Gene Modification

FDA Lifts Clinical Hold on Intellia’s MAGNITUDE-2 Phase 3 Trial for Nex-Z in ATTRv-PN

1 day ago talkbio0Tagged ATTRv-PN, Clustered Regularly Interspaced Short Palindromic Repeats, Gene Modification, Intellia, MAGNITUDE-2, nex-z, Study on Hold, United States Food and Drug Administration

FDA; Intellia Therapeutics; clinical hold; MAGNITUDE-2; nex-z; ATTRv-PN; gene editing; CRISPR

Eli Lilly Partners with Seamless Therapeutics on Up to $1.12B Recombinase Gene Editing Deal for Hearing Loss

2 days ago talkbio0Tagged Collaboration, Deafness, Eli, Eli Lilly, Gene Modification, recombinase

Eli Lilly; Seamless Therapeutics; recombinase; gene editing; hearing loss; collaboration; $1.12B

CRISPR Pioneers Jennifer Doudna and Fyodor Urnov Launch Aurora Therapeutics for Custom CRISPR Therapies

3 weeks ago talkbio0Tagged Aurora Therapeutics, Clustered Regularly Interspaced Short Palindromic Repeats, Fyodor, Gene Modification, Infrequent, Jennifer Doudna, Menlo Ventures, personalized therapies

Aurora Therapeutics; CRISPR; gene editing; personalized therapies; Jennifer Doudna; Fyodor Urnov; rare diseases; Menlo Ventures

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

3 months 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 months 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

3 months 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

4 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

5 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

8 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

8 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