LEADING EDGE BIO

Why Mitochondrial Gene Editing May Become the Next Platform Technology in Genetic Medicine One of the most interesting trends in biotechnology is that entirely new companies are increasingly being built around a single enabling platform rather than a single therapeutic asset. The recent launch of Primera Therapeutics is an excellent example. While headlines focused on the company's strategic collaboration with Cellectis and a partnership valued at up to $750 million, the more

Why One-Time Gene Therapies Are Redefining the Business of Medicine For decades, the pharmaceutical industry has operated under a remarkably consistent economic model. Develop a medicine. Treat patients over many years. Generate recurring clinical benefit and recurring revenue. That model is beginning to change. Gene therapies are introducing an entirely new economic framework, one in which a single treatment has the potential to provide years, or perhaps decades, of clinical

What FDA Approvals Really Tell Us About the State of Biopharmaceutical Innovation The FDA approved 37 novel medicines in 2022, a figure that sits modestly above the historical average. On the surface, it is an encouraging statistic. Beneath the headline, however, it tells a much more interesting story. Given the disruption caused by the COVID-19 pandemic, this level of productivity was far from inevitable. Clinical trial recruitment slowed dramatically during 2020 and 2021. H