Browse Companies by Terms

Copy and paste the "+" delimited list of TIDs into the "Arguments" field of the BTC Company Activity Group page.

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Selected Terms: id:281 Rare Diseases (60)

60 Organization(s) Selected

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3billion

Description: 

Developing genetic tests to detect rare diseases.

Aeglea BioTherapeutics

Description: 

Aeglea is committed to developing engineered human enzymes for treating ...

Agios Pharmaceuticals

Description: 

Agios works on cellular metabolism and precision medicine across three ...

AGTC

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Description: 

AGTC is developing genetic therapies to treat patients with rare ...

Akcea Therapeutics

Business Area(s): 
Description: 

Akcea is developing treatments for unaddressed or under-addressed serious and ...

Alexion

Business Area(s): 
Description: 

Alexion is a global biopharmaceutical company focused on developing life-changing ...

Alexion Pharmaceuticals

Description: 

The company is developing treatments for rare diseases.

COVID-19: ...

Allievex

Business Area(s): 
Description: 

Allievex is developing tralesinidase alfa for the treatment of children ...

Amicus Therapeutics

Description: 

Amicus Therapeutics is a global, biotech company focused on discovering, ...

Amryt Pharma

Business Area(s): 
Description: 

Developing treatments for Epidermolysis Bullosa, a group of rare and ...

Apic Bio

Description: 

Apic Bio develops gene therapy for patients with genetic ...

Attune Pharmaceuticals

Description: 

Attune Pharmaceuticals is a private biotechnology company focused on discovering ...

Azafaros

Description: 

Azafaros was founded in 2018 to address rare genetic metabolic ...

Beloteca_

Business Area(s): 
Description: 

Generics & 505(b)(2) drugs

BioCryst Pharmaceuticals

Description: 

BioCryst develops drugs to treat rare diseases. Galidesivir (BCX4430), an ...

BioMarin Pharmaceuticals

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Description: 

BioMarin focuses on developing therapeutics for patients who live with ...

Bioneur

Business Area(s): 
Description: 
Orphan-Drug Consultants

Bionure

Business Area(s): 
Description: 

SGK agonists for the treatment of rare, ophthalmological diseases

BioXcel Therapeutics

Description: 

BioXcel is a biopharmaceutical company that uses artificial intelligence and ...

BridgeBio

Business Area(s): 
Description: 

Bridgebio is a collection of individuals focused on discovering and ...

Castle Creek Pharma

Business Area(s): 
Description: 

Castle Creek Pharma is a biopharmaceutical company with a focus ...

Centogene

Description: 

CENTOGENE develops tests for the genetic diagnostics for rare hereditary ...

Chiesi Farmaceutici

Description: 

Chiesi Farmaceutici S.p.A. is an Italian family controlled global ...

Citius Pharmaceuticals

Business Area(s): 
Description: 
505(b)(2) drugs

Clementia Pharmaceuticals

Description: 

Fibrodysplasia ossificans progressiva treatments

CureVac

Description: 

CureVac is a biopharmaceutical company headquartered in Tübingen, Germany, that ...

Cyclerion

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Description: 

Cyclerion was founded with the goal of unlocking the full ...

Emalex Biosciences

Business Area(s): 
Description: 

Emalex Biosciences develops treatments for central nervous system movement disorders ...

Emmaus

Description: 

Emmaus Life Sciences, Inc. is a biopharmaceutical company engaged in ...

Exodos Life Science Partners

Business Area(s): 
Description: 
Early-stage research on 505(b)(2) products

GeneDx

Description: 

GeneDx provides genomics testing with expertise in rare and ultra-rare ...

Harmony Biosciences

Description: 

Harmony Biosciences, LLC , is a private biopharmaceutical company ...

Healx

Description: 

Developed an AI platform for rare disease drug discovery.

Homology Medicines

Business Area(s): 
Description: 

Homology Medicines is working to use gene editing and gene ...

HotSpot Therapeutics

Description: 

Developing a drug discovery platform for allosteric medicines.

Ipsen

Description: 

Ipsen develops and markets medications used in oncology, neuroscience and ...

Kamada

Description: 

They focus on plasma-derived protein therapeutics for treatment of individuals ...

Leadiant Biosciences

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Description: 

Leadiant Biosciences is a research-based pharmaceutical company that dedicates considerable ...

Levo Therapeutics

Business Area(s): 
Description: 

Levo Therapeutics is working to understanding the pathophysiology of Prader-Willi ...

Lumos Pharma

Business Area(s): 
Description: 
Rare disease treatment
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