The functions of the Gene Therapy Program will transition out of Penn on Oct 1, 2024. You can find us on LinkedIn at Gemma Biotherapeutics and Franklin Biolabs and on the web at gemmabiotx.com and franklinbio.com.
Gene Therapy Program | University of Pennsylvania
生物技术研究
Philadelphia，Pennsylvania 13,722 位关注者
Catalyzing the development of transformative genetic-based therapeutics from concept to clinic.
关于我们
Dedicated to discovery + translational research and led by Dr. James Wilson, the Gene Therapy Program (GTP) at the University of Pennsylvania focuses on the development of next-generation gene transfer vectors and their application in the treatment of a variety of acquired and inherited diseases. Dr. Wilson's commitment to patients with rare diseases stems from his days as a graduate student and medical student at the University of Michigan between 1977 and 1984. He was discouraged by the lack of available treatments for rare disease patients, but optimistic that science could yield solutions with his initial focus being gene therapy. He completed his training in Internal Medicine at the Massachusetts General Hospital followed by a postdoctoral fellowship at the Whitehead Institute where he began his work in gene therapy. After launching his faculty career in the Howard Hughes Medical Institute and the University of Michigan, he moved to the University of Pennsylvania in 1993. Jim’s laboratory has made seminal contributions to the technology of gene transfer and has paved the way for translation of these technologies into the clinic. He has published more than 550 papers and is named on more than 110 patents. Today, the goals of the Gene Therapy Program are to develop and commercialize transformative, genetic-based therapeutics. These goals are driven by the unmet needs of patients with inherited genetic diseases. The Gene Therapy Program currently employs more than 350+ full-time employees with an annual operating budget based on funding from a diverse group of public and private sponsors.
- 网站
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https://gtp.med.upenn.edu/
Gene Therapy Program | University of Pennsylvania的外部链接
- 所属行业
- 生物技术研究
- 规模
- 201-500 人
- 总部
- Philadelphia，Pennsylvania
- 类型
- 非营利机构
- 创立
- 1993
- 领域
- Higher Education、AAV、Translational Research、Genome Editing、Capsid Engineering、Discovery Research、Vector Design、Biomedical Research、Rare Diseases、Clinical Research、GLP、Good Manufacturing Practice/GMP、Orphan Disease、Patient Registry、Gene Therapy、Good Laboratory Practice/GLP、Lipid Nanoparticles、Adeno-Associated Viruses、Genetic Medicine 和Genetic Therapies
地点
Gene Therapy Program | University of Pennsylvania员工
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Malcolm Montgomery, Ph.D.
Gene Therapy Program at the University of Pennsylvania
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Casey Hare
Associate Director, Human Resources at Gene Therapy Program | University of Pennsylvania
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Jessica Chichester
Senior Director, Immunology Core at Gene Therapy Program
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Carla Bass
Director, Stability Programs at the University of Pennsylvania - Gene Therapy Program
动态
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Check out the recent news coverage by Human Gene Therapy on two recent publications by the Gene Therapy Program at the University of Pennsylvania! The item highlights a lipid nanoparticle-based treatment approach developed by Jenny Greig and her team to address all possible genetic mutations that can cause maple syrup urine disease (MSUD). The item also showcases the identification of a novel family of adeno-associated virus (AAV) variants with desirable biodistribution properties by Jacob A. Hoffman, PhD. Please see the posting from the Mary Ann Liebert, Inc. newsroom. https://lnkd.in/eVp7vUZP #GeneTherapy #LNP #AAV
Mary Ann Liebert, Inc.
home.liebertpub.com
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The Gene Therapy Program at the University of Pennsylvania will be transitioning effective October 1, 2024, into two new companies in order to continue our groundbreaking research.? ? GEMMA Biotherapeutics (GEMMABio) is the new therapeutics company, and it will comprise the research and innovation arm to speed the research of and global access to life-changing advanced therapies for those living with rare diseases (www.gemmabiotx.com).?Franklin Biolabs is the new genetic medicines contract research organization (CRO), and it will be the services and production arm, providing comprehensive solutions from discovery to development and distribution for the global genetic medicines industry (www.franklinbio.com).? ? For more information on the two new companies, please see this press release that was issued on July 31, 2024 (https://lnkd.in/e9A8Dz6S).?The news was covered by a variety of media outlets, including the Philadelphia Inquirer (https://lnkd.in/eGwtEaUc) and Biocentury (https://lnkd.in/eez-g964). ? For clients of the vector core and immunology core: we look forward to working with you these next several weeks as part of the University of Pennsylvania, and then going forward in our new capacity.?These services will continue this fall as part of Franklin Biolabs.?The transition has just started, and you will be receiving more information from us in the coming weeks. In the meantime, please continue to correspond with us and place orders according to the current processes.?Please feel free to reach out to your contacts at GTP with any immediate questions.
GEMMABio
gemmabiotx.com
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We are excited to share the recent publication of a GTP manuscript in Human Gene Therapy! ?? Read the article from Dr. Jake Hoffman and GTP colleagues titled “Modulation of AAV9 Galactose Binding Yields Novel Gene Therapy Vectors and Predicts Cross-Species Differences in Glycan Avidity.” https://lnkd.in/eiJqE2n5 #GeneTherapy #AAV
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We are pleased to share exciting news! GTP’s Dr. Jenny Greig and her team recently had a manuscript published in Human Gene Therapy. ?? Read the article, titled “Lipid nanoparticle mRNA therapy improves survival and reduces serum branched-chain amino acids in mouse models of maple syrup urine disease." #GeneTherapy #LNP #mRNA
Lipid nanoparticle mRNA therapy improves survival and reduces serum branched-chain amino acids in mouse models of maple syrup urine disease | Human Gene Therapy
liebertpub.com
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Check out this write up from the University of Pennsylvania that details a recent manuscript published in Nature Chemistry! ?? GTP’s Dr. Claude Warzecha and Dr. Jim Wilson worked alongside Penn Engineering researchers in the lab of Dr. Michael J Mitchell where this work originated. #LNP #mRNA #AID
Penn pioneers a ‘one-pot platform’ to promptly produce mRNA delivery particles | Penn Today
penntoday.upenn.edu
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We are pleased to share the recent publication of a manuscript, with GTP co-authors Dr. Claude Warzecha and Dr. Jim Wilson, in Nature Chemistry! GTP is proud to have collaborated with Penn Engineering researchers in the lab of Michael J Mitchell at the University of Pennsylvania where this work originated. Read the article here: #LNPs #mRNA #AID
Fast and facile synthesis of amidine-incorporated degradable lipids for versatile mRNA delivery in vivo - Nature Chemistry
nature.com
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Gene Therapy Program | University of Pennsylvania转发了
THANK YOU to our cyclists, walkers, spectators, and donors for a fantastic MDBR!!! Another successful year in the books!!! #PennMedMDBR2024 ??Jordan Bush Photography
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iECURE, inc., our partner and University of Pennsylvania spinout, was recently featured in the Philadelphia Business Journal as the first company to receive FDA approval to conduct clinical trials of a #GeneEditing therapy in newborns and infants. Check out the interview with iECURE's CEO, Joe Truitt!
University of Pennsylvania gene-editing spinout iEcure hits the FDA trifecta - Philadelphia Business Journal
bizjournals.com
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Registration for this year's Million Dollar Bike Ride is open! ?? Register to ride by June 2nd, and learn about the annual event, organized by the Orphan Disease Center | University of Pennsylvania: https://bit.ly/2024MDBR?#MDBR
??♂? Register for the 11th annual Million Dollar Bike Ride! Register online by June 2nd for the best price. Choose from our 10-mile, 30-mile, or 70-mile routes, or join our mile walk for those who are unable to ride. Don't wait—lock in your spot now and let's hit the road together! bit.ly/2024MDBR ?? #pennmedmdbr2024 #MillionDollarBikeRide #RegisterNow #MDBR #cycling #bikeride #upenn #philadelphia #philly
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