Oncology

Developing targeted therapies for solid tumors

Our initial approach in oncology is to leverage our targeted therapy platforms to deliver potentially game-changing therapies to patients with desmoid tumors and Neurofibromatosis Type 1 (NF1). We’re also exploring how these therapies may work in combination with immunotherapy treatments to improve patient response.

Desmoid Tumor

Desmoids are rare soft-tissue invasive tumors that occur in approximately 900 patients annually in the U.S. and typically strike patients between 20 and 30 years old. While non-metastatic, desmoids can grow aggressively and cause significant morbidity and mortality if located near vital organs. There are currently no approved therapies, and high recurrence post-surgical resection makes local treatment unfeasible for many patients.

Current Treatments

Unapproved medical therapy, radiation therapy, thermal ablation and surgery, which can be dangerous, costly and offer limited effectiveness.

Impact

  • ~900people diagnosed/year in U.S.
  • Mostly between 15-60 years old
  • Patients can undergo dozens of surgical and pharmacologic options with limited success

Neurofibromatosis Type 1

NF1 is an autosomal dominant disorder caused by NF1 gene mutations that causes unchecked activation of the MAPK pathways leading to numerous nervous system tumors. The disease affects between 1 and 3,000 births annually, with symptoms typically beginning in infancy and childhood. NF1 has been linked to a spectrum of disorders such as malignant peripheral nerve sheath tumors, blindness, deafness, disfigurement, bone abnormalities, learning disabilities and severe pain. There are currently no approved therapies, and nervous system infiltration typically makes local treatment unfeasible for many patients.

Current Treatments

Chemotherapy, radiation therapy and surgery, which often results in regrowth.

Impact

  • NF1 affects 1 in 3,000 individuals
  • Usually diagnosed in infancy and early childhood
  • Can cause significant disfigurement, compression of vital structures, neurologic dysfunction and pain