Jaks capped in arthritis – but the FDA might not be finished
The US regulator’s restrictions spell trouble for the Jak inhibitor class – though there will be beneficiaries.
Astellas’s gene therapy aspirations crumble
AT132 has prompted more safety concerns, this time at a lower dose than before.
Toxicity concerns send Larimar down
The company hopes that its novel approach in Friedreich’s ataxia could still have legs, but other assets are further ahead.
Jury still out on Sarepta’s next generation
The group’s lead next-gen exon skipper beats a low bar set by Exondys, but adverse events loom large.
