Research & Development
A Pipeline Built on Novel Science
Four programs. Three therapeutic areas. One unwavering standard: mechanisms that matter, targets that are validated, and a path that leads to patients.
KRAS-mutant solid tumors
Program details
Overcoming KRAS resistance
NG-101 is a first-in-class small molecule targeting a novel allosteric site on mutant KRAS G12C and G12D. Unlike existing KRAS inhibitors that bind covalently to the GDP-bound state, NG-101 engages an induced-fit pocket accessible in both GDP- and GTP-bound conformations — a mechanism designed to overcome the primary resistance pathway seen with first-generation KRAS inhibitors.
Scientific rationale
KRAS mutations drive approximately 25% of all human cancers. Despite recent approvals, resistance emerges in nearly all patients within 12 months. NG-101's differentiated binding mode is designed to address this gap.
Current status
Phase I dose escalation ongoing. Initial cohorts dosed. Safety and PK data expected H2 2026.
Key highlights
- Novel allosteric binding site
- Active in G12C and G12D mutations
- Designed to overcome acquired resistance
- Oral once-daily dosing
Hematologic malignancies
Program details
T-cell redirection for blood cancers
NG-418 is a bispecific antibody that simultaneously engages CD3 on T cells and a novel surface antigen overexpressed on malignant hematologic cells. Early Phase I data show durable responses in heavily pre-treated patients, with a manageable cytokine release syndrome profile.
Scientific rationale
Hematologic malignancies remain a leading cause of cancer mortality. T-cell engaging bispecifics have demonstrated proof-of-concept; NG-418's target selection and format are designed to improve depth and durability of response.
Current status
Phase I dose expansion ongoing. Preliminary efficacy signals observed. Data presentation planned for ASH 2026.
Key highlights
- Novel hematologic target
- Durable responses in early data
- Manageable CRS profile
- IV infusion every 2 weeks
Pediatric lysosomal storage disorder
Program details
CNS-penetrant enzyme replacement
NG-312 is an engineered enzyme replacement therapy designed to address a rare pediatric lysosomal storage disorder with no approved treatment. The program uses a proprietary CNS-penetrant delivery platform to overcome the blood-brain barrier — a critical limitation of existing ERT approaches in neurological LSDs.
Scientific rationale
The target disorder affects approximately 1 in 100,000 live births and leads to progressive neurological decline. No approved therapy exists. NG-312's CNS delivery capability addresses the root cause of neurological progression.
Current status
IND-enabling studies underway. IND filing targeted for Q1 2027.
Key highlights
- Proprietary CNS delivery platform
- No approved therapy exists
- Orphan Drug Designation planned
- Pediatric rare disease focus
ALS — TDP-43 pathology
Program details
Restoring TDP-43 function in ALS
NG-204 is an RNA-targeting therapeutic designed to restore normal TDP-43 function in motor neurons. TDP-43 mislocalization and aggregation is a hallmark of ALS pathology present in over 97% of ALS cases. NG-204 uses a novel RNA-binding modality to correct aberrant splicing events downstream of TDP-43 dysfunction.
Scientific rationale
ALS is uniformly fatal with a median survival of 2-5 years from diagnosis. Existing approved therapies provide modest benefit. TDP-43 pathology is the most prevalent and mechanistically compelling target in ALS.
Current status
Lead optimization ongoing. Target candidate selection expected Q3 2026.
Key highlights
- Targets TDP-43 pathology in 97%+ of ALS cases
- Novel RNA-binding modality
- Addresses root cause, not symptoms
- Collaboration with leading ALS research center
Scientific platform
How we find what others miss
Our target identification platform combines computational structural biology, phenotypic screening in patient-derived models, and a systematic approach to mechanism validation. We look for targets where the biology is unambiguous, the patient population is defined, and the therapeutic window is real.
Structural biology-guided design
We use cryo-EM and computational modeling to identify cryptic binding sites and allosteric pockets invisible to traditional screening approaches.
Patient-derived disease models
Every program is validated in patient-derived organoids and primary cells before advancing — ensuring our biology reflects human disease, not cell line artifacts.
Mechanism-first target selection
We start with the mechanism, not the target class. If we can't articulate why a patient would respond, we don't advance the program.
Interested in our science?
We welcome conversations with potential partners, collaborators, and investors who share our commitment to rigorous, patient-first drug development.