Novel Gene Therapy Platform for the Treatment and Accelerated Healing of Orthopedic Injuries

Summary

• Musculoskeletal disorders such as orthopedic injuries are a leading cause of physical disability in the United States
• A major hurdle in effective treatment of orthopedic injuries is ineffective biological processes such as poor blood supply and harsh intra-articular environment
• Dr. Crystal is a world-renowned KOL in the gene therapy space
• In order to overcome these treatment burdens, Dr. Crystal is leading a team of experts to develop novel gene therapies to treat orthopedic sports injuries

Technical Overview

• Dr. Crystal and his team are developing gene therapy viral vectors that code for healing-promoting proteins that will be delivered at the site of orthopedic injury and surgical repair
• The 3 core components of the program are:
• Vector selection and design:
• The team is developing replication deficient adenoviruses that express the target gene for 1-3 weeks. Once administered, the vector starts expressing after 8 hours and peaks at 7 days
• Therapeutic gene targets:
• Ideal gene targets will focus on enhancing post surgical healing such as: Induction of angiogenesis, growth factors, and increasing antioxidants at the site of injury
• Appropriate injury model:
• The team will use a variety of rodent models that mimic sports injuries such as rotator cuff tears, and interior cruciate ligament tears

Market Opportunity

• Musculoskeletal conditions are among the most disabling and costly conditions suffered by millions of people around the world
• The orthopedic trauma market was estimated to be 5.3 billion in 2016 with an expected 8.3% yearly growth
• Gene therapies offer a unique approach to effectively treating severe injuries that would otherwise take months to heal
• Dr. Crystal is developing a new modality to accelerate healing and recovery for a range of orthopedic injuries

Partnering Opportunity

Weill Cornell Medicine is seeking an industrial partner with deep domain expertise in gene therapy, orthopedics and sports medicine to advance the development of this novel modality and translate it to the clinic

Figure 1: Schematic of using adenovirus gene therapy for accelerated healing of orthopedic injuries.