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Therapies from industry leaders BioMarin and Ascendis Pharma supply a key hormone that promotes bone growth. In order to move the field forward, challengers are looking to address the underlying cause of the rare, genetic disease.

New analysis from Jefferies shows that rare disease and cancer drugs granted the status are especially likely to be approved.

Takeda wanted to create something new in the cell therapy world by combining the technology with T cell engagers. A series of acquisitions in 2021 started the process.

As the industry loses one of its key female leaders in GSK CEO Emma Walmsley, BioSpace profiles the women leading the industry’s smaller biopharmas.

Drug pricing criticism often fixates on a price at a single point in time but drug pricing is never static.

Cell and gene therapy experts question where the FDA designation fits in an environment that features a range of intersecting regulatory perks.

As industry leaders gather at the annual event in Phoenix, the cell and gene therapy space remains in a state of flux, with M&A activity and regulatory support signaling momentum while commercialization challenges continue to hinder broader investor interest.

Smarter design through targeted delivery and human-relevant testing can save the industry from costly safety failures.

While Bruton’s tyrosine kinase inhibitors are often hailed as the next big breakthrough in multiple sclerosis, Immunic Therapeutics and others are leveraging neuroprotective targets and remyelination to keep the disease at bay.

Following up on previous, dimly received issuances, a new set of ideas published by the FDA to streamline regulatory pathways for cell and gene therapies ‘for small populations’ is receiving a warmer welcome—but experts warn it will take more to turn the tide for the fraught therapeutic space.