OTS Member Highlight – Keith T. Gagnon, PhD
As a child, before Keith Gagnon knew anything about science, he told his mom he wanted to be a scientist when he grew up. Now, decades later, Dr. Keith Gagnon is a biochemist and molecular biologist specializing in RNA biology, nucleic acid therapeutics, repeat expansion disorders, and CRISPR-Cas systems, serving as an Associate Professor in the Department of Biochemistry ...
Interesting Findings on the Impact of Antisense Oligonucleotides on Genome Stability and DNA-Repair Enzyme Activation
Antisense oligonucleotides (ASOs) have emerged as promising drug candidates for the treatment of genetic diseases. Because of their ability to target virtually any disease-related gene product, several ASO drugs have been approved, and many more are in development (1). While they remain among the most precise therapies, investigations are ongoing to find improvements and increase safety. A recent study from ...
Beyond the “Too Rare” Barrier: Platforms for Rare Mutations Leverage New Regulatory Tool
In November, Food and Drug Administration (FDA) officials introduced the Plausible Mechanism Pathway, a regulatory tool designed to make it easier to approve treatments — specifically investigational gene therapies — for rare conditions. An article published in The New England Journal of Medicine (1) discussed the successful treatment of KJ Muldoon, a baby born with a rare, life-threatening liver ...
Phase 3 Trials of Intellia’s Nex-z CRISPR Therapy Resume after the Clinical Holds are Lifted
Intellia Therapeutics recently announced that the U.S. Food and Drug Administration (FDA) has lifted the hold on its clinical trial for the investigational CRISPR-based gene-editing therapy, nexiguran ziclumeran, also known as nex-z or NTLA-2001. The four-month hold was implemented after a patient in the MAGNITUDE study experienced severe liver toxicity, specifically elevated liver transaminases and increased total bilirubin, in ...
OTS Member Highlight – Dr. Timothy Yu
The sometimes twisty, sometimes intuitive, sometimes amazingly elegant hidden logic of the natural world has always fascinated Dr. Timothy Yu. Knowledge we now take for granted — the genetic code, machinery of transcription, or the layered biological logic of developmental patterning — were once completely mysterious. The ability to discover this hidden knowledge and apply it in the real ...
FDA’s Plausible Mechanism Pathway for Personalized Therapies to Treat Rare Diseases
The clock was ticking as soon as baby KJ was born in the summer of 2024. Within two days of his birth, he was lethargic and struggled to breathe. His blood test results showed elevated ammonia levels, leading to a diagnosis of carbamoyl-phosphate synthetase 1 (CPS1) deficiency. This ultra-rare disease affects around 1 in 1,300,000 individuals, and only half ...
OTS Member Highlight – Keith T. Gagnon, PhD
As a child, before Keith Gagnon knew anything about science, he told his mom he ...
Interesting Findings on the Impact of Antisense Oligonucleotides on Genome Stability and DNA-Repair Enzyme Activation
Antisense oligonucleotides (ASOs) have emerged as promising drug candidates for the treatment of genetic diseases. Because ...
Beyond the “Too Rare” Barrier: Platforms for Rare Mutations Leverage New Regulatory Tool
In November, Food and Drug Administration (FDA) officials introduced the Plausible Mechanism Pathway, a regulatory ...
Phase 3 Trials of Intellia’s Nex-z CRISPR Therapy Resume after the Clinical Holds are Lifted
Intellia Therapeutics recently announced that the U.S. Food and Drug Administration (FDA) has lifted the ...
OTS Member Highlight – Dr. Timothy Yu
The sometimes twisty, sometimes intuitive, sometimes amazingly elegant hidden logic of the natural world has ...
FDA’s Plausible Mechanism Pathway for Personalized Therapies to Treat Rare Diseases
The clock was ticking as soon as baby KJ was born in the summer of ...
