Precision Medicine

What Three Gene-Therapy Deaths Have in Common - The Atlantic

Fri, 16 Oct 2026 11:30:00 GMT

What Three Gene-Therapy Deaths Have in Common The Atlantic

Researchers test gene therapy as less invasive treatment for hydrocephalus - University of Cincinnati

Tue, 20 Oct 2026 18:27:27 GMT

Researchers test gene therapy as less invasive treatment for hydrocephalus University of Cincinnati

AAV gene therapy could provide a less invasive hydrocephalus treatment - RegMedNet

Tue, 20 Oct 2026 15:36:42 GMT

AAV gene therapy could provide a less invasive hydrocephalus treatment RegMedNet

New gene therapy gives some kids born deaf the ability to hear - NBC News

Tue, 20 Oct 2026 18:35:02 GMT

New gene therapy gives some kids born deaf the ability to hear NBC News

Blind Patients Recover Partial Vision in Gene Therapy Trial - SingularityHub

Tue, 20 Oct 2026 21:05:34 GMT

Blind Patients Recover Partial Vision in Gene Therapy Trial SingularityHub

Gene editing is coming for common diseases. Are we ready? - Michigan Medicine

Mon, 19 Oct 2026 12:00:00 GMT

Gene editing is coming for common diseases. Are we ready? Michigan Medicine

A Radical Proposal to Preserve Cell and Gene Therapy Innovation - MedCity News

Tue, 20 Oct 2026 13:17:25 GMT

A Radical Proposal to Preserve Cell and Gene Therapy Innovation MedCity News

RGX-202 Duchenne Gene Therapy Met Its Phase 3 Goal as New Data Detail Two Resolved Serious Side Effects - Medical Daily

Tue, 20 Oct 2026 11:00:20 GMT

RGX-202 Duchenne Gene Therapy Met Its Phase 3 Goal as New Data Detail Two Resolved Serious Side Effects Medical Daily

New gene therapy helps doctors restore hearing for children born deaf - WLWT

Thu, 15 Oct 2026 03:22:00 GMT

New gene therapy helps doctors restore hearing for children born deaf WLWT

Solid Biosciences to Present Research Across Gene Therapy - GlobeNewswire

Tue, 20 Oct 2026 12:00:00 GMT

Solid Biosciences to Present Research Across Gene Therapy GlobeNewswire

Dog cognitive decline treatment: USF Health develops new gene therapy - FOX 13 Tampa Bay

Tue, 20 Oct 2026 01:48:38 GMT

Dog cognitive decline treatment: USF Health develops new gene therapy FOX 13 Tampa Bay

Trial of gene therapy for CLN7 Batten disease coming to Canada - Batten Disease News

Mon, 19 Oct 2026 16:30:16 GMT

Trial of gene therapy for CLN7 Batten disease coming to Canada Batten Disease News

New Gene Therapy Helps Doctors Bring Hearing to Children Born Deaf - People.com

Thu, 15 Oct 2026 23:26:39 GMT

New Gene Therapy Helps Doctors Bring Hearing to Children Born Deaf People.com

UCLA Gene Therapy Pioneer Dr. Donald Kohn Elected to National Academy of Medicine | Newswise - Newswise

Mon, 19 Oct 2026 23:10:00 GMT

UCLA Gene Therapy Pioneer Dr. Donald Kohn Elected to National Academy of Medicine | Newswise Newswise

New gene therapy gives some kids born deaf the ability to hear - Modern Ghana

Tue, 20 Oct 2026 19:05:34 GMT

New gene therapy gives some kids born deaf the ability to hear Modern Ghana

Second gene-editing death in China disclosed after long delay - Science | AAAS

Thu, 06 Aug 2026 21:32:14 GMT

Second gene-editing death in China disclosed after long delay Science | AAAS

The hidden coordination burden behind cell and gene therapy access | HSES 2026 - Managed Healthcare Executive

Fri, 16 Oct 2026 17:59:49 GMT

The hidden coordination burden behind cell and gene therapy access | HSES 2026 Managed Healthcare Executive

AAV3B antibodies affect eligibility for liver-targeted gene therapy - Bioengineer.org

Tue, 20 Oct 2026 13:23:07 GMT

AAV3B antibodies affect eligibility for liver-targeted gene therapy Bioengineer.org

Cleveland Clinic First-In-Human Trial of CRISPR Gene-Editing Therapy Shown to Safely and Continuously Lower Cholesterol and Triglycerides After One Year - Cleveland Clinic

Fri, 28 Aug 2026 07:00:00 GMT

Cleveland Clinic First-In-Human Trial of CRISPR Gene-Editing Therapy Shown to Safely and Continuously Lower Cholesterol and Triglycerides After One Year Cleveland Clinic

Exclusive: Death of girl in Chinese gene-editing trial was never made public - Science | AAAS

Fri, 24 Jul 2026 07:00:00 GMT

Exclusive: Death of girl in Chinese gene-editing trial was never made public Science | AAAS

Doctoral student uses machine learning to transform gene therapy - The University of North Carolina at Chapel Hill

Sun, 11 Oct 2026 07:00:00 GMT

Doctoral student uses machine learning to transform gene therapy The University of North Carolina at Chapel Hill

Cell and Gene Therapy Market Size, Share | Growth Report 2034 - Fortune Business Insights

Mon, 05 Oct 2026 07:00:00 GMT

Cell and Gene Therapy Market Size, Share | Growth Report 2034 Fortune Business Insights

Gene therapy can partly restore sight in blind people, researchers reveal - The Guardian

Wed, 07 Oct 2026 07:00:00 GMT

Gene therapy can partly restore sight in blind people, researchers reveal The Guardian

Ocugen Signs Gene Therapy Deal Worth Up to $259 Million - MyChesCo

Tue, 20 Oct 2026 12:02:02 GMT

Ocugen Signs Gene Therapy Deal Worth Up to $259 Million MyChesCo

OTP Town Hall: Gene Therapy Manufacturing CMC and Facility Readiness for BLAs and Post-licensure Changes - U.S. Food and Drug Administration (.gov)

Tue, 15 Sep 2026 07:00:00 GMT

OTP Town Hall: Gene Therapy Manufacturing CMC and Facility Readiness for BLAs and Post-licensure Changes U.S. Food and Drug Administration (.gov)

CRISPR roadblocks: Scientists identify genes blocking gene therapy success - Phys.org

Thu, 13 Aug 2026 07:00:00 GMT

CRISPR roadblocks: Scientists identify genes blocking gene therapy success Phys.org

Boy’s brain tumor tied to gene therapy - Science | AAAS

Wed, 13 May 2026 07:00:00 GMT

Boy’s brain tumor tied to gene therapy Science | AAAS

One-Year Anniversary of World’s First Personalized CRISPR Gene Therapy for Child with Rare Genetic Disease - Children's Hospital of Philadelphia

Tue, 24 Feb 2026 08:00:00 GMT

One-Year Anniversary of World’s First Personalized CRISPR Gene Therapy for Child with Rare Genetic Disease Children's Hospital of Philadelphia

Gene therapy for hearing loss: Tag-teaming from the lab to the clinic - Boston Children's Answers

Thu, 23 Apr 2026 07:00:00 GMT

Gene therapy for hearing loss: Tag-teaming from the lab to the clinic Boston Children's Answers

Gene therapy in Abu Dhabi frees patient from transfusions — The National - UA.NEWS

Tue, 20 Oct 2026 17:38:02 GMT

Gene therapy in Abu Dhabi frees patient from transfusions — The National UA.NEWS

UMass Chan researchers receive FDA authorization to advance gene therapy clinical trial for Tay-Sachs and Sandhoff diseases - UMass Chan Medical School

Tue, 21 Jul 2026 07:00:00 GMT

UMass Chan researchers receive FDA authorization to advance gene therapy clinical trial for Tay-Sachs and Sandhoff diseases UMass Chan Medical School

Chinese university launches probe into girl’s gene-editing death - Science | AAAS

Thu, 30 Jul 2026 07:00:00 GMT

Chinese university launches probe into girl’s gene-editing death Science | AAAS

Advancing Gene Therapy for Sickle Cell and Thalassemia in the Middle East - Cleveland Clinic

Wed, 07 Oct 2026 07:00:00 GMT

Advancing Gene Therapy for Sickle Cell and Thalassemia in the Middle East Cleveland Clinic

Next-Generation CRISPR tools transform gene editing and gene therapy - News-Medical

Tue, 09 Jun 2026 13:35:48 GMT

Next-Generation CRISPR tools transform gene editing and gene therapy News-Medical

Investigational CRISPR therapy succeeds in patient with drug-resistant E. Coli - Clinical Trials Arena

Wed, 12 Aug 2026 07:00:00 GMT

Investigational CRISPR therapy succeeds in patient with drug-resistant E. Coli Clinical Trials Arena

Penn, CHOP team awarded Breakthrough Prize for blindness gene therapy - Penn Medicine

Sat, 18 Apr 2026 07:00:00 GMT

Penn, CHOP team awarded Breakthrough Prize for blindness gene therapy Penn Medicine

Epicrispr wins $8M CIRM grant for FSHD gene therapy trial - Dealroom

Mon, 19 Oct 2026 13:30:54 GMT

Epicrispr wins $8M CIRM grant for FSHD gene therapy trial Dealroom

Clinical response to systemic AAV gene therapy in a large animal model of late-stage lysosomal storage disease - Nature

Mon, 04 May 2026 07:00:00 GMT

Clinical response to systemic AAV gene therapy in a large animal model of late-stage lysosomal storage disease Nature

How a CRISPR Gene Therapy Could Change Life for Young Children With Sickle Cell Disease - Discover Magazine

Wed, 08 Jul 2026 07:00:00 GMT

How a CRISPR Gene Therapy Could Change Life for Young Children With Sickle Cell Disease Discover Magazine

Top 10 Best-Selling Gene Therapies - Genetic Engineering and Biotechnology News

Fri, 01 May 2026 07:00:00 GMT

Top 10 Best-Selling Gene Therapies Genetic Engineering and Biotechnology News

Historic FDA approval brings first gene therapy for genetic deafness - Nature

Tue, 28 Apr 2026 07:00:00 GMT

Historic FDA approval brings first gene therapy for genetic deafness Nature

Strong foundations to deliver cell and gene therapy - Nature

Mon, 31 Aug 2026 11:52:22 GMT

Strong foundations to deliver cell and gene therapy Nature

Dual-target gene therapy in Parkinson’s disease: a multicenter phase 1 trial - Nature

Wed, 10 Jun 2026 07:00:00 GMT

Dual-target gene therapy in Parkinson’s disease: a multicenter phase 1 trial Nature

Focused Ultrasound Panel Featured at Breakthroughs in Cell + Gene Therapy Meeting - Focused Ultrasound Foundation

Tue, 06 Oct 2026 07:00:00 GMT

Focused Ultrasound Panel Featured at Breakthroughs in Cell + Gene Therapy Meeting Focused Ultrasound Foundation

Gene Therapy Shows Promise for Life-Threatening Inherited Heart Disease - WCM Newsroom

Wed, 17 Jun 2026 07:00:00 GMT

Gene Therapy Shows Promise for Life-Threatening Inherited Heart Disease WCM Newsroom

FDA Clears IND for Gene Therapy Candidate to Treat Rare Metabolic Disorder - National Center for Advancing Translational Sciences (.gov)

Thu, 21 May 2026 07:00:00 GMT

FDA Clears IND for Gene Therapy Candidate to Treat Rare Metabolic Disorder National Center for Advancing Translational Sciences (.gov)

UAE gene therapy treats 370 global patients with rare condition over 6 years: Data - Khaleej Times

Tue, 20 Oct 2026 14:16:40 GMT

UAE gene therapy treats 370 global patients with rare condition over 6 years: Data Khaleej Times

Novel gene therapy platform restores muscle function in models of Duchenne muscular dystrophy - UT MD Anderson

Thu, 11 Jun 2026 07:00:00 GMT

Novel gene therapy platform restores muscle function in models of Duchenne muscular dystrophy UT MD Anderson

Did a boy’s life-saving gene therapy cause his brain tumour? - Nature

Thu, 21 May 2026 07:00:00 GMT

Did a boy’s life-saving gene therapy cause his brain tumour? Nature

New gene therapy approach shows promise for treating rare muscular dystrophy - Children's National

Thu, 08 Oct 2026 17:59:44 GMT

New gene therapy approach shows promise for treating rare muscular dystrophy Children's National

Donald Kohn’s Gene Therapy Breakthroughs Earn Medical Academy Honor - Bioengineer.org

Mon, 19 Oct 2026 22:01:21 GMT

Donald Kohn’s Gene Therapy Breakthroughs Earn Medical Academy Honor Bioengineer.org

World First: First Phase 3 Trial of In Vivo CRISPR Therapy Successfully Completed - CRISPR treatment comes one step closer to reality - www.amsterdamumc.org

Sat, 13 Jun 2026 07:00:00 GMT

World First: First Phase 3 Trial of In Vivo CRISPR Therapy Successfully Completed - CRISPR treatment comes one step closer to reality www.amsterdamumc.org

Citrandarin buffer sharply reduces transgene flow in sweet orange trials - Bioengineer.org

Tue, 20 Oct 2026 09:03:07 GMT

Citrandarin buffer sharply reduces transgene flow in sweet orange trials Bioengineer.org

CRISPR Trials Global Overview Q2 2026 from CMN Intelligence: 300+ Gene Editing Trials in the World's Only Manually Curated Global Database - CRISPR Medicine News

Sat, 12 Sep 2026 00:57:29 GMT

CRISPR Trials Global Overview Q2 2026 from CMN Intelligence: 300+ Gene Editing Trials in the World's Only Manually Curated Global Database CRISPR Medicine News

5-Year Data Support Safety of MRI-Guided AAV2-GDNF Gene Therapy in Advanced Parkinson Disease - NeurologyLive

Fri, 28 Aug 2026 07:00:00 GMT

5-Year Data Support Safety of MRI-Guided AAV2-GDNF Gene Therapy in Advanced Parkinson Disease NeurologyLive

FDA sees early promise in new gene therapy for BAG3-associated DCM - Cardiovascular Business

Wed, 22 Jul 2026 07:00:00 GMT

FDA sees early promise in new gene therapy for BAG3-associated DCM Cardiovascular Business

Sensorion Appoints Peter Weber Chief Medical Officer to Advance Hearing Loss Gene Therapy - citybiz

Mon, 19 Oct 2026 12:45:32 GMT

Sensorion Appoints Peter Weber Chief Medical Officer to Advance Hearing Loss Gene Therapy citybiz

Inside UCB’s new gene therapy facility - UCB

Fri, 24 Apr 2026 13:38:03 GMT

Inside UCB’s new gene therapy facility UCB

UAB doses world’s first patient in investigational gene therapy trial for BAG3-associated dilated cardiomyopathy - The University of Alabama at Birmingham

Thu, 24 Sep 2026 07:00:00 GMT

UAB doses world’s first patient in investigational gene therapy trial for BAG3-associated dilated cardiomyopathy The University of Alabama at Birmingham

CRISPR gene editing of angiopoietin-like 3: toward one-time precision therapy for dyslipidaemia - Nature

Wed, 04 Mar 2026 08:00:00 GMT

CRISPR gene editing of angiopoietin-like 3: toward one-time precision therapy for dyslipidaemia Nature

UMass Chan licenses gene therapy for maple syrup urine disease to Plowshare Therapies - UMass Chan Medical School

Mon, 27 Apr 2026 07:00:00 GMT

UMass Chan licenses gene therapy for maple syrup urine disease to Plowshare Therapies UMass Chan Medical School

Researchers Behind World’s First Patient-Tailored CRISPR Gene Editing Therapy Named to TIME100 List of the 100 Most Influential People in the World - Children's Hospital of Philadelphia

Wed, 15 Apr 2026 07:00:00 GMT

Researchers Behind World’s First Patient-Tailored CRISPR Gene Editing Therapy Named to TIME100 List of the 100 Most Influential People in the World Children's Hospital of Philadelphia

‘I’m allowed to live without pain’: UMMC gene therapy transforms life of sickle cell patient - WBAL-TV

Fri, 24 Jul 2026 07:00:00 GMT

‘I’m allowed to live without pain’: UMMC gene therapy transforms life of sickle cell patient WBAL-TV

An Experimental One-Time Treatment for High Cholesterol Shows Promising Results - Time Magazine

Fri, 28 Aug 2026 07:00:00 GMT

An Experimental One-Time Treatment for High Cholesterol Shows Promising Results Time Magazine

Ultragenyx makes swift $413M return on FDA review vouchers after summer of gene therapy nods - Fierce Pharma

Wed, 14 Oct 2026 20:19:49 GMT

Ultragenyx makes swift $413M return on FDA review vouchers after summer of gene therapy nods Fierce Pharma

HOPE-B Gene Therapy Holds Near-Normal Factor IX at Five Years - DocWireNews

Sat, 11 Jul 2026 07:00:00 GMT

HOPE-B Gene Therapy Holds Near-Normal Factor IX at Five Years DocWireNews

New Discovery Uses Salt to Overcome Major Obstacle in Gene Therapy - University of Houston

Tue, 16 Jun 2026 07:00:00 GMT

New Discovery Uses Salt to Overcome Major Obstacle in Gene Therapy University of Houston

Activity-dependent antioxidant gene therapy protects against epileptogenesis - Nature

Fri, 19 Jun 2026 07:00:00 GMT

Activity-dependent antioxidant gene therapy protects against epileptogenesis Nature

Gene Therapy Approach Reduces Muscle Spasticity After Chronic Spinal Cord Injury - UC San Diego Today

Tue, 09 Jun 2026 07:00:00 GMT

Gene Therapy Approach Reduces Muscle Spasticity After Chronic Spinal Cord Injury UC San Diego Today

National Sickle Cell Awareness Month shines a light on promising gene-editing therapy - Case Western Reserve University

Thu, 24 Sep 2026 07:00:00 GMT

National Sickle Cell Awareness Month shines a light on promising gene-editing therapy Case Western Reserve University

Teenager Becomes First Patient To Receive CRISPR Therapy for Beta-Thalassemia in Germany - Technology Networks

Fri, 02 Oct 2026 07:00:00 GMT

Teenager Becomes First Patient To Receive CRISPR Therapy for Beta-Thalassemia in Germany Technology Networks

In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia - NEJM

Tue, 26 May 2026 10:45:36 GMT

In Vivo Base Editing of PCSK9 with VERVE-102 for Hypercholesterolemia NEJM

Shooting for the stars: UVA lab’s new gene therapy may end pediatric seizures - UVA Today

Wed, 24 Jun 2026 07:00:00 GMT

Shooting for the stars: UVA lab’s new gene therapy may end pediatric seizures UVA Today

Fractyl Health’s GLP-1 gene therapy trial given green light - Clinical Trials Arena

Mon, 11 May 2026 07:00:00 GMT

Fractyl Health’s GLP-1 gene therapy trial given green light Clinical Trials Arena

Gene editing advance shows promise as long-lasting treatment for rare liver disease - Medical Xpress

Fri, 11 Sep 2026 07:00:00 GMT

Gene editing advance shows promise as long-lasting treatment for rare liver disease Medical Xpress

Chronic Lung Infection Surprisingly Sustains Gene Therapy Expression in Cystic Fibrosis Rat Lungs - Bioengineer.org

Mon, 19 Oct 2026 17:06:21 GMT

Chronic Lung Infection Surprisingly Sustains Gene Therapy Expression in Cystic Fibrosis Rat Lungs Bioengineer.org

Cystic fibrosis: new study identifies gene therapy - healthcare-in-europe.com

Thu, 30 Apr 2026 07:00:00 GMT

Cystic fibrosis: new study identifies gene therapy healthcare-in-europe.com

FDA approves Ultragenyx’s gene therapy Fayuvi for rare neurodegenerative disorder - Fierce Pharma

Thu, 17 Sep 2026 07:00:00 GMT

FDA approves Ultragenyx’s gene therapy Fayuvi for rare neurodegenerative disorder Fierce Pharma

BDMS invests in gene therapy - Bangkok Post

Tue, 20 Oct 2026 00:07:00 GMT

BDMS invests in gene therapy Bangkok Post

Subretinal Gene Therapy for X-Linked Retinoschisis - NEJM

Wed, 10 Jun 2026 21:39:53 GMT

Subretinal Gene Therapy for X-Linked Retinoschisis NEJM

What CRISPR Gene Therapy Means for HAE: FAQ on This 1-Time Treatment - HCPLive

Fri, 18 Sep 2026 12:08:37 GMT

What CRISPR Gene Therapy Means for HAE: FAQ on This 1-Time Treatment HCPLive

Hematopoietic Stem-Cell Gene Therapy for Cystinosis - NEJM

Wed, 18 Feb 2026 08:00:00 GMT

Hematopoietic Stem-Cell Gene Therapy for Cystinosis NEJM

First-in-the-world gene therapy delivers missing gene directly to infant's brain, marking historic milestone in precision medicine - EurekAlert!

Mon, 08 Jun 2026 07:00:00 GMT

First-in-the-world gene therapy delivers missing gene directly to infant's brain, marking historic milestone in precision medicine EurekAlert!

Regulation of sodium/calcium homeostasis by BacNav gene therapy rescues cardiac dysfunction in chronic heart failure - Science | AAAS

Fri, 20 Mar 2026 07:00:00 GMT

Regulation of sodium/calcium homeostasis by BacNav gene therapy rescues cardiac dysfunction in chronic heart failure Science | AAAS

Exploring gene editing as a potential therapeutic strategy for hemophilia - Frontiers

Thu, 15 Jan 2026 22:11:20 GMT

Exploring gene editing as a potential therapeutic strategy for hemophilia Frontiers

Virginia budget expands Medicaid access to sickle cell gene therapy - Virginia Mercury

Thu, 09 Jul 2026 07:00:00 GMT

Virginia budget expands Medicaid access to sickle cell gene therapy Virginia Mercury

Gene therapy reverses Fragile X deficits in mice - University of California, Riverside

Thu, 18 Jun 2026 07:00:00 GMT

Gene therapy reverses Fragile X deficits in mice University of California, Riverside

Gene therapy ‘switch’ may offer non-addictive pain relief - Penn Medicine

Wed, 07 Jan 2026 08:00:00 GMT

Gene therapy ‘switch’ may offer non-addictive pain relief Penn Medicine

Gene Therapy to Treat Profound Hearing Loss - NEJM

Wed, 11 Mar 2026 07:00:00 GMT

Gene Therapy to Treat Profound Hearing Loss NEJM

Ultragenyx, after FDA speed bump, lands 1st gene therapy nod with green light in glycogen storage disease - Fierce Pharma

Wed, 19 Aug 2026 07:00:00 GMT

Ultragenyx, after FDA speed bump, lands 1st gene therapy nod with green light in glycogen storage disease Fierce Pharma

September: Bristol researchers take major step towards Alzheimer’s gene therapy | News and features - University of Bristol

Mon, 28 Sep 2026 07:00:00 GMT

September: Bristol researchers take major step towards Alzheimer’s gene therapy | News and features University of Bristol

J&J axes eye disease gene therapy after key phase 2b readout - Fierce Biotech

Wed, 15 Jul 2026 07:00:00 GMT

J&J axes eye disease gene therapy after key phase 2b readout Fierce Biotech

Cell & gene therapy: Preparing for the next phase of employer health plan risk - Lockton

Mon, 29 Jun 2026 07:00:00 GMT

Cell & gene therapy: Preparing for the next phase of employer health plan risk Lockton

Engineered red blood cell vesicles improve gene therapy delivery - News-Medical

Mon, 17 Aug 2026 07:00:00 GMT

Engineered red blood cell vesicles improve gene therapy delivery News-Medical

Gene therapy shows promise for treating Dravet syndrome - Mayo Clinic

Tue, 24 Mar 2026 07:00:00 GMT

Gene therapy shows promise for treating Dravet syndrome Mayo Clinic

A new hope for dogs with dementia: USF Health researchers explore gene therapy - the University of South Florida

Wed, 07 Oct 2026 14:38:42 GMT

A new hope for dogs with dementia: USF Health researchers explore gene therapy the University of South Florida

Study Uncovers Potential Gene Therapy for Late-Stage Parkinson’s Side-Effects - Northwestern University

Wed, 03 Jun 2026 07:00:00 GMT

Study Uncovers Potential Gene Therapy for Late-Stage Parkinson’s Side-Effects Northwestern University

Regenxbio gene therapy refiling plan derailed by FDA hold over spine MRI findings - Fierce Biotech

Mon, 24 Aug 2026 07:00:00 GMT

Regenxbio gene therapy refiling plan derailed by FDA hold over spine MRI findings Fierce Biotech

FDA Grants RMAT Designation to Inhalable Gene Therapy for Advanced Lung Tumors - Targeted Oncology

Wed, 11 Feb 2026 08:00:00 GMT

FDA Grants RMAT Designation to Inhalable Gene Therapy for Advanced Lung Tumors Targeted Oncology

Gene Therapy for Severe Mucopolysaccharidosis Type I (MPS I): Adam’s Story - Children's Hospital of Philadelphia

Tue, 02 Jun 2026 20:31:08 GMT

Gene Therapy for Severe Mucopolysaccharidosis Type I (MPS I): Adam’s Story Children's Hospital of Philadelphia