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 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.
Reata refused to take what seemed like a dead-end trial as a losing proposition. Instead, the company went to work mining the trial for the very insights that subsequently saved it.
Cydan is using a new business model in which they challenge the ways companies dealt with orphan drugs in the past.
In this series on "Life Science Leadership In Action," we discuss PolarityTE, which focuses on regenerating lost tissues in their original complex forms.
Always stick with your original goals, even when you reap another, off-the-scale success. Like many biopharma companies, Prometic invented a novel technology platform, initially to make new medicines available to unserved populations.
Can a biopharma company have a soul? If so, the soul should be one that endures. “The biology is the soul of our company,” says Robert Blum, president and CEO of Cytokinetics. “We have pioneered an area of biology — muscle activation — proven to offer a compelling pharmacology.
Why shouldn’t our mitochondria want us to live long, prospering in good health? Why shouldn’t they — as symbiotic microbes turned cellular organelles with their own mini-genomes — carry genes that help ensure our healthful survival?
Clinical challenges lurk all along the pathway for any company developing new vaccine candidates and technology — and that goes at least twice for Novavax. As we go to press with this, the company is dealing with an anxious investment community about the “failed” Phase 3 trial of its RSV F vaccine in older adults, for protection against respiratory syncytial virus (RSV). (See “Press-Time Thunder.”)
Lee Jones, the CEO and cofounder of Rebiotix, describes building the company in an “of course, this is how you do it” manner. The larger issue — whether the company’s Microbiota Restoration Therapy (MRT) platform will succeed in a somewhat besieged field — can only be resolved over time. But it will most likely be biology that decides the matter, not the typical lack of clear direction or organization that plagues so many biopharma startups.
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