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Gene Therapy Centers

This list of gene therapy centers in the U.S. is intended for use by patients, advocates, and caregivers who are seeking gene therapy research for a rare disease. This list excludes oncology (cancer) due to the wide scope of cancers and number of centers working on them. The Blood & Marrow Transplant Information Network has a list of medical centers offering CAR T-cell therapy. If you are unsure whether a clinical trial is open for your disease, search by diagnosis using the ASGCT Clinical Trials Finder. To suggest a center or make an update to a center listed please contact us.

Center  

 

Disease focus area* 

 

Location 

Contact 

Boston Children’s Hospital Gene Therapy Program 

adrenoleukodystrophy (ALD) 

beta thalassemia 

hemophilia A and B 

inherited retinal disorders 

ornithine transcarbamylase (OTC) deficiency 

sickle cell disease 

spinal muscular atrophy (SMA) 

Wiskott-Aldrich syndrome 

X-linked severe combined immunodeficiency (SCID-X1) 

X-linked chronic granulomatous disease (X-CGD) 

relapsed or treatment-resistant B-cell acute lymphoblastic leukemia (ALL)  

relapsed or refractory large B-cell lymphoma 

Boston Children’s Hospital  

300 Longwood Avenue, Boston MA 02115 

617-919-1992 

https://www.childrenshospital.org/programs/gene-therapy-program 

Cell and Gene Therapy Collaborative at CHOP 

Beta thalassemia 

Choroideremia 

Hemophilia 

Leber congenital amaurosis (LCA) 

Methylmalonic acidemia  

Mucopolysaccharidosis (MPS I, MPS II)  

Phenylketonurias (PKU) 

Sickle cell disease 

Spinal muscular atrophy (SMA) 

 

The Children’s Hospital of Philadelphia 

3501 Civic Center Blvd 

Philadelphia, PA 19104 

Contact  

Learn more 

Cell and Gene Therapy Center at RowanSOM / Cooper Hospital 

Canavan disease 

Neurological disorders 

 

Rowan University, School of Osteopathic Medicine 

2 Medical Center Drive, Science Center #253 

Stratford, NJ 08084 

 

P: (856) 566-6334 

Email 

Learn more 

 

 

 

Center for Cell and Gene Therapy at Baylor College of Medicine 

AIDS 

Cardiovascular disease 

 

Baylor College of Medicine 
One Baylor Plaza, Rm N1002 
Houston, TX 77030 
 

P: (832) 824-4670 

Email 

Learn more 

 

 

 

Center for Cell Engineering at Memorial Sloan Kettering Cancer Center 

Blood disorders 

Neurologic diseases 

Center for Cell Engineering 
Memorial Sloan Kettering Cancer Center, Box 182 
1275 York Avenue 
New York, NY 10065 

 

P: (212) 639-6394 

Email: becharde@mskcc.org 

Department of Genetics and Genomic Sciences at Mount Sinai 

Alzheimer's disease 

Cardiovascular illness 

Crohn's disease 

Diabetes 

Infectious diseases 

Lysosomal storage disorders 

Psychiatric illnesses 

Ulcerative colitis 

 

One Gustave L. Levy Place 
Box 1498 
New York, NY 10029 

P: (212) 659-8542 

Email 

Learn more 

Department of Microbiology & Molecular Genetics 

Cystic fibrosis 

Cardiovascular disease 

University of Pittsburgh 

523 Bridgeside Point II 

450 Technology Drive 

Pittsburgh, PA 15219 

 

P: (412) 648-9570 

Email 

Learn more 

Gene Therapy Program at UPenn 

Blood diseases  

Eye disorders 

Infectious diseases 

Lung diseases 

Metabolic diseases 

Neurologic diseases 

Neuromuscular disorders 

Renal/liver diseases 

 

Suite 1200 Translational Research Labs (TRL) 

125 S. 31st Street 

Philadelphia, PA 19104 

P: (215) 898-1281 

Email 

Learn more 

 

Grousbeck Gene Therapy Center 

Blindness 

Deafness 

Neurosensory disorders 

Other 

Gene Delivery Technology Development 

 

MEE/SERI – 2W 

20 Stanford Street 

Boston, MA 02114 

 

P: (617)-573-6307 

Email direct 

Email admin  

Learn more 

 

Horae Gene Therapy Center 

Alpha-1 Antitrypsin Deficiency 
Canavan disease 
Huntington's disease 
Inherited and acquired cardiac arrhythmias 
Leber congenital amaurosis (LCA) 
Lou Gehrig's disease (ALS) 
Neurofibromatosis Type 2 
Retinitis pigmentosa 
Stargardt disease 
Tay-Sachs and Sandhoff disease 

VLCAD Deficiency  

 

UMass Medical School  

368 Plantation Street Worcester, MA 01605 

P: (508) 856-3394 

Email 

Learn more 

 

IU School of Medicine Brown Center for Immunotherapy  

Alzheimer’s disease 

 

Walther Hall 
980 W. Walnut Street 
Indianapolis, IN 46202 
 

P: (317) 278-6969 

Learn more 

 

Mathews Center for Cellular Therapy  

Adult stem cell transplant  

Allergy and immunology 

Solid organ transplant  

 

Northwestern Memorial Hospital  

710 N. Fairbanks Court  

Rm 7-613 

Chicago, IL 60611 

 

P: (312) 926-2210 

Email 

Learn more 

Nationwide Children’s Center for Gene Therapy   

Infectious Diseases 

Ischemia 

Lysosomal storage disorders 

Neuromuscular disorders 

Neurodegenerative diseases 

Neonatal hypertension 

Reperfusion injury 

 

 

The Abigail Wexner Research Institute at Nationwide Children's Hospital 

Research Bldg. II 

Columbus, OH 43205 

P: (614) 722-5615 

Email 

Learn more 

Nemours Children’s Hospital - Central Florida 

  • Duchenne Muscular Dystrophy
  • Spinal muscular atrophy (SMA)

Nemours Children’s Hospital

6535 Nemours Parkway, Orlando Florida 32827

(407) 650-7715

https://www.nemours.org/services/neuromuscular-disorders.html

Powell Gene Therapy Center 

and 

Powell Center for Rare Disease Research  

 

Cardiovascular diseases 

Central nervous system disorders 

Endocrine disorders 

Eye diseases 

Inflammatory disorders 

Metabolic disorders 

Neuromuscular disorders 

Pulmonary diseases 

 

University of Florida 

Academic Research Building College of Medicine 

1200 Newell Drive 

Gainesville, FL 32610 

Gene Therapy Center 

P: (352) 294-5171 

Email 

 

Center for Rare Disease Research 

Email   

The Vaccine and Gene Therapy Institute at OHSU 

AIDS 

Chronic viral infection-associated diseases 

Infectious diseases 

Infectious diseases of the elderly 

Newly emerging viral diseases 

 

Oregon Health & Science University – West Campus 

505 N.W. 185th Avenue 

Beaverton, OR 97006 

 

P: (503) 418-2700  

Email 

Learn more 

Tulane Center for Stem Cell Research & Regenerative Medicine 

New therapies for common diseases including stroke, and multiple sclerosis 

Tulane University School of Medicine 

1430 Tulane Avenue 

New Orleans, LA 70112 

 

P: 504-988-5800 

Learn more 

UNC Gene Therapy Center 

 

Eye disorders 

Hemophilia 

Muscular dystrophies 

Neurological disorders (epilepsy) 

Neuropathic lysosomal storage diseases (MPS and Krabbe disease) 

 

University of North Carolina 

7119 Thurston Bowles Bldg. 

104 Manning Drive 

Campus Box 7352 

Chapel Hill, NC 27599 

P: 919-962-3285 

Email 

Learn more 

University of Iowa Gene Therapy Center  

Cystic fibrosis 

Central nervous system disorders 

Diseases affecting muscle, 

vessels, skin, the liver and the lungs 

 

Roy J. and Lucille A. Carver College of Medicine 

451 Newton Road 

200 Medicine Admin. Building 

Iowa City, IA 52242 

Learn more 

University of Minnesota Gene Therapy Center 

Mucopolysaccharidoses (MPS) and related diseases 

Medical School Department of Pediatrics 

2450 Riverside Avenue 

6th Floor, East Building 

Delivery Code 8952A 

Minneapolis, MN 55454 

 

P: (612) 624-3113 

Email 

 

UT Southwestern Medical Center  

Central nervous system disorders 

Neurodegenerative diseases 

Neuromuscular disorders 

Lysosomal storage disorders 

Metabolic disorders 

Genetic epilepsies 

Neurodevelopmental disorders 

 

UT Southwestern Medical Center 

5323 Harry Hines Blvd. 

Dallas, TX 75390 

Email  

Learn more 

Last Updated: 11/07/2024

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