Nucleic Acid, Nucleoside, or Nucleotide – Page 6

mRNA manufacturing, risk management, preclinical to clinical transition, quality control, regulatory compliance, raw material selection, process automation, analytical methods.

Revolutionizing AATD Treatment: Emerging Therapies to Replace Augmentation Therapy

8 months ago talkbio0Tagged alpha 1-Antitrypsin Deficiency, Alpha-1, Antitrypsin Deficiency, augmentation therapy, Clustered Regularly Interspaced Short Palindromic Repeats, Gene Modification, Hereditary Diseases, RNA Interference

Alpha-1 Antitrypsin Deficiency (AATD), Genetic Disorder, New Therapies, Augmentation Therapy, RNA Interference (RNAi), Gene Editing, CRISPR

Intellia’s CRISPR Therapy NTLA-2002 Shows 81% Reduction in Hereditary Angioedema Attacks in Phase 2 Study

8 months ago talkbio0Tagged Angioedemas, Hereditary, Clustered Regularly Interspaced Short Palindromic Repeats, Functional, Gene Modification, Intellia, NTLA-2002, Phase 2 Clinical Trials, Swelling attacks

CRISPR therapy, Intellia Therapeutics, NTLA-2002, Hereditary Angioedema (HAE), Gene editing, Phase 2 study, Swelling attacks, Functional cure

Intellia’s CRISPR Gene Editing Therapy Shows Promising Results in Reducing HAE Attacks

8 months ago talkbio0Tagged Angioedemas, Hereditary, Clinical Trials, Clustered Regularly Interspaced Short Palindromic Repeats, Gene Modification, gene therapy, Infrequent, Intellia

Intellia Therapeutics, CRISPR Gene Editing, Hereditary Angioedema (HAE), NTLA-2002, Clinical Trials, Gene Therapy, Rare Diseases

Roche and Dyno Therapeutics Launch $1B+ Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Neurological Diseases

8 months ago talkbio0Tagged AAVS1 gene, Cloning Vectors, Collaboration, Dyno, gene delivery process, gene therapy, LEAP, Neurological diseases, Roche (company), Technology

Roche, Dyno Therapeutics, AAV gene therapy vectors, Neurological diseases, AI-driven gene delivery, LEAP technology, Gene therapy collaboration

Sangamo Therapeutics Fast-Tracks Fabry Gene Therapy with FDA Accelerated Approval Pathway

8 months ago talkbio0Tagged Accelerated, approval pathway, Fabry Disease, gene therapy, Isaralgagene civaparvovec, Sangamo, ST-920, STAAR, United States Food and Drug Administration

Sangamo Therapeutics, Fabry disease, gene therapy, isaralgagene civaparvovec, FDA, accelerated approval pathway, ST-920, STAAR study

Roche Abandons Tau Antibody Project, Returns Rights to UCB After $120M Investment

8 months ago talkbio0Tagged Alzheimer 's disease, Antibodies, Bepranemab, Investments, Roche (company), UCB, uridine triacetate

Roche, UCB, tau antibody, Alzheimer’s disease, bepranemab, $120M investment