Gene Therapy - News, Trends & Market Analysis - Benzinga

Gene Therapy

Gene therapy is a biomedical approach that involves modifying or replacing genes within a patient's cells to treat or prevent disease. It can be used to correct genetic defects, introduce beneficial genes, or silence harmful ones. Advances in biotechnology and molecular medicine have expanded its potential applications, from rare inherited disorders to cancer and cardiovascular disease. Investors and researchers closely monitor developments in gene therapy for its transformative impact on healthcare and the pharmaceutical industry.

Congressman Who Was Top 2025 Stock Picker Buys Shares Of $5M Company You've Never Heard Of

Congressman Who Was Top 2025 Stock Picker Buys Shares Of $5M Company You've Never Heard Of

This Medical Stock Soared Over 80% In Pre-Market After Advancing Gene Therapy Program For ALS, Alzheimer's And Parkinson's Diseases

This Medical Stock Soared Over 80% In Pre-Market After Advancing Gene Therapy Program For ALS, Alzheimer's And Parkinson's Diseases

16-Minute Gene Therapy Restores Hearing To British Baby, A World First

16-Minute Gene Therapy Restores Hearing To British Baby, A World First

This Medical Stock Soared Over 80% In Pre-Market After Advancing Gene Therapy Program For ALS, Alzheimer's And Parkinson's Diseases

This Medical Stock Soared Over 80% In Pre-Market After Advancing Gene Therapy Program For ALS, Alzheimer's And Parkinson's Diseases

16-Minute Gene Therapy Restores Hearing To British Baby, A World First

16-Minute Gene Therapy Restores Hearing To British Baby, A World First

Second Death - Pfizer Reports Young Boy's Death After One Year Of Gene Therapy Treatment In Muscle Wasting Disorder Trial

Second Death - Pfizer Reports Young Boy's Death After One Year Of Gene Therapy Treatment In Muscle Wasting Disorder Trial

Gene Therapy Side Effects: Verve Therapeutics Halts Enrollment Of Lead Gene Therapy Trial In Patients With Bad Cholesterol

Gene Therapy Side Effects: Verve Therapeutics Halts Enrollment Of Lead Gene Therapy Trial In Patients With Bad Cholesterol

Second Death - Pfizer Reports Young Boy's Death After One Year Of Gene Therapy Treatment In Muscle Wasting Disorder Trial

Second Death - Pfizer Reports Young Boy's Death After One Year Of Gene Therapy Treatment In Muscle Wasting Disorder Trial

Gene Therapy Side Effects: Verve Therapeutics Halts Enrollment Of Lead Gene Therapy Trial In Patients With Bad Cholesterol

Gene Therapy Side Effects: Verve Therapeutics Halts Enrollment Of Lead Gene Therapy Trial In Patients With Bad Cholesterol

Astellas Pharma Licenses Promising Gene Therapy KT430 from Kate Therapeutics Amidst Clinical Hurdles

Astellas Pharma Licenses Promising Gene Therapy KT430 from Kate Therapeutics Amidst Clinical Hurdles

FDA AdComm Narrowly Supports Approval Of Sarepta's Gene Therapy For Rare Neuromuscular Disorder

FDA AdComm Narrowly Supports Approval Of Sarepta's Gene Therapy For Rare Neuromuscular Disorder

Gene Therapy Recent News

$2M Price Tag for Sickle Cell Gene Therapies Could Be Cost-Effective Under Certain Scenarios, Finds ICER Analysis

$2M Price Tag for Sickle Cell Gene Therapies Could Be Cost-Effective Under Certain Scenarios, Finds ICER Analysis

6 Questions With Rocket Pharma's CEO On World Health Day: How The Biotech Is 'Changing The Narrative'

6 Questions With Rocket Pharma's CEO On World Health Day: How The Biotech Is 'Changing The Narrative'

Rocket Pharma (NASDAQ: RCKT) CEO Gaurav Shah is on a mission to "change the narrative" when it comes to rare diseases.

Tesla, Meta, Thermogenesis, Adobe, UiPath: Why These 5 Stocks Are Drawing Investors' Attention Today

Tesla, Meta, Thermogenesis, Adobe, UiPath: Why These 5 Stocks Are Drawing Investors' Attention Today

Regenxbio Says Diabetic Retinopathy Gene Therapy Shows Meaningful Improvements In Disease Severity

Regenxbio Says Diabetic Retinopathy Gene Therapy Shows Meaningful Improvements In Disease Severity

What Happened With Ocugen Shares During Wednesday's After-Hours Session

What Happened With Ocugen Shares During Wednesday's After-Hours Session

Ocugen Inc (NASDAQ: OCGN) shares traded marginally higher by 1.22% at $1.66 during Wednesday's after-hours session.

This COVID-19 Shot Stock Could Have Eye Disease Gene Therapy As Significant Driver, Mizuho Analyst Says

This COVID-19 Shot Stock Could Have Eye Disease Gene Therapy As Significant Driver, Mizuho Analyst Says

Bluebird's Gene Therapy Scores US Approval For Genetic Blood Disorder, Probably The Most Expensive

Bluebird's Gene Therapy Scores US Approval For Genetic Blood Disorder, Probably The Most Expensive

Homology Medicines Announces Preclinical Gene Therapy Candidate For Genetic Storage Disorder

Homology Medicines Announces Preclinical Gene Therapy Candidate For Genetic Storage Disorder

Homology Medicines Inc (NASDAQ: FIXX) announced the details of HMI-204, its optimized, in vivo, one-time gene therapy candidate for metachromatic leukodystrophy (MLD).

This Penny Stock Touts Encouraging Long-Term Data For Hemophilia Gene Therapy

This Penny Stock Touts Encouraging Long-Term Data For Hemophilia Gene Therapy

Small biotech Freeline Therapeutics Holdings plc (NASDAQ: FRLN) hopes new long-term data will help boost its case.

Adverum Posts New Data From Wet AMD Gene Therapy Trial

Adverum Posts New Data From Wet AMD Gene Therapy Trial

Sarepta Reports One Heart Inflammation Case In Duchenne Gene Therapy Trial

Sarepta Reports One Heart Inflammation Case In Duchenne Gene Therapy Trial

MeiraGTx Posts Encouraging Gene Therapy Data From Eye Disorder Trial

MeiraGTx Posts Encouraging Gene Therapy Data From Eye Disorder Trial

Astellas's Pompe Disease Gene Therapy Trial Put On FDA Clinical Hold

Astellas's Pompe Disease Gene Therapy Trial Put On FDA Clinical Hold

UniQure Shares Are Up After Low-Dose Cohort Data From Huntington's Gene Therapy Trial

UniQure Shares Are Up After Low-Dose Cohort Data From Huntington's Gene Therapy Trial

BMO Capital Says Risk/Reward For This Gene Stock Assumes Clinical Success

BMO Capital Says Risk/Reward For This Gene Stock Assumes Clinical Success

Analysts Consider CRISPR Therapeutics' Blood Disorder Trial Data Encouraging, Impressive

Analysts Consider CRISPR Therapeutics' Blood Disorder Trial Data Encouraging, Impressive

CRISPR Therapeutics (NASDAQ: CRSP) presented results from its ongoing Phase 1 COBALT-LYM CTX130 (exa-cel) trial, an allogeneic CAR-T cell therapy targeting CD70 for solid tumors and certain hematologic malignancies.

Mizuho Comments On UniQure's Upcoming Low-Dose Huntington's Gene Therapy Trial Data

Mizuho Comments On UniQure's Upcoming Low-Dose Huntington's Gene Therapy Trial Data

The much-awaited one-year data for the entire low-dose cohort for UniQure NV's (NASDAQ: QURE) AMT-130 gene therapy in Huntington's disease is expected at any time.

Mustang Bio Posts Encouraging Interim Data From Gene Therapy Trial In Immunodeficiency

Mustang Bio Posts Encouraging Interim Data From Gene Therapy Trial In Immunodeficiency

Rocket Pharma's Gene Therapy Shows 100% Overall Survival In Immune Deficiency Disorder

Rocket Pharma's Gene Therapy Shows 100% Overall Survival In Immune Deficiency Disorder

Avrobio's Pompe Disease Gene Therapy Shows Efficacy, Safety In Preclinical Study

Avrobio's Pompe Disease Gene Therapy Shows Efficacy, Safety In Preclinical Study

AVROBIO Shares Jumps On Encouraging Data From Early-Stage Gene Therapy Study

AVROBIO Shares Jumps On Encouraging Data From Early-Stage Gene Therapy Study

The Daily Biotech Pulse: Pfizer-BioNTech's COVID-19 Booster For Kids 5-11 Coming Soon, Ultragenyx In-Licenses Gene Therapy For Genetic Disorder, Ampio's AP-013 Trial Under Independent Investigation

The Daily Biotech Pulse: Pfizer-BioNTech's COVID-19 Booster For Kids 5-11 Coming Soon, Ultragenyx In-Licenses Gene Therapy For Genetic Disorder, Ampio's AP-013 Trial Under Independent Investigation

Here's a roundup of top developments in the biotech space over the last 24 hours:

Applied Genetic's Vision Loss Gene Therapy Shows Encouraging Safety, Efficacy At Three Months

Applied Genetic's Vision Loss Gene Therapy Shows Encouraging Safety, Efficacy At Three Months