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

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

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

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

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

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

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

Intellia Pauses Two CRISPR Trials After Severe Liver Toxicity Hospitalizes Patient

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

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

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

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

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

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

CRISPR Therapeutics’ gene editing therapy reduces bad cholesterol and triglycerides by as much as 80%

Gene Modification, Clustered Regularly Interspaced Short Palindromic Repeats, Triglycerides, LDL Cholesterol Lipoproteins, In Vivo, CTX310, Clinical Trials, cholesterol, Genes