Gene Therapy for ADA-SCID
Adenosine Deaminase Deficient SCID
PreclinicalIND-enabling
Key Facts
Indication
Adenosine Deaminase Deficient SCID
Phase
Preclinical
Status
IND-enabling
Company
About United Therapeutics
Founded on a personal mission to treat pulmonary arterial hypertension, United Therapeutics has grown into a diversified, multi-platform biotech leader with a $26B+ market cap. The company successfully commercializes a portfolio of PAH therapies while aggressively pursuing its 'Mozart' moonshot to create an unlimited supply of transplantable organs. This dual strategy balances near-term financial strength from its rare disease franchise with transformative, long-term value creation in organ manufacturing and regenerative medicine.
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