Nightstar Therapeutics plc
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Latest From Nightstar Therapeutics plc
By agreeing to pay $7.3bn for Reata and its Friedrich’s ataxia drug Skyclarys, “recalibrating” Biogen can be more patient with launches of Alzheimer’s drug Leqembi and ALS therapy Qalsody.
With a platform of interesting preclinical assets and a Phase III-ready program from a collapsed biotech business, new company Beacon Therapeutics can claim unconventional origins.
The UK firm’s substantial series A raise will help it advance a pipeline of gene therapy candidates for both rare and common retinal conditions, including a Phase II candidate rescued from a struggling biotech.
Ring Therapeutics is turning to a previously unexplored family of viruses to improve the safety and efficacy of gene therapy. The preclinical science suggests using these anelloviruses as vectors can deliver payloads precisely and without causing a significant immune response. Human trials will need to demonstrate the technology can live up to its disruptive potential.
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