Rare Diseases Companies and Organizations

 

29 Organization(s)

Aeglea BioTherapeutics

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Aeglea is committed to developing engineered human enzymes for treating rare diseases and cancers associated with abnormal amino acid metabolism.

Agios Pharmaceuticals

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Agios works on cellular metabolism and precision medicine across three major focus areas: cancer metabolism, rare genetic diseases and metabolic immuno-oncology. 

Akcea Therapeutics

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Akcea is developing treatments for unaddressed or under-addressed serious and rare diseases with leading-edge, RNA-targeted medicines. 

Beloteca_

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Generics & 505(b)(2) drugs

Belrose Pharma

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Belrose Pharma focuses on rare diseases and uses its proprietary “releasable linker” conjugation technology and other techniques to improve the clinical efficacy of both novel therapeutics and ...

BioBlast Pharma

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BioBlast Pharma manufactures Trehalose, a therapeutic to potentially treat a number of devastating PolyA/PolyQ diseases. Trehalose acts as a chemical chaperone to protects against pathological ...

BioCryst Pharmaceuticals

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BioCryst develops drugs to treat rare diseases.

Bioneur

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Orphan-Drug Consultants

Bionure

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SGK agonists for the treatment of rare, ophthalmological diseases

BioXcel Therapeutics

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BioXcel is a biopharmaceutical company that uses artificial intelligence and big data analytics to develop medicines in immuno-oncology, neuroscience, and rare diseases with high unmet need

BridgeBio

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Bridgebio is a collection of individuals focused on discovering and developing drugs for patients with grievous genetic diseases.  This is done by:

Finding the right starting points to ...

Centogene

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CENTOGENE develops tests for the genetic diagnostics for rare hereditary diseases 

Citius Pharmaceuticals

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505(b)(2) drugs

Clementia Pharmaceuticals

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Fibrodysplasia ossificans progressiva treatments

Emmaus

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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 disease ...

Exodos Life Science Partners

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Early-stage research on 505(b)(2) products

Leadiant Biosciences

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Leadiant Biosciences is a research-based pharmaceutical company that dedicates considerable scientific and financial resources to the research, development, and distribution of novel and effective ...

Lumos Pharma

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Rare disease treatment

Qrativ

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Qrativ uses the power of combining the nference artificial intelligence (AI)-driven knowledge synthesis platform with Mayo Clinic’s medical expertise and clinical ...

Rare Disease Therapeutics

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Rare Diseases

RDMD

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Rare diseases. 

Rhythm Pharma

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Rhythm is a biopharmaceutical company focused on developing and commercializing peptide therapeutics for the treatment of rare genetic deficiencies that result in life-threatening metabolic ...

Shire (Takeda)

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Shire is a biotechnology company focused on serving people with rare diseases.

Sigma Tau

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Orphan Disease Treatments

Soleno Therapeutics

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Developing therapeutics for the treatment of rare diseases.

Therachon

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Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases that currently have no available treatments. The company’s lead pipeline candidate, TA-46, is ...

Tremeau Pharmaceuticals

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Tremeau Pharmaceuticals is a Massachusetts based privately funded clinical stage company focused on commercializing non-opioid pain therapies targeting serious rare diseases impacted by the ...

Valerion Therapeutics

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Biotherapeutics for orphan genetic diseases

Vtesse

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Description: 
Orphan Indicated Small Molecule
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