Nucleic Acid, Nucleoside, or Nucleotide

Biogen, Stoke Boost Epilepsy Drug’s Blockbuster Potential With Early Seizure Reduction Data

2 days ago talkbio0Tagged American Epilepsy Society 2025, Antisense Oligonucleotides, Biogen, blockbuster drug, EMPEROR Phase III trial, Epilepsy, Infantile Severe Myoclonic Epilepsy, Jefferies analysts, natural history controls, SCN1A gene, Seizures, Stoke Therapeutics, Zorevunersen

Biogen; Stoke Therapeutics; zorevunersen; Dravet syndrome; epilepsy; antisense oligonucleotide; seizure reduction; blockbuster drug; EMPEROR Phase III trial; American Epilepsy Society 2025; Jefferies analysts; natural history controls; SCN1A

Profluent Raises $106M to Accelerate Frontier AI Models for Programmable Biology, Backed by Jeff Bezos

3 weeks ago talkbio0Tagged AI biotech, Altimeter Capital, Bezos Expeditions, Biological Factors, Clustered Regularly Interspaced Short Palindromic Repeats, Drug Development, Funding, Jeff, Profluent, Programmable Biology, Protein Atlas, protein design, Synthetic Biology

Profluent; AI biotech; protein design; programmable biology; Jeff Bezos; Altimeter Capital; $106M funding; Bezos Expeditions; CRISPR; biologics; Protein Atlas; synthetic biology; drug development

Arrowhead Secures Maiden FDA Approval for Redemplo in Rare Genetic Metabolic Disorder (FCS)

3 weeks ago talkbio0Tagged Approved, Arrowhead Pharmaceuticals, commercial, Familial chylomicronemia syndrome, Hereditary Diseases, Infrequent, Interferes with, Plozasiran, Redemplo, RNA, Triglycerides, TRiM platform, United States Food and Drug Administration

Arrowhead Pharmaceuticals; FDA approval; Redemplo; plozasiran; familial chylomicronemia syndrome; rare genetic disorder; RNA interference; TRiM platform; triglyceride reduction; first commercial drug

Pfizer Reduces Stake in BioNTech; Candel Pauses Pancreatic Cancer Program

3 weeks ago talkbio0Tagged Acquisition, BioNTech, Comirnaty, expansion, Metsera, mRNA Vaccine, Pancreatic carcinoma, Pfizer, pipeline

Pfizer; BioNTech; stake reduction; Candel Therapeutics; pancreatic cancer; Comirnaty; mRNA vaccine; Metsera acquisition; pipeline expansion

FDA Unveils ‘Plausible Mechanism Pathway’ to Accelerate Personalized Therapies, Inspired by Baby KJ

4 weeks ago talkbio0Tagged Baby KJ, Clustered Regularly Interspaced Short Palindromic Repeats, Gene Modification, Infrequent, mechanism pathway, personalized therapies, regulatory innovation, United States Food and Drug Administration

FDA; personalized therapies; gene editing; plausible mechanism pathway; Baby KJ; rare diseases; CRISPR; regulatory innovation

Voyager Therapeutics Partners with Transition Bio for Small Molecule Neurodegenerative Drug Discovery; CRISPR Therapeutics Ends Work on CAR-T Therapy

4 weeks ago talkbio0Tagged Amyotrophic Lateral Sclerosis, CAR-T therapy, Clustered Regularly Interspaced Short Palindromic Repeats, Drug Development, Neurodegenerative Disorders, Partnership, Pick Disease of the Brain, small molecule drug discovery, TARDBP gene, Transition Bio, Voyager Therapeutics

Voyager Therapeutics; Transition Bio; ALS; frontotemporal dementia; TDP-43; small molecule drug discovery; neurodegenerative diseases; CRISPR Therapeutics; CAR-T therapy; partnership; drug development

Ionis Claims Curative Potential for Tryngolza in Severe Triglyceride Disease

4 weeks ago talkbio0Tagged Antisense Oligonucleotides, curative potential, Hypertriglyceridemia, Ionis, Pancreatitis, Acute, Phase 3 Clinical Trials, RNA-targeted therapy, Severe (severity modifier), Submission, Triglycerides, TRYNGOLZA, United States Food and Drug Administration

Ionis; Tryngolza; severe hypertriglyceridemia; curative potential; triglyceride reduction; acute pancreatitis; antisense oligonucleotide; FDA submission; Phase III trial; RNA-targeted therapy

Jennifer Doudna’s Azalea Therapeutics Launches with $82M to Advance In Vivo CAR-T Using Novel CRISPR Delivery Technology

1 month ago talkbio0Tagged Autoimmune Diseases, Azalea Therapeutics, B-cell malignancies, Cancer Therapeutic Procedure, CAR-T, Clustered Regularly Interspaced Short Palindromic Repeats, EDV technology, Funding, Gene Modification, in vivo, Jennifer Doudna, Series A

Jennifer Doudna; Azalea Therapeutics; CRISPR; in vivo CAR-T; gene editing; EDV technology; cancer therapy; autoimmune disease; Series A funding; B-cell malignancies

Wave Life Sciences Obesity Biomarker Data Raise Hopes for Future Readouts

1 month ago talkbio0Tagged Activin E :* *, Biological Markers, Body Composition, Clinical Trials, INHBE gene, INLIGHT trial, Obesity, RNA therapeutics, RNA, Small Interfering, Wave Life Sciences, Weight Loss, WVE-007

Wave Life Sciences; WVE-007; obesity; biomarker data; siRNA; Activin E; INHBE gene; RNA therapeutics; clinical trial; body composition; weight loss; INLIGHT trial

FDA Places Clinical Hold on Two Intellia CRISPR Trials After Life-Threatening Liver Toxicity Case

1 month ago talkbio0Tagged ATTR-CM, ATTR-PN, Clustered Regularly Interspaced Short Palindromic Repeats, gene therapy, Intellia, Liver, MAGNITUDE trial, Nexiguran, Senile cardiac amyloidosis, Study on Hold, Toxicity aspects, United States Food and Drug Administration, ziclumeran

FDA; Intellia; CRISPR; clinical hold; liver toxicity; nexiguran ziclumeran; MAGNITUDE trial; transthyretin amyloidosis; ATTR-CM; ATTR-PN; gene therapy