Phase 1
The FDA recently approved the first cellular therapy for Type 1 diabetes and others may not be far behind. But experts say challenges still exist to the widespread application of these treatments.
Early-stage data shows that Viridian’s thyroid eye disease candidate induces clinically meaningful improvements in eye protrusion after six weeks of treatment.
Gene editing technologies are advancing rapidly in the clinic, with the potential first approval later this year, but challenges remain.
Phase I/II data on AbbVie and Genmab’s recently approved bi-specific antibody in a second cancer indication positions them to talk to regulators about filings to challenge Roche’s Lunsumio.
The company halted studies of the treatment for a rare, advanced form of eye cancer after clinical results showed just one response in 47 patients.
While early, the Phase I study results for 12 patients represent a promising return on Bayer’s investment in BlueRock, which it launched with Versant Ventures in 2016 and fully acquired three years later.
A year after treatment, Vertex’s VX-880 has eliminated the need for insulin injections in two Type 1 diabetes patients in a Phase I/II clinical trial of its investigational stem cell-based therapy.
The Huntington’s disease space saw a flurry of activity Wednesday as PTC and uniQure released data from their respective mid-phase trials.
The Bespoke Gene Therapy Consortium intends to bring AAV-based gene therapies to patients whose diseases are often ignored by commercial interests.
Eli Lilly has secured the rights to Verve Therapeutics’ gene editing approach, a “one-and-done” method that the companies hope will lower the cardiovascular risk factor lipoprotein(a).
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