News

Five new endowed chairs have been established at UMass Chan Medical School, thanks to the generosity of Donna and Robert Manning, proud alumni and preeminent pillars of the University of Massachusetts.

ALS research at UMass Chan Medical School is the big beneficiary as select Boston Marathon runners raise tens of thousands of dollars each year. This year, five competitors in the April 18 event are running as part of the UMass Chan ALS Cellucci Fund Boston Marathon team and two sons of the late Jake Kennedy will run in support of the Jake Kennedy ALS Fund.

Charles P Emerson Jr., PhD, and colleagues at UMass Chan Medical School have developed a technology to isolate human skeletal muscle stem cells, or progenitor cells, from induced pluripotent stem cells.

An international team of investigators has discovered that an inorganic polyphosphate released by nerve cells known as astrocytes in people with ALS and frontotemporal dementia contributes to the motor neuron death that is the signature of these diseases.

Hosted by the Departments of Neurobiology and Psychiatry and the NeuroNexus Institute at UMass Chan Medical School on Saturday, Feb. 5, the annual Brain Bee competition was created to encourage high school students to learn more about the neurosciences.

Robert H. Brown Jr., DPhil, MD, Jonathan Watts, PhD, and colleagues showed the ability to suppress mutant forms of the ALS gene known as C9ORF72 in a single-patient pilot study. The paper reporting the results is part of the 2022 STAT News STAT Madness competition.

In a piece written for The Conversation, Miguel Sena-Esteves, PhD, talks about research leading to the first ever gene therapy for Tay-Sachs disease and the launch of the Translational Institute for Molecular Therapeutics at UMass Chan.

An Oregon couple whose baby was diagnosed with an ultra-rare genetic disorder has launched a foundation to raise money to accelerate development of therapies for the condition and recently signed a gift agreement with UMass Chan Medical School to jump start the research.

The Translational Institute for Molecular Therapeutics leverages UMass Chan Medical School’s extensive experience in researching and developing gene therapies for early-stage clinical trials. Miguel Sena-Esteves, PhD, is director of the new institute.

Robert H. Brown Jr., DPhil, MD, Jonathan Watts, PhD, and colleagues have shown the ability to suppress mutant forms of an ALS gene in a single-patient pilot study.

Sio Gene Therapies, a clinical-stage company focused on developing gene therapies for neurodegenerative diseases, presented positive interim data from its Phase I/II study of AXO-AAV-GM1, an adeno-associated viral vector-based gene therapy candidate for GM1 gangliosidosis that was licensed from UMass Chan.

Riaan Research Initiative, a non-profit organization, and UMass Chan Medical School are entering into an agreement to fund, research and develop a gene replacement therapy to combat Cockayne syndrome, a fatal autosomal recessive disorder.

Two studies from the lab of Daryl Bosco, PhD, offer new insights into the biochemical and molecular mechanisms that cause amyotrophic lateral sclerosis.

Five runners are participating in the 125th running of the Boston Marathon on Oct. 11 to raise money for UMass Medical School’s ALS Cellucci Fund.

The Dan and Diane Riccio Fund for Neuroscience has awarded four teams of UMass Medical School researchers $50,000 seed grants for interdisciplinary collaborations leading to innovative discoveries that deepen understanding of normal brain function and what goes awry in neurological diseases.

Heather Gray-Edwards, PhD, DVM; Miguel Sena-Esteves, PhD; and Raymond Dunn, MD, are the recipients of the Office of Business Development & Innovation 2021 BRIDGE Fund grants.

Thomas Feldborg and Daria Rokina, of Denmark, chose to enroll their baby daughter, Alissa, in an investigational gene therapy for the treatment of GM2 gangliosidosis, which causes Tay-Sachs and Sandhoff diseases, to “let hope conquer the fears,” according to a report in USA Today chronicling their journey.

Apic Bio, a gene therapy company developing treatment options for patients with rare genetic diseases and co-founded by UMass Medical School’s Robert H. Brown Jr., DPhil, MD, announced that the FDA has cleared its investigational new drug application for APB-102, a gene therapy candidate designed to treat a common cause of familial amyotrophic lateral sclerosis.

The annual event, designed to stimulate high school students’ interest in neuroscience, was hosted by the Departments of Psychiatry and Neurology and the NeuroNexus Institute at UMass Medical School via Zoom on Saturday, March 20.

The 15th annual Brain Bee competition for Massachusetts high school students will be hosted by the Departments of Psychiatry and Neurology and the NeuroNexus Institute at UMass Medical School via Zoom.

Oguz Cataltepe, MD, describes the innovative neurosurgical procedure used to treat patients in the new clinical trial for a gene therapy targeting infantile Tay-Sachs and Sandhoff diseases.

Sio Gene Therapies announced that the first patient with infantile Tay-Sachs disease has been dosed in a Phase I/II trial evaluating AXO-AAV-GM2, an investigational gene therapy for the treatment of GM2 gangliosidosis, which causes Tay-Sachs and Sandhoff diseases. Miguel Sena-Esteves, PhD, is a principal scientist of the research program at UMMS.

Axovant Gene Therapies, a clinical-stage company developing innovative gene therapies based on UMass Medical School research discoveries, has announced that the FDA has lifted its clinical hold and cleared Axovant’s Investigational New Drug application to initiate a registrational study of gene therapy to treat patients with Tay-Sachs disease and Sandhoff disease.

On behalf of the UMass Medical School community, we extend our sincere condolences to Jake Kennedy’s family and to all who knew and loved him. Jake Kennedy died Tuesday, Oct. 13, at age 65, in his Salem, N.H., home of amyotrophic lateral sclerosis.

Jonathan Smith, of Worcester; Alyssa Smith, of Woodstock, Vt.; and Jennifer Bolanos, of Wilmington, are running the virtual Boston Marathon to support the UMass ALS Cellucci Fund.

UMass Medical School and Massachusetts General Hospital are the first to safely treat two research participants with a synthetic microRNA, delivered into the spinal fluid, designed to silence a human disease-causing gene. Details of the treatment appear in the New England Journal of Medicine.

Salome Funes, PhD candidate in the Graduate School of Biomedical Sciences, and the Society for the Advancement of Chicanos and Native Americans in Science are reaching out to organizations like Girls Inc. to teach young women about careers in science.

In a new Voices of UMassMed podcast, Kara Smith, MD, talks about early warning signs of Parkinson’s disease and new developments in research.

The Angel Fund for ALS Research donated $1 million last year to support the research of Robert H. Brown Jr., DPhil, MD. The sole mission of the nonprofit organization is to find a treatment and cure for ALS.