Our lead program represents the most promising strategy to both cure existing Huntington’s disease (HD) patients and prevent onset of HD in individuals at risk of developing HD in the future.
IG-HD01 is a first-in-class investigational therapeutic that combines a pre-formed CRISPR ribonucleoprotein (RNP) complex with Incisive Genetics’ proprietary lipid nanoparticle (LNP) delivery platform.
A differentiated approach to Huntington’s Disease
Unlike approaches that act downstream at the RNA or protein level, IG-HD01 enables direct, allele-selective excision of the disease-causing portions of the mutant HTT gene, while preserving normal HTT function, addressing the full spectrum of known pathogenic mechanisms in Huntington’s disease.
Allele-selective excision
of mutant HTT without disrupting wild-type HTT
Efficient biodistribution
demonstrated in preclinical models
Favorable safety profile
including repeat dosing in HD mice
Technology that determines therapeutic success
Our proprietary platform is engineered to deliver gene editing machinery with the control, efficiency, and safety profile necessary for in vivo therapeutic gene editing.
Pre-formed RNP delivered ready to edit DNA
Eliminates intracellular assembly steps and enables immediate genome editing activity.
Pre-formed RNP acts quickly and clears rapidly
Short-lived activity helps minimize prolonged exposure and downstream off-target effects.
Large cargo capacity supports complex edits
Enables delivery of multiple gRNAs and repair templates within a single therapeutic.
Higher editing efficiency enables lower dosing
Efficient delivery of functional RNP reduces the amount of material required per dose.
No protein translation required
Avoids cellular production of gene editing components, reducing steps where variability and off-target risk can emerge.
Repeat dosing enables adjustable genome editing
Therapeutic effect can be tuned over time rather than locked in after one administration.
Our Pipeline
Towards treatments for high-impact genetic diseases
IG-HD01 is being advanced with a clear path to the clinic, informed by decades of Huntington’s disease expertise and aligned with established clinical trial infrastructure.
Development is supported by collaboration with the Huntington Study Group (HSG), the world’s leading HD clinical trial network.
Pipeline by target area, program, indication, delivery method, editing target, and development stage progress.
Target
Program
Indication
Delivery
Editing Target
Research
Lead Optimization
IND Enabling
Phase 1/2 Studies
Brain
IG-HD01
Huntington's disease
Intracranial Infusion
IG HTT SNP 1
IG-HD02
Huntington's disease
Intracranial Infusion
IG HTT SNP 2
IG-HD03
Huntington's disease
Intracranial Infusion
IG HTT SNP 3
Bone Marrow
IG-SCD 1
Sickle Cell disease & Thalassemia
Intraosseus Infusion
Activation of fetal hemoglobin
IG-SCD 2
Sickle Cell disease & Thalassemia
Intraosseus Infusion
Activation of fetal hemoglobin
Liver
IG-AT01
Alpha-1 antitrypsin deficiency
Intravenous Infusion
Undisclosed
While IG-HD01 is the lead program, our delivery platform supports additional programs across genetic diseases. Pipeline assets are advanced with strategic focus and partnership potential, extending the impact of the core delivery technology.
