Health ❯ Healthcare ❯ Medical Research

Gene Therapy

Huntington's Disease CRISPR Technology Clinical Trials Hearing Improvement Duchenne Muscular Dystrophy Spinal Muscular Atrophy Rare Diseases Cellular Reprogramming Metachromatic Leukodystrophy Gene Regulation

3 ARTICLES

2w ago

Journal and University Open Probes After Reporting Reveals Child’s Death and Family Funding in Gene‑editing Study

Revelations that a family paid $860,000 and a child died after an experimental treatment have prompted scrutiny of the paper’s data, disclosure and clinical oversight.

3 ARTICLES

2w ago

Researcher Investigated for Concealing Child’s Death After Experimental Gene‑Editing Trial

4 ARTICLES

2w ago

Undisclosed Patient Death in Shanghai Base‑Editing Trial Exposes Oversight and Reporting Failures

7 ARTICLES

last mo.

FDA Agrees Three‑Year Phase I/II Data Can Support uniQure’s AMT‑130 Accelerated BLA

4 ARTICLES

2mo ago

FDA Grants Accelerated Approval to Kresladi, First Gene Therapy for Severe Pediatric LAD-1

3 ARTICLES

5mo ago

Brazil Treats First Infant in In‑Country Gene Therapy Trial for Severe Spinal Muscular Atrophy

10 ARTICLES

5mo ago

FDA Proposes ‘Plausible Mechanism’ Pathway for Individualized Rare-Disease Therapies

3 ARTICLES

8mo ago

FDA Approves One-Time Intrathecal Gene Therapy Itvisma for Older SMA Patients

3 ARTICLES

8mo ago

Exa‑cel Posts Complete Primary-End Point Responses in Early Pediatric Trials, With One Fatal Busulfan Event

3 ARTICLES

8mo ago

FDA Meeting Minutes Undercut uniQure’s Plan to File Huntington’s Gene Therapy for Approval

4 ARTICLES

9mo ago

Single-Infusion CRISPR Therapy Lowers LDL and Triglycerides in First Human Trial

12 ARTICLES

9mo ago

One-Time CRISPR Edit Halves LDL and Triglycerides in Early Human Trial

11 ARTICLES

10mo ago

NEJM Study Finds 95% Long-Term Success for ADA-SCID Gene Therapy

3 ARTICLES

11mo ago

Capsida Halts STXBP1 Gene Therapy Trial After First Pediatric Participant Dies

3 ARTICLES

this yr.

Kidney Organoids Reveal NFκB‑Linked AAV2 Toxicity, With Bardoxolone Methyl Mitigating Damage

5 ARTICLES

last yr.

Dual-Vector Gene Therapy Restores Sight in Usher Syndrome Patient; Trial Advances to Higher Doses

Gene Therapy

Journal and University Open Probes After Reporting Reveals Child’s Death and Family Funding in Gene‑editing Study

Researcher Investigated for Concealing Child’s Death After Experimental Gene‑Editing Trial

Undisclosed Patient Death in Shanghai Base‑Editing Trial Exposes Oversight and Reporting Failures

FDA Agrees Three‑Year Phase I/II Data Can Support uniQure’s AMT‑130 Accelerated BLA

Life Biosciences Doses First Patient With Eye Reprogramming Gene Therapy

Single Infusion Gene Editing Lowers LDL by Up to 62% in Early Trial

CRISPR Enzyme Cas12a2 Selectively Destroys Cells Based on RNA in Lab Tests

Roche Starts Placebo-Controlled Phase 3 Trial of Elevidys to Pursue EU Approval

FDA Grants Accelerated Approval to Kresladi, First Gene Therapy for Severe Pediatric LAD-1

Brazil Treats First Infant in In‑Country Gene Therapy Trial for Severe Spinal Muscular Atrophy

Never miss stories about

Gene Therapy

FDA Proposes ‘Plausible Mechanism’ Pathway for Individualized Rare-Disease Therapies

FDA Approves One-Time Intrathecal Gene Therapy Itvisma for Older SMA Patients

Exa‑cel Posts Complete Primary-End Point Responses in Early Pediatric Trials, With One Fatal Busulfan Event

FDA Meeting Minutes Undercut uniQure’s Plan to File Huntington’s Gene Therapy for Approval

Single-Infusion CRISPR Therapy Lowers LDL and Triglycerides in First Human Trial

One-Time CRISPR Edit Halves LDL and Triglycerides in Early Human Trial

NEJM Study Finds 95% Long-Term Success for ADA-SCID Gene Therapy

Capsida Halts STXBP1 Gene Therapy Trial After First Pediatric Participant Dies

Kidney Organoids Reveal NFκB‑Linked AAV2 Toxicity, With Bardoxolone Methyl Mitigating Damage

Dual-Vector Gene Therapy Restores Sight in Usher Syndrome Patient; Trial Advances to Higher Doses

Never miss stories about

Gene Therapy