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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

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
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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
Email
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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
Email
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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
Email
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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

MEE/SERI – 2W
20 Stanford Street
Boston, MA 02114

 

(617)-573-6307
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
Email
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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
Email
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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
(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

 

(503) 418-2700
Email
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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
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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
Email
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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
Email

 

UT Southwestern Medical Center

  • Aspartylglucosaminuria (AGU)
  • Batten Diseases
  • Charcot-Marie Tooth
  • Giant Axonal Neuropathy (GAN)
  • Krabbe disease
  • Leigh Syndrome
  • Multiple Sulfatase Deficiency
  • Rett Syndrome
  • Sandhoff disease
  • SLC6A1
  • Tay-Sachs disease

UT Southwestern Medical Center
5323 Harry Hines Blvd.
NA2.508
Dallas, TX 75390

(214) 648-0671
Email
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*This list excludes oncology as a disease focus area.

 

Last Updated: 03/30/2021