Rare Diseases

 

15 Organization(s)

Aeglea BioTherapeutics

Business Area(s): 
Description: 

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

Beloteca_

Business Area(s): 
Description: 

Generics & 505(b)(2) drugs

Belrose Pharma

Business Area(s): 
Description: 

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

Business Area(s): 
Description: 

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 ...

Bioneur

Business Area(s): 
Description: 

Orphan-Drug Consultants

Bionure

Business Area(s): 
Description: 

SGK agonists for the treatment of rare, ophthalmological diseases

Citius Pharmaceuticals

Business Area(s): 
Description: 

505(b)(2) drugs

Clementia Pharmaceuticals

Business Area(s): 
Description: 

Fibrodysplasia ossificans progressiva treatments

Corbus Pharmaceuticals

Business Area(s): 
Description: 

Orphan inflammatory-fibrotic diseases

Cot_ Orphan

Business Area(s): 
Description: 

Orphan regulatory affairs, risk evaluation, and streamlined project management

Exodos Life Science Partners

Business Area(s): 
Description: 

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

Lumos Pharma

Business Area(s): 
Description: 

Rare disease treatment

Sigma Tau

Business Area(s): 
Description: 

Orphan Disease Treatments

Valerion Therapeutics

Business Area(s): 
Description: 

Biotherapeutics for orphan genetic diseases

Vtesse

Business Area(s): 
Description: 

Orphan Indicated Small Molecule