News
After a patient safety signal and then death, the FDA in October 2025 placed holds on two of the company’s CRISPR programs for hereditary transthyretin amyloidosis.
FEATURED STORIES
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis by the Rare Disease Company Coalition shows. That could mean $4 billion in missed revenue for already cash-strapped biotechs.
Together with robust data-driven modeling, rethinking regulation and data use could push forward a notoriously challenging field.
From opening new therapeutic mechanisms to repairing neuronal damage, investigational molecules from Ventyx Therapeutics, AC Immune, Gain Therapeutics and more could shape the future of Parkinson’s disease treatment.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
The FDA’s rare pediatric disease priority review voucher program missed reauthorization at the last minute in 2024; advocates have been fighting to get it back ever since.
THE LATEST
An FDA committee’s September 2024 vote to limit the use of Merck’s Keytruda and BMS’ Opdivo in stomach and esophageal cancers based on PD-L1 expression levels reflects an emerging trend that leverages ever-maturing datasets.
Among the 55 novel drugs that crossed the regulatory finish line last year were notable new mechanisms of action, coming particularly in the oncology and neurosciences spaces.
In this short teaser, BioSpace’s Head of Insights Lori Ellis talks to CBER Director Peter Marks and Tom Whitehead, Co-Founder of the Emily Whitehead Foundation about anticipated discussions at the upcoming GenScript Biotech Forum.
Staff cuts will leave IGM Biosciences with 37 employees. The company is also halting development of two bispecific antibody T cell engagers for autoimmune diseases.
After the successful development of mavacamten and the sale of MyoKardia to BMS in 2020, former executives of the biotech are back together with a mission to make cardiovascular disease curable and preventable.
The Biotech Ecosystem Venture Fund will combine the sourcing capabilities of venture capital firm Andreessen Horowitz (a16z) with Eli Lilly’s expertise in R&D—plus half a billion in capital from the Big Pharma.
Research has shown remnant cholesterol is a significant factor in cardiovascular disease risk.
If approved, Pfizer’s sasanlimab will distinguish itself from Merck’s blockbuster Keytruda as the first PD-1 inhibitor indicated in combination with BCG for high-risk non-muscle invasive bladder cancer patients who had not previously undergone BCG treatment.
Concurrently, a preprint from the industry-backed Vital Transformation found a 50% drop in company investments into small-molecule drug development.
Eli Lilly’s idiopathic pulmonary fibrosis deal with Mediar is centered around MTX-463, an anti-WISP1 antibody that early studies have found to be safe and capable of engaging its target.