Alisa Shakarian of Cambridge Biomarketing discusses the challenges pharma and biotech companies face in bringing drugs into the rare disease market.
Amit Rakhit, MD, MBA, Chief Medical and Portfolio Officer of Ovid Therapeutics, discusses his company’s approach in developing treatments for patients with rare neurologic disorders. Ovid Therapeutics is a biopharmaceutical company focused exclusively on developing impactful medicines for patients and families living with rare neurological disorders. Ovid’s drug candidate, OV101, is currently in development for the treatment of symptoms of Angelman syndrome and Fragile X syndrome. Ovid is also developing OV935 in collaboration with Takeda Pharmaceutical Company Limited for the treatment of rare epileptic encephalopathies. Ovid has initiated the Phase 2 STARS trial of OV101 in adults with Angelman syndrome and a Phase 1 trial in adolescents with Angelman and Fragile X syndrome to identify doses suitable for younger patients.
Lara Sullivan, MD, President and Founder at SpringWorks Therapeutics discusses desmoid tumors, a rare, non-metastatic tumor of connective tissue cells, which can cause severe morbidity, pain and loss of function in children and adults. Desmoid tumors can show up in almost any part of the body, and desmoids that are faster growing or located near vital organs can cause life-threatening problems. Approximately 900-1,200 people are diagnosed with desmoid tumors each year in the U.S. Currently available treatments include unapproved medical therapy, radiation therapy, thermal ablation and surgery, which can be dangerous, costly and offer limited effectiveness. SpringWorks Therapeutics is planning to initiate a Phase 3 program to establish safety and efficacy of nirogacestat (PF-03084014), its gamma-secretase inhibitor, and will work …
Alisa Shakarian of Cambridge Biomarketing discusses the role of Advocates in rare diseases and the challenges of diagnosing rare diseases.
Julian Adams discusses Gamida Cell’s NAM technology to develop two of its lead clinical programs by expanding blood forming stem and progenitor cells from umbilical cord blood to create highly functional, stand-alone grafts that serve as a universal bone marrow transplant solution. Gamida Cell has successfully harnessed its NAM platform technology to leverage the advantages of cord blood and overcome its limitations by increasing the number of CD34+ stem and progenitor cells, while enhancing their homing and engraftment efficiency.