Boehringer will leverage ExpressionEdits' Genetic Syntax Engine which generates optimized introns to increase protein expression.

ExpressionEdits' Genetic Syntax Engine platform is designed to enhance gene expression within gene therapies by restoring introns.

One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...

Orchard Therapeutics has tasked Robin Kenselaar with accelerating growth of Lenmeldy, the world’s most expensive drug, ...

Adverum Biotechnologies Inc. (NASDAQ:ADVM) stock has reached a 52-week low, dipping to $3.95, with a market capitalization of just $83.2 million. According to InvestingPro analysis, the company faces ...

Robin Fields ProPublica is a nonprofit newsroom that investigates abuses of power. Sign up to receive our biggest stories as soon as they’re published. Vincent Gaynor remembers, almost to the minute, ...

GenSight Biologics Announces Five-Year Efficacy and Safety Results for LUMEVOQ® Gene Therapy at the Conclusion of the REFLECT Study ...

Kyle Registre's childhood dream of becoming a pilot can finally come true now that he says he's been cured of sickle cell complications.

This is an important and solid study that examines the role of TFAM, a protein that helps maintain mtDNA, in mtDNA mutator mice. The authors have demonstrated that TFAM's counteractive role in mtDNA ...

Researchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...

Scientists in the polo-obsessed nation have produced world's first genetically edited horses, designed to outrun champion ...