🧬

Gene Therapy

Genetic medicines including gene replacement, gene editing (CRISPR), and RNA-based therapeutics.

Companies

Pipeline Drugs

Key People

Gene Therapy Pipeline (68 drugs)

Commercial: 36Platform/Service: 6Approved/Commercial: 5Phase 1/2: 4Phase 3: 2Pre-clinical: 2Phase 2: 2Phase 1: 2Preclinical/Phase 1: 2Preclinical: 2Development: 1Clinical: 1NDA/BLA Filed: 1Phase 2/3: 1Service: 1

Drug	Company	Indication	Phase	Status
Treprostinil (Remodulin)	United Therapeutics	Pulmonary Arterial Hypertension	Commercial	Approved
Treprostinil (Tyvaso)	United Therapeutics	PAH and PH-ILD	Commercial	Approved
Treprostinil (Orenitram)	United Therapeutics	Pulmonary Arterial Hypertension	Commercial	Approved
Selexipag (Uptravi)	United Therapeutics	Pulmonary Arterial Hypertension	Commercial	Approved
Ralinepag	United Therapeutics	Pulmonary Arterial Hypertension	Phase 3	Active
Aurora-GT	United Therapeutics	AADC Deficiency	Phase 1/2	Active
Xenotransplant Program	United Therapeutics	Organ Transplantation	Pre-clinical	Active
RemUnity Pump	United Therapeutics	PAH Drug Delivery	Development	Active
VOXZOGO (vosoritide)	BioMarin Pharmaceutical	Achondroplasia	Commercial	Active
PALYNZIQ (pegvaliase-pqpz)	BioMarin Pharmaceutical	Phenylketonuria (PKU)	Commercial	Active - recently approved for adolescents
Enzyme Replacement Therapy	BioMarin Pharmaceutical	Mucopolysaccharidosis I	Commercial	Active
Gene Therapy	BioMarin Pharmaceutical	Hemophilia A	Commercial	Active
Metabolic Therapy	BioMarin Pharmaceutical	Rare Metabolic Condition	Commercial	Active
ENPP1 deficiency program	BioMarin Pharmaceutical	ENPP1 deficiency/GACI	Clinical	Active
VYJUVEK	Krystal Biotech	Dystrophic Epidermolysis Bullosa	Commercial	FDA Approved - First redosable gene therapy
NucleoCounter® NC-203™	ChemoMetec	Cell Counting / GMP Workflows	Commercial	Marketed
NucleoCounter® NC-202™	ChemoMetec	Therapeutic Antibody Production / GMP Workflows	Commercial	Marketed
NucleoCounter® NC-200™	ChemoMetec	Cell Counting / Research	Commercial	Marketed
XcytoMatic® 30	ChemoMetec	Bioprocessing / Automated Labs	Commercial	Marketed
XcytoMatic® 40	ChemoMetec	High-Throughput Bioprocessing / Cell Therapy	Commercial	Marketed
NucleoCounter® NC-3000™	ChemoMetec	Advanced Image Cytometry / Custom Assays	Commercial	Marketed
Via2-Cassette™	ChemoMetec	Consumable for Cell Counting	Commercial	Marketed
Via3-Cassette™	ChemoMetec	Consumable for Cell Counting	Commercial	Marketed
opDNA® Platform	4basebio	DNA template for mRNA therapeutics/vaccines (partner program)	Phase 1/2	Active - Patient dosing initiated by partner
Non-viral, cell-targeting nucleic acid delivery platform	4basebio	Targeted delivery for gene therapy	Pre-clinical	Internal R&D
ZYCUBO® (copper histidinate)	Fortress Biotech	Menkes Disease	Approved/Commercial	Approved
Emrosi™ (Minocycline Hydrochloride ER Capsules, 40 mg)	Fortress Biotech	Rosacea	Approved/Commercial	Approved
Qbrexza®	Fortress Biotech	Primary Axillary Hyperhidrosis	Approved/Commercial	Approved
Targadox® (doxycycline hyclate)	Fortress Biotech	Severe Acne	Approved/Commercial	Approved
UNLOXCYT™ (cosibelimab-ipdl)	Fortress Biotech	Cutaneous Squamous Cell Carcinoma (cSCC)	Approved/Commercial	Approved (Royalty Asset)
CUTX-101	Fortress Biotech	Menkes Disease	NDA/BLA Filed	NDA Resubmission Accepted
CAEL-101	Fortress Biotech	Light Chain (AL) Amyloidosis	Phase 3	Active
BAER-101 (AZD7325)	Fortress Biotech	CNS Disorders	Phase 2	Active
MB-101 (IL-13Rα2 CAR T)	Fortress Biotech	Cancer (e.g., Glioblastoma)	Phase 1/2	Active
MB-102 (CD123 CAR T)	Fortress Biotech	Cancer (e.g., AML, BPDCN)	Phase 1/2	Active
MB-103 (HER2 CAR T)	Fortress Biotech	Cancer (e.g., Glioblastoma, Breast)	Phase 1	Active
MB-104 (CS1 CAR T)	Fortress Biotech	Cancer (Multiple Myeloma)	Preclinical/Phase 1	Active
MB-105 (PSCA CAR T)	Fortress Biotech	Cancer (Prostate, Pancreatic)	Preclinical/Phase 1	Active
MB-108 (C134 oncolytic virus)	Fortress Biotech	Glioblastoma Multiforme	Phase 1	Active
Triplex / ConVax	Fortress Biotech	Cytomegalovirus (CMV) Prevention	Phase 2/3	Active
CEVA-101 (pediatric & adult)	Fortress Biotech	Traumatic Brain Injury (TBI)	Phase 2	Active
CEVA-102	Fortress Biotech	Traumatic Brain Injury (TBI)	Preclinical	Active
ATX-04	Fortress Biotech	Pompe Disease	Preclinical	Active
Gibson SOLA Platform	Telesis Bio	AI/ML Model Training for Biology	Platform/Service	Commercial
BioXp System	Telesis Bio	Automated Gene Synthesis & Library Prep	Platform/Service	Commercial
Scanning & Combinatorial Library Services	Telesis Bio	Gene Synthesis Service	Service	Commercial
Cell & Gene Therapy Synthetic Promoters	Chromatin Bioscience	Cell-selective gene expression for therapeutic systems	Platform/Service	Active R&D and Client Services
Biologics Manufacturing Promoters	Chromatin Bioscience	Increased protein yield in bioproduction	Platform/Service	Active R&D and Client Services
Industrial Biotechnology Promoters	Chromatin Bioscience	Metabolic engineering in chassis organisms	Platform/Service	Active R&D and Client Services
Agricultural Biotechnology Promoters	Chromatin Bioscience	Trait expression in crops (tissue-specific, stress-responsive)	Platform/Service	Active R&D and Client Services