Des Moines, Iowa – Most of the 15 million children with a rare disease have no FDA-approved treatments available to them. And when it comes to the most-rare conditions, there isn’t even a pipeline. Late last week, the world heard an incredible story: a baby with an exceptionally rare genetic...
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Santa Cruz, Calif. – A handful of billion-dollar deals in the rare disease space highlights the uptick in Big Pharma’s investment, but it’s still extremely low compared to the money flowing to more common indications. They’re rare, they’re confounding, and R&D for therapies to address them is painfully underfunded. Rare...
Washinton, DC – On March 15, the FDA’s Oncologic Drugs Advisory Committee will meet to discuss two CAR T cell therapies for multiple myeloma—Abecma, from Bristol Myers Squibb and 2seventy bio, and Carvykti, from Johnson & Johnson and Legend Biotech. Both treatments are currently approved for the treatment of adults...
Washington, DC – As the oncology space moves away from single oncogene-targeted treatments, immunotherapies that aim to prevent immune fatigue and enhance immune cell engagement are emerging as promising therapeutic trends, experts told BioSpace. But these therapies are not always intended to be deployed on their own. Increasingly, scientists and...
Washington, DC – Advances in antibody-drug conjugates, including novel payloads and new targets, will be featured at the American Association for Cancer Research’s annual conference, taking place this week in San Diego, where companies, including Elevation Oncology and Tubulis, will present new data from their respective programs. The first antibody-drug...
Washington, DC – Over the last few years, we’ve witnessed a significant shift in the regulatory space for rare disease therapeutics, with the FDA and other authorities making conscious efforts to implement much-needed regulatory flexibility to accommodate the practical challenges of trial design for these conditions. For small biotechs and...
San Diego, Calif. – The American Association for Cancer Research’s annual conference kicks off Friday as more than 20,000 attendees descend on San Diego. Analysts and researchers who spoke with BioSpace said they’re looking forward to early scientific data, and antibody-drug conjugates (ADCs) will be a key focus. Stay tuned to...
Basel, Switzerland – The past two years have seen a pair of new treatments approved for a particularly intractable neurodegenerative disease—amyotrophic lateral sclerosis. But while Amylyx’s Relyvrio and Biogen’s Qalsody have crossed the FDA finish line, others have stumbled in a regulatory space that experts say is still evolving. The...
New research presented this week at ACR Convergence, the American College of Rheumatology’s annual meeting, shows that Black people living with systemic sclerosis may have more severe disease and worse prognosis than patients in other racial or ethnic groups, and these worrying disparities may be driven by several socioeconomic factors...
Barcelona, Spain – Bladder cancer has one of the highest incidence rates in the world and ranks as the fourth most common tumour in men. Despite its relatively low mortality rate, nearly half of bladder tumours resurface within 5 years, requiring ongoing patient monitoring. Frequent hospital visits and the need...