Grant Award Details

A Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study of GNSC-001 Evaluating Safety, Pharmacodynamics, and Biomarkers in Knee OA
Grant Number: 
Project Objective: 
  • The objective of this award is to complete a phase 1b clinical trial evaluating safety, pharmacodynamics and biomarkers of the AAV gene therapy GNSC-001 for treatment of knee osteoarthritis. This includes study preparation and site start-up activities, enrolling and treating fifty patients, follow-up assessments on those patients, bioanalytical assessments of patient samples, and manufacturing process improvement and scale-up. In addition, regulatory activities will include application for RMAT designation and an End of Study Type B meeting with FDA.
Disease Focus: 
Bone or Cartilage Disease
Human Stem Cell Use: 
Somatic Cell
Award Value: 

Grant Application Details

Application Title: 
  • A Phase 1b, Randomized, Blinded, Placebo-Controlled Dose-Ranging Study of GNSC-001 Evaluating Safety, Pharmacodynamics, and Biomarkers in Knee OA
Public Abstract: 

Therapeutic Candidate or Device

GNSC-001 is an adeno-associated vector (AAV) expressing an optimized form of IL-1Ra, a naturally occurring protein that blocks IL-1 signaling.


Osteoarthritis of the knee

Therapeutic Mechanism

GNSC-001 is an adeno-associated virus (AAV) gene therapy vector that expresses IL-1Ra and confers long-term IL-1 inhibition following a single, local injection into the affected joint. By blocking IL-1 signaling, GNSC-001 is expected to improve inflammation, symptoms (pain/function), and slow or reverse disease progression (cartilage degeneration) in osteoarthritis patients.

Unmet Medical Need

Osteoarthritis is a degenerative joint disease that is the leading cause of disability. Current treatments are only palliative; nothing can slow or stop osteoarthritis progression. GNSC-001 is injected into the knee, blocking IL-1 signaling to reduce inflammation, pain, and joint degeneration.

Project Objective

Phase 1b trial completed; ready for Phase 2b

Major Proposed Activities

  • Enroll 50 osteoarthritis patients in a Phase 1b trial; assess safety and pharmacodynamics of GNSC-001 at two dose levels.
  • Evaluate the effect of GNSC-001 on symptoms and biomarkers of disease progression over time.
  • Prepare for next phase of development by scaling up the GNSC-001 manufacturing process and meeting with FDA to discuss requirements for approval.
Statement of Benefit to California: 

Arthritis affects over 5.9 million Californians (20%). Osteoarthritis is the leading cause of disability; current treatments are limited to pain management. GNSC-001 has the potential to alleviate osteoarthritis pain and function, and slow or reverse its progression, for millions of Californians suffering from this disease. In addition, this research supports Genascence as a California corporation, and includes clinical work in California, directly fostering innovation and creating local jobs.