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Rare Disease Suppliers & Manufacturers

89 companies found

Population Bio, Inc. a Precision Medicine Company

Population Bio, Inc. a Precision Medicine Company

based inMelville, NEW YORK (USA)
Population Bio, Inc. is a privately held precision medicine company that is empowering pharmaceutical companies to develop targeted therapies and companion diagnostics faster and more cost effectively. We are accelerating the delivery of precision ...
CONTACT SUPPLIER

Certara

Certara

based inPrinceton, NEW JERSEY (USA)
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. ...
CONTACT SUPPLIER

Oncodesign

Oncodesign

based inDijon, FRANCE
Oncodesign is a biopharmaceutical company that offers you powerful technologies and tools to guide you through the development of your drug, from medicinal chemistry to drug candidate. Our therapeutic areas are expanding. In addition to oncology, ...
Oncodesign - Model Chi-Mice - Humanized Technology

Oncodesign - Model Chi-Mice - Humanized Technology

This is the best tool you can use to evaluate new therapeutic approaches in conditions that are as close as possible to a clinical situation: ...
CONTACT SUPPLIER

Taiwan Liposome Company, Ltd. (TLC)

Taiwan Liposome Company, Ltd. (TLC)

based inNangang Distric, TAIWAN
We are a clinical-stage specialty pharmaceutical company dedicated to the development and commercialization of best-in-class novel nanomedicines that combine our proprietary lipid-assembled drug delivery platform with approved active pharmaceutical ...
CONTACT SUPPLIER

Confo Therapeutics

Confo Therapeutics

based inGhent/Zwijnaarde, BELGIUM
Confo Therapeutics’ unparalleled technology stabilizes functional conformations of GPCRs (G protein-coupled receptors) to uncover a wide range of previously inaccessible GPCRs as drug targets. This technology platform combined with the pharmacologic ...
CONTACT SUPPLIER

Evox Therapeutics Limited

Evox Therapeutics Limited

based inOxford, UNITED KINGDOM
Evox Therapeutics is a privately held, Oxford-based biotechnology company focused on improving the natural delivery capabilities of exosomes, and developing an entirely new class of therapeutics. Backed by leading venture capital groups and ...
Evox - Model Type C - Lysosomal Storage Disorder

Evox - Model Type C - Lysosomal Storage Disorder

Niemann-Pick C disease (NPC) is a lysosomal storage disorder which affects the brain and multiple other ...
CONTACT SUPPLIER

RareStone Group

RareStone Group

based inShanghai, CHINA
RareStone Group is dedicated to building the first rare disease ecosystem in China to improve the lives of patients and families impacted by rare diseases, by bringing the best-in-the-world therapeutic solutions and comprehensive and sustainable ...
Rare Liver and Renal Disease Services

Rare Liver and Renal Disease Services

Chronic kidney disease (CKD) is a non-communicable disease usually caused by diabetes and hypertension. The prevalence of chronic kidney disease in China is reported to be 10.8%, and is predicted to ...
CONTACT SUPPLIER

Curome BioSciences

Curome BioSciences

based inheongju-si, SOUTH KOREA
Curome Biosciences is an innovative R&D company developing therapies to treat mitochondrial dysfunction-related diseases with high unmet medical needs. Curome Biosciences wants to provide solutions for treatment of diseases with high unmet medical ...
Researching Services

Researching Services

Mitochondria are an essential cellular organelle that is responsible for the generation of energy (known as the ‘powerhouse of the cell’). Mitochondrial dysfunction is related with numerous diseases. Curome Biosciences ...
CONTACT SUPPLIER

Vertex Pharmaceuticals

Vertex Pharmaceuticals

based inBoston, MASSACHUSETTS (USA)
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — ...
CONTACT SUPPLIER

SoftGenetics, LLC.

SoftGenetics, LLC.

based inState College, PENNSYLVANIA (USA)
SoftGenetics, LLC specializes in the development of genetic analysis tools for both research and diagnostic ...
NextGENe - Next Generation Sequencing Software

NextGENe - Next Generation Sequencing Software

NextGENe software is the perfect analytical partner for the analysis of desktop sequencing data produced by Illumina® iSeq, Miniseq, MiSeq, NextSeq, HiSeq, and NovaSeq systems, Ion Torrent Ion GeneStudio S5, PGM, and Proton systems as well as ...
CONTACT SUPPLIER

Horizon Therapeutics plc

Horizon Therapeutics plc

based inSaint Kevin`s, IRELAND
At Horizon, we believe science and compassion must work together to transform lives. Our mission to deliver medicines for rare, autoimmune and severe inflammatory diseases and provide compassionate support comes from our strong and simple philosophy ...
CONTACT SUPPLIER

Oligomerix, Inc.

Oligomerix, Inc.

based inWhite Plains, NEW YORK (USA)
Oligomerix is a biotechnology company focused on developing disease-modifying and treatment therapeutics for neurodegenerative diseases characterized by aberrant tau protein ranging from rare tauopathies such as progressive supranuclear palsy ...
Oligomerix - Model OLX07010 - Rare Tauopathies

Oligomerix - Model OLX07010 - Rare Tauopathies

There exist numerous rare diseases that tau dysfunction is associated with known as tauopathies. While Alzheimer’s is the largest tauopathy, other tauopathies are rare ...
CONTACT SUPPLIER

CENTOGENE N.V.

CENTOGENE N.V.

based inRostock, GERMANY
Since 2006, CENTOGENE has helped physicians diagnose thousands of rare disease patients around the world. A quick and accurate diagnosis can end an odyssey of medical tests and visits to multiple medical specialists. It also helps physicians better ...
CentoCloud - Version SaaS - Cloud-Based Software

CentoCloud - Version SaaS - Cloud-Based Software

Analyze, interpret, and report genomic variants with our decentralized diagnostic solution – no matter where you are located. CentoCloud is a cloud-based Software as a Service (SaaS) platform specifically designed for the rapid bioinformatic ...
CONTACT SUPPLIER

Andelyn Biosciences

Andelyn Biosciences

based inColumbus, OHIO (USA)
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 ...
Clinical Manufacturing Services

Clinical Manufacturing Services

Andelyn Biosciences provides clients with a broad range of viral vector manufacturing platforms and solutions, including AAV and Lentiviral adherent and suspension systems along with plasmid manufacturing. We support our clients throughout every ...
CONTACT SUPPLIER

Lupagen Inc.

Lupagen Inc.

based inDallas, TEXAS (USA)
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 ...
Lupagen Side CAR-T - Delivery System for Cell Therapy and Safety & Clinical

Lupagen Side CAR-T - Delivery System for Cell Therapy and Safety & Clinical

Lupagen avoids the cost & complexity of cell therapy and the safety & clinical challenges of viral-based gene therapy by a simple patient-connected gene delivery procedure done at the bedside. Our Side CAR-T® delivery system, a ...
CONTACT SUPPLIER

Amicus Therapeutics, Inc.

Amicus Therapeutics, Inc.

based inPhiladelphia, PENNSYLVANIA (USA)
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 ...
CONTACT SUPPLIER

Precision NanoSystems

Precision NanoSystems

based inVancouver, BRITISH COLUMBIA (CANADA)
Precision NanoSystems is a global leader in technologies, solutions and services for the development of non-viral delivery of genomic medicines, including mRNA vaccines and therapeutics. We work with the world’s leading drug developers to understand ...
CONTACT SUPPLIER

TQ Therapeutics GmbH

based inPlanegg, GERMANY
TQ Therapeutics GmbH is pioneering a novel approach to cell and gene therapy with its TQx platform, which transforms the production of cellular therapies into a rapid, automated process that occurs at the patient's side. This method leverages ...
TQx - Modular Target Cell-specific Modification Platform Technology

TQx - Modular Target Cell-specific Modification Platform Technology

TQx is developing a modular target cell-specific modification platform including relevant technology components for the generation of gene modified cell therapy ...
CONTACT SUPPLIER

Chimeron Bio

Chimeron Bio

based inPhiladelphia, PENNSYLVANIA (USA)
Chimeron Bio is building a portfolio of self-amplifying RNA therapeutics and vaccines to bring more effective gene therapies and personalized medicines to patients. We are developing a compelling portfolio of innovative and personalized saRNA-based ...
CONTACT SUPPLIER

MitoImmune Therapeutics Inc.

MitoImmune Therapeutics Inc.

based inGangnam-gu, SOUTH KOREA
MitoImmune Therapeutics Inc. is a drug discovery and development biotech company that harnesses the power of mitochondria and its proprietary technology targeting mitochondria as the source for developing new potential breakthrough therapeutics. Our ...
CONTACT SUPPLIER
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