Nucleic Acid, Nucleoside, or Nucleotide – Page 2

Intellia Pauses Two CRISPR Trials After Severe Liver Toxicity Hospitalizes Patient

1 month ago talkbio0Tagged Clustered Regularly Interspaced Short Palindromic Repeats, Gene Modification, Hospitalization, Intellia, Liver, Pauses, Phase 3 Clinical Trials, Safety, Senile cardiac amyloidosis, Toxicity aspects

Intellia Therapeutics; CRISPR; transthyretin amyloidosis; Phase III trials; gene editing; liver toxicity; patient hospitalization; safety pause

Novartis CEO on $12B Avidity Deal: ‘Could Have Been Twice as Big’

1 month ago talkbio0Tagged Acquisition, Avidity Biosciences, financial outlook, growth strategy, Merger, Neuromuscular Diseases, Novartis, RNA, SpinCo ', Vas Narasimhan

Novartis; Avidity Biosciences; Vas Narasimhan; merger; acquisition; biotech; RNA therapeutics; $12 billion deal; neuromuscular diseases; SpinCo; financial outlook; growth strategy

Moderna Halts Development of CMV Vaccine After Phase 3 Failure

2 months ago talkbio0Tagged Clinical trial failure, Congenital Cytomegalovirus Infection, Cytomegalovirus Infections, Effectiveness, Moderna, mRNA-1647 cytomegalovirus vaccine, Phase 3 trial, Vaccines

Moderna; CMV vaccine; mRNA-1647; Phase 3 trial; Congenital cytomegalovirus; Vaccine efficacy; Clinical trial failure

CHMP Rejects Sanofi’s Rezurock Over Clinical Data Issues, Recommends Approval of BTK Inhibitor Wayrilz

2 months ago talkbio0Tagged Autoimmune thrombocytopenia, belumosudil, cGVHD, CHMP, chronic graft-versus-host disease, Clinical Data, Inosine Triphosphate, Multiple Acyl Coenzyme A Dehydrogenase Deficiency, regulatory approval, Rezurock, Rilzabrutinib, sanofi, Wayrilz

Sanofi; CHMP; EMA; Rezurock; Wayrilz; Belumosudil; Rilzabrutinib; chronic graft-versus-host disease; cGVHD; immune thrombocytopenia; ITP; regulatory approval; clinical data

Bristol Myers Squibb Enters In Vivo CAR-T Field with $1.5B Acquisition of Orbital Therapeutics

2 months ago talkbio0Tagged Acquisition, Autoimmune Diseases, Automobiles, Cell Therapy, immune system reprogramming, in vivo, Myers Squibb, NUSAP1 gene, Obstetric Delivery, Orbital Therapeutics, OTX-201, RNA medicines, RNA, Circular, T-cell therapy

Bristol Myers Squibb; Orbital Therapeutics; in vivo CAR T-cell therapy; autoimmune diseases; RNA medicines; OTX-201; cell therapy; acquisition; LNP delivery; circular RNA; immune system reprogramming

NanoPhoria Secures €83.5M Series A for Heart Failure Therapy; AstraZeneca and Algen Ink Up to $555M AI Drug Discovery Pact

2 months ago talkbio0Tagged AI drug discovery, Algen Biotechnologies, AstraZeneca, Clustered Regularly Interspaced Short Palindromic Repeats, Deal Value, gene modulation, Heart failure, Immunology, nano-in-micro delivery, NanoPhoria, NP-MP1, Series A

NanoPhoria; Series A; heart failure; NP-MP1; nano-in-micro delivery; AstraZeneca; Algen Biotechnologies; AI drug discovery; immunology; CRISPR; gene modulation; deal value

Meeting on the Mesa 2025 Arrives Amid Mixed Signals for Cell and Gene Therapy Sector

2 months ago talkbio0Tagged Advanced therapies, Alternative, Cells, Clinical Trials, commercialization, Gene Modification, gene therapy, Industry, meeting, MESA, partnerships, payment, Phoenix Arizona, regulatory challenges, RNA, Messenger, Study models

Cell and gene therapy; Meeting on the Mesa; Commercialization; Clinical trials; Advanced therapies; Industry partnerships; Regulatory challenges; Phoenix Arizona; mRNA; Gene editing; Alternative payment models

Ansa Biotechnologies Raises $54.4M in Series B to Scale US DNA Synthesis Manufacturing

2 months ago talkbio0Tagged Ansa Biotechnologies, Biotechnology, Cerberus Ventures, DNA Replication, Manufacturing, Series B financing, synthetic construct, U.S.

Ansa Biotechnologies; Series B financing; DNA synthesis; Cerberus Ventures; US manufacturing; biotechnology; synthetic DNA; fundraising

Rezon Bio Launches Two State-of-the-Art CDMO Facilities for Biologics in Europe

2 months ago talkbio0Tagged Biological Factors, Biosimilars, CDMO, Contract agreement, Digital innovation, Europe, Gdańsk, Guanosine Monophosphate, Manufacturing, Multiple Acyl Coenzyme A Dehydrogenase Deficiency, Rezon Bio, United States Food and Drug Administration, Warsaw-Duchnice

Rezon Bio; biologics; CDMO; contract manufacturing; Gdańsk; Warsaw-Duchnice; Europe; digital innovation; GMP manufacturing; biosimilars; EMA; FDA

Ionis Heads to FDA as Antisense Therapy Aces Pivotal Study in Rare Neurological Disease

3 months ago talkbio0Tagged access program, Alexander Disease, Antisense Oligonucleotides, Fast Track, Identifier, Improvement, Infrequent, Ionis Pharmaceuticals, nervous system disorder, orphan drug designation, Phase I – III study, Speed, United States Food and Drug Administration, Zilganersen

Ionis Pharmaceuticals; zilganersen; antisense oligonucleotide; Alexander disease; pivotal Phase I–III study; FDA Fast Track designation; rare neurological disease; expanded access program; walk speed improvement; Orphan Drug designation