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 phen
'/>"/>

SOURCE Perlstein Lab PBC
Copyright©2014 PR Newswire.
All rights reserved

Page: 1 2 3

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:11/26/2014)... 2014 SoundConnect , an award ... to announce that Darren Suders has joined the team ... will drive the partner program through innovative ... , Darren brings more than 10-years of channel ... in channel operations, from policy development, to partner education ...
(Date:11/26/2014)... 2014 Deep Research Report on ... report on the Global Palmitic Acid industry. For ... basic information, including definition, classification, application, industry chain ... etc. , For international and China market ... in China and other countries or regions ...
(Date:11/26/2014)... MINNEAPOLIS , Nov. 25, 2014 Bio-Techne ... it has appointed Robert Gavin to serve ... of the Protein Platforms Division.  Mr. Gavin will be ... business segment, which includes the ProteinSimple business acquired in ... San Jose, California -based ProteinSimple ...
(Date:11/26/2014)... 2014  Roka Bioscience, Inc. (NASDAQ: ROKA ), ... for the detection of foodborne pathogens, today announced that ... 26 th Annual Healthcare Conference on December 2, 2014 ... New York Palace in New York, NY ... the Company, its operations, strategies and prospects may be discussed. ...
Breaking Biology Technology:SoundConnect Appoints Director of Channel Development & Sales 2Palmitic Acid Industry Global 2014 Analysis & 2020 Forecasts on Market data, Statics, Development Now at ReportsnReports.com 2Palmitic Acid Industry Global 2014 Analysis & 2020 Forecasts on Market data, Statics, Development Now at ReportsnReports.com 3Bio?Techne Announces Appointment Of Robert Gavin As Leader Of New Protein Platforms Division 2Bio?Techne Announces Appointment Of Robert Gavin As Leader Of New Protein Platforms Division 3Roka Bioscience to Present at the Piper Jaffray Healthcare Conference 2
... Signature pitchman, Billy Mays ENDORSES AND USES What Odor?® , ... Phoenix, ... to announce the launch of a massive marketing campaign for their flagship ... ,Nelson Grist, Founder and CEO of For The Earth Corp, anxiously awaits ...
... - BioMS Medical Corp (TSX: MS), a leading developer ... effective on March 16, 2009 the exercise term of ... holder to purchase one Class A common share at ... from March 23, 2009 to December 31, 2009. , ...
... invivodata, inc., ( http://www.invivodata.com ) the industry ... solutions and services for global clinical research, ... at upcoming international events to be delivered ... and regulatory consulting division, PRO Consulting(R) ( ...
Cached Biology Technology:Billy Mays Endorses New Odor Eliminating Product, What Odor? 2BioMS Medical warrant extension 2invivodata and PRO Consulting to Present at International Industry Events 2invivodata and PRO Consulting to Present at International Industry Events 3
(Date:11/6/2014)... occur because of metastasis, yet progress in preventing ... , "It,s been particularly challenging to design drugs ... fellow in systems biology at Harvard Medical School. ... after they,ve already metastasized." , Gujral and colleagues ... help researchers better understand how metastasis begins. Their ...
(Date:11/5/2014)... career to understanding the Earth,s climate and communicating ... deputy director and regional climatologist for the Western ... Redmond has more than three decades of experience ... data to the general public. , At its ... December 15-19, 2014 the American Geophysical Union (AGU) ...
(Date:11/4/2014)... PA) – Temple University School of Medicine announced ... winner, an initiative funded by the Bill & ... Professor of Obstetrics, Gynecology and Reproductive Sciences; and ... Temple University School of Medicine will pursue an ... "Maternal Blood Biomarkers to Assess Fetal Neurodevelopment.", Grand ...
Breaking Biology News(10 mins):Migration negation 2Migration negation 3Nevada climatologist to be honored at AGU Fall Meeting for lifetime of public outreach 2Temple University School of Medicine receives Grand Challenges Explorations grant 2
... Université Laval biologist Connie Lovejoy has discovered new ... findings are reported in the January 12 edition ... discovered a new group of microscopic organisms, which ... extremely small size, measured in millionths of a ...
... to protect against human papillomavirus, has moved from scientific ... to make progress. While HPV vaccine efforts had the ... researchers are developing vaccines for cancers that are not ... system into attacking cancerous cells. Today at the ...
... from West Africa were cracking nuts with stone tools ... The result suggests chimpanzees developed this behaviour on their ... trait inherited from our common ancestor. Julio Mercader, Christophe ... site in Cote d'Ivoire, the only known prehistoric chimpanzee ...
Cached Biology News:Cancer vaccines -- Taking a jab at cancer by stimulating the immune system 2Cancer vaccines -- Taking a jab at cancer by stimulating the immune system 3Cancer vaccines -- Taking a jab at cancer by stimulating the immune system 4Cancer vaccines -- Taking a jab at cancer by stimulating the immune system 5The chimpanzee stone age 2
Human CCL26/Eotaxin-3 MAb (Clone 115002)...
Normal sheep serum collected from healthy normal sheep...
Agarose-ME, 500 g. Suitable for immunoelectrophoresis.Gel point (1.5%): 36 1.5 C, EEO (-mr): 0.16-0.19, Gel strength (1%): > 1000 g/cm2. Category: Nucleotides & Enzymes & Biochemicals, Ultrapure Bio...
Pooled normal sreum with 0.1% sodium azide as preservative...
Biology Products: