Karen Aiach had no background in healthcare when her young daughter received a diagnosis of a rare form of Sanfilippo syndrome. Aiach built her company, Lysogene, to create the world’s first treatment for the terrible disease.
The ARM Foundation for Cell and Gene was launched in June, and it marks a new stage in development of the RM space as it moves from an early era or “promise” to one of fulfillment. Today, nearly 900 companies worldwide are developing RM therapies.
The unusual story of how Apellis was formed and what led it to target C3, the central factor in the innate-immunity complement system, to treat autoimmune diseases — starting with the ultra-rare blood condition, PNH.
The heads of the East and West Coast Innovation Centers for Bayer Healthcare discuss their parallel missions, therapeutic areas of focus, and dealmaking models.
Symbiotix Biotherapies is an early pioneer of the microbiome space, preparing to enter clinical-stage development of its lead compound for treating MS and IBD.
“We are a vaccine company,” says CEO Mei Mei Hu. “We’re developing a new class of vaccines for endogenous proteins, leveraging a commercially successful technology platform in which we’ve invested more than 20 years.”
This year’s IO update is more compact because the essential story has contracted. The odds of success have shifted dramatically and disparately among the various players — all due to Merck’s Keytruda.
Acceleron has four drugs in development in the hematology, neuromuscular, and pulmonary disease areas. Its lead drug luspatercept has completed Phase 3 trials for treating myelodysplastic syndromes (MDS).
“We had to decide whether to spend a lot of money to bring on a sales force and marketing team, and expand manufacturing to meet demand, or take our resources and refocus them on these rare disorders.”
Alexis Howerton is a first-time pharma CEO who saw potential in an older drug class that had failed in its targeted indications. She then jumped feet first into obtaining the ideal candidate compound and starting a company to develop it.
From founding the company to funding it, the CEO of Aurinia Pharmaceuticals explains the genesis of the company and why he left retirement to return to guide the company.
Drawing from his 27 years in the pharma industry, Chris Garabedian shares useful lessons for combining business savvy and scientific leadership in drug development.
This French company is addressing vertigo and hearing loss inside the inner ear.
Beginning her career with the query — how does biology cause disease? — Vicki Sato progressed to business, turning scientific understanding into new therapeutic drugs. Sato has been a biopharma leader since the industry’s formative years, helping drive the growth of Biogen and Vertex, and she is still guiding new companies today.
