drugs. At the current rate of 10-20 new orphan drug approvals per year, it will take another 450 years before drugs are approved for all orphan diseases. Perlstein Lab's scalable platform generates patient-specific small-molecule orphan drug candidates by combining: (1) patient mutation data, (2) high-throughput screens of genome-edited primordial disease models, and (3) lead optimization and validation studies in genetically matched patient cells. The successful cystic fibrosis drug Kalydeco, which was developed by Vertex Pharmaceuticals with financial contributions from the Cystic Fibrosis Foundation, proves that specific mutations can be targeted and suppressed by small molecules.

First, Perlstein Lab customizes primordial disease models for orphan disease patients by editing the genomes of yeast cells, worms, fruit flies and zebrafish strains to express patient-derived mutations in the ancestral versions of human orphan disease genes. Although most preclinical research currently uses laboratory rodents, basic researchers have used non-mammalian "simple model organisms" for decades to elucidate fundamental aspects of human biology. Drug screening with genetically personalized fruit flies was key to the development of the approved thyroid cancer drug Caprelsa, which was developed by AstraZeneca with scientific contributions from the laboratory of Dr. Ross Cagan.

Next, Perlstein Lab will perform whole-organism phenotypic screens to produce mutation-specific lead compounds faster, cheaper and more systematically than current approaches. Finally, Perlstein Lab will funnel optimized leads into toxicology and clinical biomarker studies in patient-derived cells. The end results will be IND-ready orphan drug candidates that will be co-developed with commercial partners via licensing deals. This genetically informed approach improves the likelihood of bringing personalized orphan drugs to market at scale, and with minimized use of lab mice and rats.

"We will initially focus on lysosomal storage diseases, which are not only de-risked scientifically, but also have some of the most well-organized patient advocacy groups," said CEO and founder Ethan O. Perlstein, Ph.D. Before leaving academia in 2012, Ethan Perlstein received a Ph.D. in molecular and cell biology from Harvard University and completed an independent postdoctoral fellowship at Princeton University. Perlstein Lab scientists include Dr. Nina DiPrimio, Dr. Tom Hartl, Dr. Sangeetha Iyer and Kiran Singh. Henry Perlstein is co-founder and CTO. Dr. Jun Axup is the Director of Outreach. Tom Ruginis, CEO of HappiLabs, is the virtual lab manager. Rounding out the core team are Dr. John Alan Tucker, a medicinal chemistry advisor, and Dr. Laura Shawver, a business development advisor. More about the team and the science can be found at www.perlsteinlab.com.

Contact: Ethan Perlstein, ethan@perlsteinlab.com

Logo - http://photos.prnewswire.com/prnh/20140805/133384


'/>"/>
SOURCE Perlstein Lab PBC
Copyright©2014 PR Newswire.
All rights reserved

Related biology technology :

1. Avaxia Biologics, Inc. Raises $2.2 Million in First Closing of Series A Financing to Advance Oral Antibodies for Inflammatory Bowel Disease
2. Cowen Healthcare Royalty Partners Raises $1 Billion
3. Biotech Firm Spun Off from Childrens Hospital Raises $7 Million to Advance Magnetic-Based Vascular Treatment
4. Cerecor Raises $22 Million Series A Financing
5. Sangart Raises Additional $50M Series G Funding Following Interim Safety Analysis Of Phase 2b Study Of MP4OX In Severe Trauma Patients With Hemorrhagic Shock
6. Rhythm Raises $25M in Series B Financing for Diabetes and Obesity Therapeutics
7. Juventas Therapeutics Raises $22.2 Million Series B Financing
8. Relmada Therapeutics Raises $3.0 Million In Initial Closing Of Series A Financing
9. Safeway Raises Over $9 Million to Fund Prostate Cancer Research
10. MiMedx Group Raises over $4.9 Million through the Exercise of Callable Warrants
11. OxThera Raises Capital for Further Clinical Development of Oxabact(R)
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:4/27/2016)... ... 2016 , ... Shimadzu Scientific Instruments (SSI) will be showcasing ... Conference and Expo. Shimadzu’s high-performance instruments enable laboratories to test cannabis products for ... stop by booth 1021 to learn how Shimadzu’s instruments can help improve QA/QC ...
(Date:4/27/2016)... ... April 27, 2016 , ... Most consumers engage with biometrics ... for secure access, voice recognition for hands-free communication, and facial recognition to help ... biometrics technology today. But if they asked Joey Pritikin, Vice President of ...
(Date:4/27/2016)... and RESEARCH TRIANGLE PARK, N.C. ... UTHR ) announced today that Martine ... United Therapeutics will provide an overview and update on ... Annual Health Care Conference. The presentation ... 10:00 a.m. Eastern Time, and can be accessed via ...
(Date:4/27/2016)... ... April 27, 2016 , ... NDA Partners Chairman ... company as an Expert Consultant. Mr. Clark was formerly a Vice President ... development of small molecule monographs based on analytical methods. NDA Partners Expert ...
Breaking Biology Technology:
(Date:4/14/2016)... Israel , April 14, 2016 ... Authentication and Malware Detection, today announced the appointment of ... assumed the new role. Goldwerger,s leadership appointment ... on the heels of the deployment of its platform ... BioCatch,s behavioral biometric technology, which discerns unique cognitive and ...
(Date:3/29/2016)... , March 29, 2016 ... "Company") LegacyXChange "LEGX" and SelectaDNA/CSI Protect are pleased to ... ink used in a variety of writing instruments, ensuring ... of originally created collectibles from athletes on LegacyXChange will ... analysis of the DNA. Bill Bollander ...
(Date:3/21/2016)... 2016 Unique technology combines ... superior security   Xura, Inc. ... secure digital communications services, today announced it is working ... enterprise customers, particularly those in the Financial Services Sector, ... authentication within a mobile app, alongside, and in combination ...
Breaking Biology News(10 mins):