Gene Modification

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

2 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

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

3 weeks 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

Intellia Pauses Two CRISPR Trials After Severe Liver Toxicity Hospitalizes Patient

4 weeks 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

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

Editas Medicine Reboots With Cholesterol Gene Therapy (EDIT-401) as Lead Asset

3 months ago talkbio0Tagged Clinical Development, EDIT-401, Editas Medicine, Gene Modification, gene therapy, Hyperlipidemia, in vivo, LDL Cholesterol Lipoproteins, LDLR gene, strategic pivot

Editas Medicine; EDIT-401; gene therapy; hyperlipidaemia; LDL cholesterol; LDLR gene; in vivo gene editing; strategic pivot; clinical development

Eli Lilly Pays $1B Upfront to Acquire Verve Therapeutics, Expanding Cardiovascular Gene Editing Portfolio

5 months ago talkbio0Tagged Acquisition, Cardiovascular Diseases, eli lilly, Gene Modification, Heart Diseases, PCSK9 protein, human, VERVE-102

Eli Lilly; Verve Therapeutics; gene editing; cardiovascular disease; acquisition; PCSK9; VERVE-102; biotech; heart disease

Analysts Downplay Intellia Adverse Event, but Gene Therapy Safety Remains Under Scrutiny

6 months ago talkbio0Tagged Adverse event, ATTR-CM, Event, Gene Modification, gene therapy, Intellia, liver transaminase, MAGNITUDE trial, Response process, Safety, Stock Decline

Intellia Therapeutics; gene therapy; safety event; adverse event; MAGNITUDE trial; liver transaminase; stock decline; ATTR-CM; gene editing; analyst response

Post-Hoc: AI bio needs to heed the overhyped history of CRISPR

6 months ago talkbio0Tagged Artificial Intelligence, Clustered Regularly Interspaced Short Palindromic Repeats, Gene Modification, overhype, reproductive health biotech

CRISPR; AI in biotech; overhype; gene editing; reproductive health biotech

CRISPR Therapeutics Partners with Sirius for siRNA Therapies, Expanding Beyond Gene Editing

6 months ago talkbio0Tagged Anticoagulants, biotech partnerships, Cardiovascular Diseases, Clustered Regularly Interspaced Short Palindromic Repeats, Factor XI, Gene Modification, RNA, Small Interfering, Sirius, SRSD107, thrombotic disease

CRISPR Therapeutics; Sirius Therapeutics; siRNA; gene editing; SRSD107; cardiovascular diseases; thrombotic disease; biotech partnerships; Factor XI; anticoagulant therapies

Federal Appeals Court Revives CRISPR Patent Dispute Between Broad Institute and University of California

7 months ago talkbio0Tagged Broad Institute, Clustered Regularly Interspaced Short Palindromic Repeats, Eukaryotic Cells, Federal Circuit, Gene Modification, Intellectual Property, Legal patent, PTAB, University of California

CRISPR; patent dispute; Broad Institute; University of California; Federal Circuit; PTAB; gene editing; intellectual property; eukaryotic cells