Rare Diseases Suppliers In Usa

AGC Biologics offers deep industry expertise and uniquely customized services for the scale-up and cGMP manufacture of protein-based therapeutics (using mammalian and microbial systems), mRNA, pDNA, viral vector and cell therapy products, from ...

Pluristem Therapeutics Inc. is a clinical-stage regenerative medicine company using placental cells and a unique, proprietary, three-dimensional (3D) technology platform to develop cell therapies for conditions such as inflammation, muscle injuries, ...

Emmaus Life Sciences, Inc. is a biopharmaceutical company engaged in the discovery, development and commercialization of innovative treatments and therapies primarily for rare and orphan diseases. Our initial efforts have focused on treatments for ...

Established in 1997, BioMarin is a world leader in developing and commercializing first- or best-in-class therapies for rare genetic diseases. We take pride in going where the science leads us, pioneering breakthrough treatments for debilitating and ...

Lupagen is a privately held early stage biotech company developing first-in-class bedside enabled cell & gene therapies (CAR-T cell, gene editing and immunotherapy products) based on their patent-pending bedside point-of-care C and GT production and ...

Our company is a globally operating service provider and one of the world leaders in the development and delivery of advanced drug therapies for rare diseases. We not only provide global customers with a wide range of services for rare disease ...

HemoShear Therapeutics is a clinical stage company developing novel treatments for patients with rare diseases. We are implementing a new vision for how advanced biological and computational models can accelerate the discovery of innovative ...

The drug discovery and development process is long and expensive with more failures than successes. At Certara, we anticipate and address your critical drug discovery and development risks and decisions using biosimulation, technology and services. ...

Andelyn Biosciences is a biopharmaceutical CDMO Pioneering Solutions That Turn Hope into Reality™. With 20+ years of experience manufacturing viral vectors, Andelyn’s scientific expertise for development and characterization has led to GMP material ...

MorphoSys is a commercial-stage biopharmaceutical company dedicated to the discovery, development, and delivery of innovative cancer medicines. At MorphoSys, our ambition is to redefine how cancer is treated. As a commercial-stage biopharmaceutical ...

People living with rare and devastating diseases are our Guiding Star. We believe it is our responsibility to listen to, understand, and change the lives of patients and those who work tirelessly to help them. Our mission is to transform the lives ...

Protara is committed to identifying and advancing transformative therapies for people with cancer and rare diseases. The Protara team prioritizes creativity, diverse perspectives and tenacity to expedite our goal of bringing life-changing therapies ...

MedGenome Inc. is a global leader in personalized medicine with unique genomic solutions in immuno-oncology, diabetes, ophthalmology, cardiology and other rare diseases. Founded in 2013, MedGenome currently has a Next Generation Sequencing (NGS) ...

BTG Specialty Pharmaceuticals provides rescue medicines typically used in emergency rooms and intensive care units to treat patients for whom there are limited treatment options. We are dedicated to delivering quality medicines that make a real ...

We are a biotechnology company at the forefront of advanced therapies to treat a range of devastating rare diseases. Amicus Therapeutics (Nasdaq:FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and ...

Palvella Therapeutics was founded to serve individuals suffering from rare diseases by developing safe and efficacious therapies that will dramatically enhance their quality of life. We focus on developing therapies that specifically target the root ...

Enhancing global compliance, creating a world where quality and compliance professionals, regulators, and government agencies come together to help the world comply with the intent and the spirit of laws, policies and mandates, ensuring continuous ...

Nutcracker Therapeutics is an early-stage company that is pioneering a revolutionary therapeutic development and manufacturing platform designed to enable the advancement of RNA-based treatments for as many diseases as possible, as fast as possible. ...

The Jeeva eClinical Cloud was developed by researchers with empathy who listen and learn to help clinical researchers, hospital sites, academic medical centers, CROs, and biopharmaceutical sponsors accelerate patient enrollment by 3x faster. Jeeva ...

Avero Diagnostics is dedicated to making a positive impact on the lives of patients and the physicians who treat them. We bring a patient-centered approach to every aspect of our organization, including our laboratory and research facility. It’s ...