Scripps Research and its nonprofit drug development arm, the Calibr-Skaggs Institute for Innovative Medicines, has officially confirmed that it is working on an assortment of novel regenerative medicines that can repair tissues damaged by age-related disease.
According to certain reports, these medicines act on endogenous stem cells in the body to control their fate. With many of the stated therapies already in preclinical and phase 1 clinical trials, they are expected to treat, upon launch, conditions like osteoarthritis, inflammatory bowel disease, lung diseases, heart failure, macular degeneration and more.
Talk about the specific drug development programs that Scrip is currently working on, they begin from the one focused on lung health. You see, the mechanism to repair damage to lungs by stimulating growth of stem cells in the lower airway, called type 2 alveolar epithelial cells (AEC2s), can reverse loss of lung function. Hence, Scripps Research and Calibr-Skaggs have developed a once-weekly, inhaled drug, CMR316, which promotes AEC2 stem cell expansion and the formation of new lung cells responsible for gas exchange.
More on the same would reveal how CMR316 promotes lung repair in several animal models of lung damage, including emphysema, chronic obstructive pulmonary disease (COPD) and IPF. At present, the therapeutic is in phase 1 clinical trial, where it is being used across healthy volunteers and IPF patients.
Scripps’ next focal point is heart. Here, the two organizations strive to address a mechanism during which cardiomyocytes, the muscle cells of the heart, are unable to regenerate tissue after a heart attack. Such incapacity has historically showed to cause progressive disease and scarring.
In response, Scripps Research and Calibr-Skaggs are developing a small molecule drug (CMV852) which can activate a regenerative pathway through YES-associated protein 1 (YAP). Once activated, this pathway can stimulate the growth of new heart cells. Meant to be injected precisely at the site of damage, the drug has already displayed an ability to induce new functional cardiac tissue and significantly improve heart function in mice and pigs after a heart attack.
Moving on, Scripps also has a program dedicated towards cartilage. This it does because OA, a disease involving degeneration of joint cartilage and chronic pain, currently affects more than 30 million individuals in the U.S.
Hence, Calibr-Skaggs is developing a small molecule, KA34, which may promote robust regeneration of lost cartilage in OA. By doing so, it will birth new functional joint tissue and decreased pain. Now, while KA34 has already undergone extensive preclinical testing and shown good safety in a phase 1b human trial, Calibr-Skaggs scientists are currently developing extended-release formulations of KA34 to enable the drug to stimulate cartilage growth over longer periods (3 to 6 months). The idea behind is to reduce the frequency of injections and patient burden.
Another bodily aspect that Scripps and its foundation are looking to focus on is eye. At present, age-related macular degeneration (AMD) is the most common form of vision loss associated with age, affecting nearly 13% of people aged 40 and older. As AMD is associated with the loss of retinal pigment epithelial cells (RPEs), Scripps’ researching team has identified several small molecule drugs that selectively expand RPEs to regenerate a functional RPE layer.
Currently being studied across laboratory and animal studies, the solution in question, assuming it’s validated, will be a one-time injection of a long-acting drug which will replenish the RPE layer in people with early-stage AMD.
Rounding up the highlights would be a program rooted in intestine health, with specific attention to conditions like IBD, Crohn’s, and ulcerative colitis that are caused by auto-inflammatory destruction of the gastrointestinal tract.
For this particular program, Calibr-Skaggs has developed a long-acting, chemically modified GLP-2 mimetic which robustly promotes healing in the gut and restores the mucosal barrier function. After going through a successful phase 1 clinical trial in humans, a trial where it showcased safety, a best-in-class half-life and upregulation of a key biomarker of intestinal repair, the drug is now set to for phase 2 round
