Sydney, Australia – The epigenetic ‘signature’ of a rare, hard-to-diagnose breast tumour has been found by scientists at the Garvan Institute of Medical Research. The discovery could lead to improved treatment guidelines and better outcomes for patients with this rare disease. Accounting for less than 1% of breast tumours, phyllodes...
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SAN DIEGO, Calif. — Bionano Genomics, Inc. (Nasdaq: BNGO) today announced a peer-reviewed publication from a team led by Manon Delafoy from the Institut Necker Enfants Malades (INEM) and colleagues from multiple French pediatric hematology centers showing how optical genome mapping (OGM) can be used to detect oncogenic structural variants...
Washington, DC – In 2023, cell and gene therapy saw an unprecedented surge with seven FDA approvals, and this year, an even greater number of these treatments could reach the market. So far in 2024, the regulator has given the green light to three new CGTs, and at least seven...
Tosse, France – After decades of limited progress—owing to the difficulty of treating the disease and resultant market risk—glioblastoma research is entering a new phase spurred by smarter trials, targeted funding and renewed interest from companies like Merck and Jazz Pharmaceuticals. Glioblastoma, a deadly brain cancer, is notoriously difficult to...
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...
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...