Targeting the Kidney: The Promise of RNA-Based Therapeutics
When Yessenia Gutierrez was just nine years old, her kidneys failed; at 10 years old, she received her first kidney transplant. Gutierrez was born with polycystic kidney disease (PKD), a genetic condition that makes fluid-filled cysts grow on the kidney and can lead to kidney failure. Growing up, Gutierrez had to stop attending school due to her frequent doctor ...
2024 FDA Approvals: A Wave of Innovation in Treating Serious Diseases
In 2024, the Food and Drug Administration Center for Drug Evaluation (CDER) approved 50 new small molecules, biologics, and oligonucleotide therapies (1). The approvals — the second largest in 30 years — include an oligonucleotide therapy for blood cancer, an antisense oligonucleotide shown to significantly drop triglyceride levels, and an mRNA vaccine targeting the respiratory syncytial virus.  IMETELSTAT: ...
Transporting Therapeutics: A Novel Approach to Deliver ASOs to the Brain
Antisense oligonucleotide (ASO) therapies, which bind to RNA and modify protein expression, are promising drugs for treating neurological conditions. However, their inability to cross the blood-brain barrier (BBB) sometimes makes injection directly into the cerebral spinal fluid (via intrathecal injection) necessary, which is not without its risks or drawbacks (1). However, a recent study by Denali Therapeutics, as well ...
Oligonucleotide Treatment Advances Offer Relief for Patients with Familial Chylomicronemia Syndrome
At just three weeks old, Darlene was sent to the emergency room after her mom had brought her to a pediatrician. The baby wouldn't stop crying and wasn't eating. Concerningly, her triglyceride levels were in the 20,000's, far over the normal 150 level. Darlene was referred to a lipid specialist at John Hopkins University Hospital and, after being genetically ...
The Man Behind the Molecules: John Maraganore’s RNAi Legacy
The 2024 OTS lifetime achievement winner, John Maraganore, is best known for his pioneering role in making RNA interference (RNAi) a successful therapeutic modality while CEO at Alnylam Pharmaceuticals. However, before joining and after leaving Alnylam, he was and continues to be influential in the biotechnology sphere, with his leadership skills, optimism, and willingness to take risks still changing ...
Bridge Editing: A Novel Technique for Large-Scale DNA Modifications
Link Source: Hiraizumi, M., Perry, N.T., Durrant, M.G. et al. Structural mechanism of bridge RNA-guided recombination. Nature 630, 994–1002 (2024). Gene editing has swiftly advanced since CRISPR was discovered, with CRISPR being developed not only to provide therapeutic gene editing treatments for humans but also for uses such as basic research, as a diagnostic tool, and for large-scale genetic screens, as well ...
Targeting the Kidney: The Promise of RNA-Based Therapeutics
When Yessenia Gutierrez was just nine years old, her kidneys failed; at 10 years old, ...
2024 FDA Approvals: A Wave of Innovation in Treating Serious Diseases
In 2024, the Food and Drug Administration Center for Drug Evaluation (CDER) approved 50 new ...
Transporting Therapeutics: A Novel Approach to Deliver ASOs to the Brain
Antisense oligonucleotide (ASO) therapies, which bind to RNA and modify protein expression, are promising drugs ...
Oligonucleotide Treatment Advances Offer Relief for Patients with Familial Chylomicronemia Syndrome
At just three weeks old, Darlene was sent to the emergency room after her mom ...
The Man Behind the Molecules: John Maraganore’s RNAi Legacy
The 2024 OTS lifetime achievement winner, John Maraganore, is best known for his pioneering role ...
Bridge Editing: A Novel Technique for Large-Scale DNA Modifications
Link Source: Hiraizumi, M., Perry, N.T., Durrant, M.G. et al. Structural mechanism of bridge RNA-guided recombination. Nature 630, ...
