Phase I
The New York–based biotech will present data from a Phase I trial of 98 patients showing that fianlimab combined with Libtayo led to an overall response rate of 61% in advanced melanoma.
Wave will discontinue development of WVE-004 in C9-associated ALS and frontotemporal dementia after the therapy failed to show any clinical benefit in a mid-stage trial.
ReCode plans to use its SORT LNP genetic medicines delivery technology to treat CF and PCD patients who don’t respond to current treatments.
The precision neuroscience startup has notched $109 million in seed, Series A and B financing as it looks to further develop a Phase I candidate to treat a rare X-linked neuromuscular disorder.
The approval of Reata Pharmaceuticals’ Skyclarys for Friedreich’s ataxia highlights progress being made in the treatment of rare mitochondrial diseases.
Developers of psychedelics-based therapies say the industry is poised to explode, with several reporting strong clinical trial results and the FDA granting breakthrough status for two hallucinogenic drugs.
The deal with BlissBio is the latest in a cascade of multi-million- and billion-dollar acquisitions and collaborations centered on antibody-drug conjugates.
After a brief slump, interest in ADCs is at an all-time high, highlighted by a handful of recent multi-million- and billion-dollar acquisitions.
The FDA’s new guidance on decentralized clinical trials clarifies how investigators and trial sponsors can use digital health technologies and involve primary healthcare practitioners.
Longitudinal studies that track patients’ experiences can inform and accelerate the drug approval process for rare diseases.
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