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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.



Disease focus area*




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

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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


(856) 566-6334
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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

(832) 824-4670
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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

(212) 639-6394
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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

(212) 659-8542
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Department of Microbiology & Molecular Genetics

  • Cystic fibrosis
  • Cardiovascular disease


University of Pittsburgh
523 Bridgeside Point II
450 Technology Drive
Pittsburgh, PA 15219


(412) 648-9570
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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

(215) 898-1281
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Grousbeck Gene Therapy Center

  • Blindness
  • Deafness
  • Neurosensory disorders
  • Other
  • Gene Delivery Technology Development

20 Stanford Street
Boston, MA 02114


Email direct
Email admin
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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

(508) 856-3394
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IU School of Medicine Brown Center for Immunotherapy

  • Alzheimer’s disease

Walther Hall
980 W. Walnut Street
Indianapolis, IN 46202

(317) 278-6969
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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


(312) 926-2210
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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

(614) 722-5615
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Powell Gene Therapy Center


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
(352) 294-5171

Center for Rare Disease Research

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


(503) 418-2700
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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


(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

(919) 962-3285
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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

(319) 335-7807
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


(612) 624-3113


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

Learn more

*This list excludes oncology as a disease focus area.


Last Updated: 06/27/2022

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