gene therapy – Talk Bio

Zhongmou Therapeutics Reports First-in-Human Success of Optogenetic Gene Therapy ZM-02 for Retinitis Pigmentosa

3 days ago talkbio0Tagged AAV Vector, First-in-Human Clinical Trial, gene therapy, Ophthalmology specialty, Optogenetic Gene Therapy, PsCatCh2.0, Retinitis Pigmentosa, Vision Restoration, Zhongmou, ZM-02

Zhongmou Therapeutics; Optogenetic Gene Therapy; ZM-02; Retinitis Pigmentosa; First-in-Human Clinical Trial; Vision Restoration; AAV Vector; PsCatCh2.0; Gene Therapy; Ophthalmology

FDA OKs Sarepta Study of New Immune Suppression Approach with Elevidys; Gilead Acquires Preclinical TREX1 Program

5 days ago talkbio0Tagged acute liver injury, ELEVIDYS, gene therapy, Gilead, Muscular Dystrophy, Duchenne, sirolimus, Therapeutic immunosuppression, TREX1 gene, United States Food and Drug Administration

FDA; Sarepta; Elevidys; immunosuppression; Duchenne muscular dystrophy; Gilead; TREX1; gene therapy; sirolimus; acute liver injury

FDA Approves Novartis’ Itvisma: Expanding Gene Therapy to Older Spinal Muscular Atrophy Patients

6 days ago talkbio0Tagged Adult, Approved, gene therapy, intrathecal formulation, Itvisma, Novartis, older children, Spinal Muscular Atrophy, United States Food and Drug Administration, Zolgensma

Novartis; FDA approval; Itvisma; Zolgensma; Spinal Muscular Atrophy (SMA); Gene Therapy; Older Children; Adults; Intrathecal Formulation

Spotlight On: Big Pharma’s Big Bets on In Vivo Cell Therapy

2 weeks ago talkbio0Tagged AbbVie, Acquisition, AstraZeneca, Autoimmune Diseases, CAR-T, Cell Therapy, gene therapy, Gilead, in vivo, Manufacturing, Myers Squibb, scalability

in vivo cell therapy; CAR-T; acquisition; autoimmune disease; gene therapy; manufacturing; scalability; Bristol Myers Squibb; Gilead; AbbVie; AstraZeneca

Eli Lilly to Acquire Adverum Biotechnologies in Gene Therapy Deal; Captain T Cell Secures €20M Funding

2 weeks ago talkbio0Tagged Acquisition, Adverum Biotechnologies, biotech, Captain T Cell, contingent value rights, CVR, eli lilly, financing characteristics, gene therapy, Investments, Ixo-vec, Ophthalmology specialty

Eli Lilly; Adverum Biotechnologies; Acquisition; Gene therapy; Ixo-vec; Contingent Value Rights (CVR); Ophthalmology; Captain T Cell; Financing; Biotech investment

FDA Imposes Boxed Warning and Restricts Use of Sarepta’s Elevidys Gene Therapy for Duchenne Muscular Dystrophy

2 weeks ago talkbio0Tagged Black Box Warning, ELEVIDYS, gene therapy, Indication restriction, Liver Failure, Acute, Muscular Dystrophy, Duchenne, Post-marketing study, Safety labeling, United States Food and Drug Administration

Elevidys; Sarepta Therapeutics; Duchenne muscular dystrophy; FDA; Boxed warning; Gene therapy; Acute liver failure; Safety labeling; Indication restriction; Post-marketing study

Eli Lilly Strikes Multiple Deals for Ultra-Rare Eye Disease Gene Therapy and RNAi Partnerships

3 weeks ago talkbio0Tagged AAV-AIPL4, eli lilly, gene therapy, Genetic Leap, Leber congenital amaurosis-4, MeiraGTx, Ophthalmology specialty, partnerships, RNA Interference, SanegeneBio

Eli Lilly; MeiraGTx; gene therapy; Leber congenital amaurosis-4; AAV-AIPL4; Genetic Leap; RNAi; SanegeneBio; ophthalmology; biotech partnerships

uniQure Faces Delay as FDA Walks Back Accelerated Approval for Huntington’s Gene Therapy

4 weeks ago talkbio0Tagged Accelerated, AMT-130, Approved, Clinical Trials, gene therapy, Huntington 's disease, Regulatory Setback, UniQure, United States Food and Drug Administration

uniQure; AMT-130; Huntington’s disease; gene therapy; FDA; accelerated approval; regulatory setback; clinical trial

Otsuka Acquires Asia-Pacific Rights to 4D Molecular Therapeutics’ Retinal Gene Therapy 4D-150 for $85 Million

1 month ago talkbio0Tagged 4D Molecular Therapeutics ', Asia-Pacific, gene therapy, Macular edema due to diabetes mellitus, Ophthalmology specialty, Otsuka, strategic partnership, Wet Age-Related Macular Degeneration

Otsuka; 4D Molecular Therapeutics; 4D-150; Asia-Pacific rights; gene therapy; wet age-related macular degeneration; diabetic macular edema; $85 million; strategic partnership; ophthalmology

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

1 month ago talkbio0Tagged ATTR-CM, ATTR-PN, Clustered Regularly Interspaced Short Palindromic Repeats, gene therapy, Intellia, Liver, MAGNITUDE trial, Nexiguran, Senile cardiac amyloidosis, Study on Hold, Toxicity aspects, United States Food and Drug Administration, ziclumeran

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