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Join us in engineering next-generation CRISPR-based genetic medicines.

Partner
Neurological Diseases
Huntington's Disease
Familial Amyotrophic Lateral Sclerosis
Spinal Muscular Atrophy
Spinocerebellar Ataxia
Early-onset Familial Alzheimer’s Disease
Parkinson's Disease
Progressive Supranuclear Palsy
Dravet Syndrome
Angelman Syndrome
Friedrich Ataxia
Batten Disease
Ophthalmological Disease
Retinitis Pigmentosa
Cone-rod Dystrophy
Leber's Congenital Amaurosis
Hereditary Optic Atrophy
Stargardt Macular Degeneration
Choroideremia
Glaucoma
Best Vitelliform Macular Dystrophy
Achromatopsia
Usher Syndrome
Multisystem, Muscle & Metabolic
Cystic Fibrosis
Duchenne Muscular Dystrophy
Myotonic Dystrophy
Limb-girdle Muscular Dystrophy
TTR Amyloidosis
Alpha-1 Antitrypsin Deficiency
Familial Hypercholesterolemia
Tyrosinemia
Phenylketonuria
Acute Intermittent Porphyria
Hypertriglyceridemia
Hutchinson–Gilford Progeria Syndrome
Methylmalonic Acidemia
Propionic Acidemia
Primary Hyperoxaluria
Hematopoietic Disorders
Sickle Cell Disease
Severe Combined Immunodeficiency
Fanconi Anemia
Hemophilia A/B
Chronic Granulomatous Disease
Von Willebrand Disease
Cell Therapy
Modifications can be made to to nearly any cell therapy product including but not limited to:
CAR-T
NK
TiL
HSC
iPSC
scribe
v. to mark or score materials with a pointed instrument as a guide to cutting or assembling
Scribe is designing custom tools for therapeutic use on any given genetic target or application in vivo.
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