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Gene Therapy

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

Companies

Pipeline Drugs

Key People

144

Gene Therapy Pipeline (60 drugs)

Commercial: 23Preclinical: 5Platform/Service: 4Development: 4Pre-clinical: 3Phase III: 3Pre-clinical/Research: 2Phase I/II: 2Phase I: 2Phase 2: 1Approved (EU): 1Preclinical/Clinical: 1Commercial (RUO): 1Class II Medical Device: 1Design & Development: 1Phase 1: 1Research: 1Unknown: 1Phase IIb/III: 1Phase 1/2: 1Phase II: 1

Drug	Company	Indication	Phase	Status
Ixoberogene soroparvovec (ADVM-022)	Adverum Biotechnologies	Wet Age-Related Macular Degeneration (wet AMD)	Phase 2	Active
Imlifidase	Hansa Biopharma	Desensitization in Kidney Transplantation	Approved (EU)	Commercial
HNSA-5487	Hansa Biopharma	Next-Generation IgG-cleaving Enzyme	Preclinical/Clinical	Active
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
Cell Freeze® Cryogenic Storage Containers	Charter Medical	Storage/Preservation of Hematopoietic Cells & Biologics	Commercial	Marketed
EXP-Pak™ Cell Expansion Containers	Charter Medical	Expansion/Culture of Non-adherent Cells	Commercial	Marketed
Freeze-Pak™ with FP-FLEX™	Charter Medical	Cryogenic Storage and Transport	Commercial	Marketed
Advect® Fluid Transfer Sets	Charter Medical	Sterile Fluid Transfer in Bioprocessing	Commercial	Marketed
Bio-Pak® Small Volume Bio-Containers	Charter Medical	Small-Volume Fluid Handling	Commercial	Marketed
AccuStrain™ 40μm & 150μm Filters	Charter Medical	Filtration of Microaggregates from Blood/Products	Commercial	Marketed
Fluid Transfer Bag Systems (DEHP-free)	Charter Medical	Sterile Fluid Transfer & Storage	Commercial	Marketed
Blood Transfer Bags & Aliquot Systems	Charter Medical	Blood Component Collection/Processing/Storage	Commercial	Marketed
Neonatal Syringe Set with 150μm Filter	Charter Medical	Preparation/Delivery of Filtered Blood for Neonates	Commercial	Marketed
Neonatal Syringe Set with 40μm Filter	Charter Medical	Removal of Microaggregates from Blood for Neonates	Commercial	Marketed
Plasma Processing Bags	Charter Medical	Apheresis Plasma Aliquot Processing	Commercial	Marketed
Plasma/Fluid Transfer Sets	Charter Medical	Sampling of Blood/Blood Components	Commercial	Marketed
Platelet Sampling Sets	Charter Medical	Safe Transfer of Platelet Samples	Commercial	Marketed
Pooling Harness Sets	Charter Medical	Multi-lead Harness for Component Processing	Commercial	Marketed
Freeze-Pak™ STS	Charter Medical	Frozen Storage and Transport	Commercial	Marketed
Freeze-Pak™ Bio-Containers	Charter Medical	General Bioprocessing Storage	Commercial	Marketed
Cell Expansion Containers	Charter Medical	Cell Expansion Applications	Commercial	Marketed
CryoProtect™ Bio-Containers	Charter Medical	Cryopreservation Applications	Commercial	Marketed
Salt-Active Nucleases (SAN)	ArcticZymes Technologies	Downstream Processing of Viral Vectors, Vaccines, Biologics	Commercial	Active
COD UNG (Uracil-DNA Glycosylase)	ArcticZymes Technologies	Prevention of PCR carryover contamination	Commercial	Active
GMP-Grade Nucleases Portfolio	ArcticZymes Technologies	Host Cell DNA/RNA removal in cGMP manufacturing	Commercial	Active
Specialty Enzymes for Diagnostics	ArcticZymes Technologies	Components for IVD assay development	Commercial	Active
AE1-BHK Cell Line	Agathos Biologics	Biomanufacturing Platform	Development	Active
AAV Reference Standard	Agathos Biologics	Analytics/QC Tool	Development	Active
Therapeutic Programs	Agathos Biologics	Undisclosed	Pre-clinical	Active
AAV Gene Therapy QC	NanoMosaic	AAV capsid and transgene characterization	Commercial (RUO)	Active
Blood pTau Detection	NanoMosaic	Alzheimer's Disease and Related Dementias	Development	Active
AxiLona EL-100 Microarray Analyzer	Axbio	Multiplex Nucleic Acid Detection	Class II Medical Device	Active
AxiLona AXP-100 Nanopore Gene Sequencer	Axbio	Gene Sequencing	Development	Active
AxiLona AXP-1000	Axbio	Ultra-high-throughput Gene Sequencing	Design & Development	Active
VRG-145	VRG Therapeutics	Migraine Prophylaxis	Phase 1	Active (Partnered with Novamigra Therapeutics)
Platform-derived candidates	VRG Therapeutics	Neurodegenerative Diseases	Pre-clinical	Active
CREATe Platform	VRG Therapeutics	Cell & Gene Therapy Components	Research	Active
IL-6 Driven Hyperinflammation	MediSieve	Acute Hyperinflammation (Sepsis, CRS, Acute Pancreatitis)	Pre-clinical	Active
AAV Gene Therapy Enabler	MediSieve	Depletion of pre-existing AAV neutralizing antibodies	Pre-clinical/Research	Active
Severe Malaria	MediSieve	Malaria (Plasmodium falciparum) via infected red blood cell depletion	Pre-clinical/Research	Active (Grant-funded)
Linea 1	LineaBio	iPSC line for cell‑therapy manufacturing	Unknown	Available
APC101	Andros Pharmaceutical	Chronic Pain	Phase IIb/III	Active
Cultivated Chicken Paste	Esco Aster	Food / Alternative Protein	Commercial	Active
Engineered Cancer Exosomes (HLA‑G Targeted)	Esco Aster	Cancer Immunotherapy	Preclinical	Active
Induced Retinal Pigment Epithelium (RPE) Cells	Esco Aster	Dry Age‑Related Macular Degeneration	Phase 1/2	Active
Nubeqa® (darolutamide)	Bayer	Metastatic Hormone-Sensitive Prostate Cancer (mHSPC)	Phase III	Active, recruiting
Kerendia® (finerenone)	Bayer	Chronic Kidney Disease in Type 2 Diabetes	Phase III	Active, recruiting