The Science Behind KHP-1

A single-injection regenerative gene therapy in development for canine osteoarthritis — explained simply, for pet parents.

What Is Osteoarthritis in Dogs?

Osteoarthritis (OA) is a degenerative joint disease that affects a large share of dogs, especially as they age. Cartilage breaks down, joints become inflamed, and everyday movement — walking, playing, climbing stairs — becomes painful. Current options like pain medication, joint supplements, and physical therapy can help manage symptoms, but they don’t address the underlying joint degeneration.

Senior dog resting, showing signs of joint discomfort

How KHP-1 Is Designed to Work

KHP-1 is a regenerative gene therapy, administered as a single injection directly into the affected joint. Rather than simply masking pain, it is designed to prompt the joint’s own cells to reduce inflammation and support tissue repair — targeting the biology of OA at its source.

Read the technical detail

Stress and aging drive the build-up of a lipid called ceramide in joint tissue. Ceramide pushes cells into senescence, which triggers inflammation and tissue damage — and that damage drives further senescence, reinforcing a self-perpetuating cycle.

KHP-1 delivers the gene for acid ceramidase, the enzyme that breaks down ceramide. By restoring acid ceramidase activity at the joint, the therapy is designed to:

  • Revert senescent cells to normal function
  • Interrupt senescence-induced inflammation
  • Stop tissue damage

Preclinical mechanism-of-action data; full vector/construct details are available to prospective investors in our data room upon signing an NDA.

Switching Off the Inflammation Loop

Ceramide Accumulates

Age and mechanical stress drive ceramide build-up in joint tissue.

Cells Senesce

Ceramide pushes joint cells toward senescence, releasing inflammatory signals.

The Loop Reinforces

Inflammation causes damage; damage causes more senescence. This is the engine of OA.

KHP-1 Breaks the Loop

Acid ceramidase converts ceramide into a pro-survival signal, so cells stop dying.

This is disease modification, not just pain relief — delivered as one dose, directly into the joint, by AAV gene therapy that establishes local, durable expression.

How KHP-1 Compares

KHP-1 has not been approved and these are design goals, not confirmed clinical results. Controlled efficacy will be established in the registrational study.

 KHP-1Monthly InjectableDaily NSAIDsJoint Injections
Single lifetime dose✓———
Targets disease, not just pain✓———
Delivered into the joint✓——✓
No daily chronic-use burden✓———

Comparison reflects design intent for KHP-1, an investigational product, against general categories of existing treatments. Not a claim of superiority over any specific approved product. Swipe to see all columns on mobile.

Regulatory & Safety

KHP-1 is currently in development and has not been approved for commercial use. The FDA has an established regulatory pathway for canine osteoarthritis biologics, and approval is based on a defined pivotal study rather than an open-ended timeline. A completed pilot at SpotOn Veterinary Hospital and a pre-INAD meeting with the FDA have informed the study design ahead of the registrational trial.

KHP-1’s science is protected by sixteen patents — nine composition-of-matter and seven method-of-treatment — with core intellectual property originating from the Icahn School of Medicine at Mount Sinai.

Links to scientific publications