UMass Chan Medical School and pharmaceutical company Astellas Pharma Inc. have entered into a sponsored research agreement to proceed with research for an adeno-associated virus vector mediated gene therapy for the treatment of Alexander disease, a fatal, ultra-rare disease.

The cover story for the winter 2023-24 issue of @umasschan magazine highlights how the families of children with genetic disorders inspire gene therapy research and how UMass Chan is streamlining the pathway from bench research to clinical trials for new treatments.

Top story: Boston toddler Noa Greenwood, who was one of the first to receive an investigational gene therapy for Canavan disease developed at UMass Chan Medical School, delivered a very special “thank you” to the research team in a visit with her parents earlier this year.

Boston toddler Noa Greenwood, who was one of the first to receive an investigational gene therapy for Canavan disease developed at UMass Chan Medical School, delivered a very special “thank you” to the research team in a visit with her parents earlier this year.

PhD candidate Suk Namkung has lived on three continents. He aims to investigate and advance a new generation of gene therapy viral vectors.

Jun Xie, PhD, and Guangping Gao, PhD, received funding from End AxD, a nonprofit organization dedicated to the research and treatment for Alexander disease, to investigate an RNA silencing gene therapy for the rare genetic central nervous system disorder.

UMass Chan Medical School has signed a three-year sponsored research agreement with NeuShen Therapeutics Inc. to investigate a gene therapy treatment for amyotrophic lateral sclerosis. Jun Xie, PhD, and Guangping Gao, PhD, are leading the project for UMass Chan.

UMass Chan Medical School has licensed the rights to develop and commercialize a novel second-generation gene therapy to treat spinal muscular atrophy developed by Jun Xie, PhD, Guangping Gao, PhD, and colleagues to global biopharmaceutical company CANbridge Pharmaceuticals Inc.

Guangping Gao, PhD, will be honored by the National Tay-Sachs & Allied Diseases Association for his work in identifying the Canavan gene, revitalizing gene therapy, and his many accomplishments leading to potential treatments for Canavan disease.

Progress in UMass Chan Medical School research in developing a gene therapy for a rare bone disease characterized by abnormal bone formation in the skeletal muscle and in connective tissues has reached an important milestone.

A paper from the Horae Gene Therapy Center at UMass Chan Medical School may settle a longstanding debate about how to best manufacture adeno-associated virus vectors for gene therapy.

Guangping Gao, PhD, and Dan Wang, PhD, show the first evidence that a suppressor transfer RNA therapy, delivered by a recombinant adeno-associated virus, can restore protein production up to six months after treatment in a mouse model of the rare genetic disease mucopolysaccharidosis type I.

Researchers from UMass Chan Medical School have received a five-year $13.6 million program project grant from the National Heart, Lung and Blood Institute to develop new gene therapy models for alpha-1 antitrypsin deficiency, a chronic, debilitating genetic lung disease that shortens the lifespan.

Jae-Hyuck Shim, PhD, is inspired to develop a gene therapy for fibrodysplasia ossificans progressiva (FOP) after seeing the strength of families who have a child with the rare, crippling disease.

UMass Medical School researchers Guangping Gao, PhD, and Dan Wang, PhD, are working with ASC Therapeutics to bring a gene therapy for maple syrup urine disease (MSUD) to the clinic.

UMass Medical School has entered into a three-year, collaborative research agreement with Pfizer to evaluate determinants that influence the manufacturing quality and yield of viral vectors used in gene therapy. The research at UMMS is being performed under the direction of Guangping Gao, PhD, and Dan Wang, PhD.

Six UMass Medical School scientists are among the members of the National Institutes of Health’s Somatic Cell Genome Editing Consortium to publish a paper in Nature outlining the program’s goals.

An influential scientific journal’s list of top translational biotech researchers for 2019 includes Guangping Gao, PhD. Rankings are based on total patents for the year, among other factors.

Guangping Gao, PhD, has been elected to the American Academy of Microbiology, the honorific leadership group of the American Society of Microbiology.

Inspired by patients and their families, 40 scientists at the The Li Weibo (李伟波) Institute for Rare Diseases Research are working together to discover cures for dozens of rare disorders, according to a Facebook Live interview with co-directors Michael Green, MD, PhD, and Guangping Gao, PhD.