Companies in the Rare disease industry
Name | Description | Industry | Official Website | Location | ||
|---|---|---|---|---|---|---|
Sanofi is a french pharmaceutical company founded in 2004 by Madhukar Reddy. | ||||||
A biotechnology startup company that develops messenger RNA based therapeutics to treat diseases. | ||||||
A biopharmaceutical company focused on discovery and development of small-molecule drugs to treat genetic and rare diseases. | ||||||
Amicus Therapuetics is a biotechnology company developing therapies to cure rare diseases. | ||||||
Recursion Pharmaceuticals is a biotechnology and data science company based in Salt Lake City, Utah, founded in 2013, that combines biology with artificial intelligence for drug discovery. Using human cell models of diseases, Recursion captures microscopic images to build biological datasets and computational techniques identify disease-associated. | ||||||
OrphAI Therapeutics is a biotechnology research company based in Guilford, Connecticut that develops precision therapeutics technology designed to treat cancer and rare diseases. | ||||||
LabCentral Inc. is a shared laboratory facility for biotechnology startups founded in 2012 by Peter Parker. | ||||||
A company developing gene therapies for the treatment of genetic diseases and cancer. | ||||||
A company focused on development of products to treat central nervous system disorders, pain and rare diseases | ||||||
A biopharmaceutical company focused on developing treatments for urea cycle disorders and other rare diseases | ||||||
Rallybio is a clinical-stage biotechnology company built around a team of seasoned industry leaders. | ||||||
A genetic medicines company in Massachusetts that focuses on patients suffering from rare genetic diseases. | ||||||
AllStripes is a U.S-based company offering a rare disease medical information database. | ||||||
Tevard Biosciences is a Cambridge, Massachusetts-based biotechnology company developing gene therapy platforms to target rare and unmet diseases. | ||||||
Healx is a biotechnology therapeutics company for rare diseases founded in 2014 by Tim Guilliams. | ||||||
Dicerna Pharmaceuticals is a company that focuses on the discovery and development of treatments for inherited diseases. | ||||||
Hemab Therapeutics is pioneering the first preventative treatments for rare bleeding and thrombotic disorders to ensure no patient is ever left behind | ||||||
MyoKardia is a company developing targeted therapies for cardiocavscular diseases founded in 2012 by Leslie Leinwand. | ||||||
Disc Medicine is a medical research company that develops treatments for hematologic diseases. | ||||||
Passage Bio, Inc is a Philadelphia-based biotechnology company developing genetics-based medicines. | ||||||
We are dedicated to improving the lives of patients suffering from hematological malignancies by delivering an innovative immune modulator to enhance the curative potential of allogeneic Hematopoietic Stem Cell Transplantation (HSCT). | ||||||
Imara is a Cambridge, Massachusetts-based biotechnology company. | ||||||
Entrada Therapeutics is a biologics delivered to cytosol founded in 2016. | ||||||
Dyne Therapeutics is an Antibody-Oligonucleotide Treatments for Muscle Diseases founded in 2018. | ||||||
Wilson Therapeutics is an every Day We Change Lives founded in 1992. |
