This column offers a deeper look into especially innovative or entrepreneurial biopharma companies, individuals, or institutions through our special lens — lessons learned, actionable info, benchmarking, best practices, etc.
Medicines360 On A Mission For Women
Medicines360 is using all its developed-world revenue to extend access to essential medicines to all women, regardless of the economic and healthcare circumstances in their part of the world.
Lysogene: Mother of Invention
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.
Apellis Advances On Autoimmunity
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 Pharmaceuticals: Return & Reach Beyond
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: Mission In The Orphan Space
Cydan is using a new business model in which they challenge the ways companies dealt with orphan drugs in the past.
PolarityTE: Beyond Cells, To Whole Tissue
In this series on "Life Science Leadership In Action," we discuss PolarityTE, which focuses on regenerating lost tissues in their original complex forms.
Prometic: New Tech Harvests Orphan Treatments
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.
Cytokinetics: Keeping Its Sights On Independence
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.
CohBar: Producing Mitochondria Medicine
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?
Novavax: Scouting Past The Long Trail To Market
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.”)
How Rebiotix Avoids The Typical Biopharma Startup Missteps
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.
RespireRx: Life Science Leadership In Action
Suffocation seldom gets the credit it deserves for causing death in so many conditions — from sleep apnea to heart failure to drug overdose. But if you view the large variety of those cases at a higher resolution, you will see “respiratory failure” as the common final, fatal effect.
InVivo Therapeutics: Taking On Spinal-Cord Injury
I have never seen such a bold and ambitious approach to treating, dare I say healing, one of the worst injuries anybody can sustain — a spinal cord injury. The story of InVivo’s development of a tiny scaffold inserted into the injury site merits attention in “The Enterprisers” both for its ambitious goal and its means for reaching it.
Amarantus: Risen From The Ashes
A simple twist of fate is all it takes to knock you off your original track. In the case of the company that would be reborn as Amarantus, it was another company’s bad luck in the clinic that ended its first push for partnering and funding.
BEYOND THE PRINTED PAGE
Why Top Researchers And Physicians Enter The World Of Biopharma R&D
David Chang, M.D., Ph.D., founder and CEO of Allogene Therapeutics, along with numerous other physicians have opted to become biopharmaceutical executives, but one doesn’t have to go to medical school to be driven to make a difference for patients.
Genmab’s Commercial Engine Aspirations
Jan G. J. van de Winkel, Ph.D., cofounder and CEO of Genmab, discusses the company’s aspirations to eventually commercialize its own products.
Tariff Wars, Politics, And Other Concerns Of 6 Biopharma CEOs For 2020 And Beyond
Six biopharma CEOs talk tariff wars, politics, and other concerns for 2020 and beyond.
LIFE SCIENCE LEADER BLOGS
Brightspot: How A Biopharma VC Makes A Difference For Orphans
Ed Torres, managing partner at Lilly Ventures, shares how he and his wife got involved in making a difference for U.S. orphans.
The CNS Summit — A Caring Community Of Collaborative Solution Finders
Chief editor Rob Wright explains what differentiates the CNS Summit from other biopharmaceutical industry conferences.