We believe

that gene therapy has the potential to go beyond treating rare disorders and tackle the major prevalent diseases affecting people around the world.

It is our mission to take on one of the largest problems in all of medicine – the leading cause of disability, chronic pain, and a significant risk factor for opioid addiction – osteoarthritis (OA).


Americans are affected by OA

in costs to our healthcare system

Why Osteoarthritis?

Osteoarthritis (OA) is a degenerative joint disease characterized by destruction of cartilage and structural changes in bone within a joint, such as the knee, which causes pain and disability.

OA is becoming more widespread as a result of the aging population and growing prevalence of obesity.

OA is the leading cause of disability and chronic pain, and a significant risk factor for opioid addiction.

For many patients, inflammation triggered by interleukin 1 (IL-1) causes pain and continued progression of OA.

Each year, millions of patients with OA are treated with non-steroidal anti-inflammatory drugs (NSAIDs), opioids, and steroid injections to manage their pain.

OA represents a major economic burden, owing to direct medical costs annually and loss of productivity.

There are currently no available treatments that can stop or slow progression of OA.

Treatments are limited to disease management and pain management, and fall into three categories*

Non-pharmacologic therapy

Includes a range of strategies, such as patient education and self-help, exercise programs, and weight loss

Pharmacologic therapy

Pharmacologic options may include acetaminophen, NSAIDs, opiates, and the intra-articular injection of glucocorticoids or hyaluronic acid. NSAIDs may bring partial relief to patients but are not well tolerated in many OA patients. Local injection of steroids can bring rapid relief, but the effects often last only a few weeks and cannot be injected very often.


Surgical approaches may be an option for some patients. In certain joints, such as the knee or hip, surgical replacement is an option for patients at advanced stages. Although this approach is generally successful, it involves a major and expensive surgical procedure with extensive downstream rehabilitation. In many cases, revision surgery is necessary to replace a prosthetic joint that has become dysfunctional.

*according to most recent guidelines issued by the American College of Rheumatology and the AAOS.