Rare Diseases Research News - The Li Weibo (李伟波) Institute for Rare Diseases Research

Phillip Tai, PhD, received a $1.6 million grant from the National Institute of General Medical Sciences to develop adeno-associated viral vectors for gene therapies.

Terence R. Flotte, MD, and colleagues outline plans for addressing the current market challenges for gene therapy treatment of rare and ultra-rare disorders.

Toloo Taghian, PhD, a has received a $3.2 million NIH grant to develop a gene therapy for UBA5 deficiency.

UMass Chan licenses gene therapy technology to treat maple syrup urine disease (MSUD) to Plowshare Therapies.

Robert H. Brown Jr., DPhil, MD, is one of 252 people elected to the academy this year.

Fen-Biao Gao, PhD, has been named a fellow of the American Association for the Advancement of Science.

The Angel Fund for ALS Research has awarded $800,000 to UMass Chan to accelerate research into ALS.

UMass Chan has licensed an adeno-associated virus gene therapy to the Raiden Science Foundation to develop a clinical treatment for UBA5 disorder.

Top story: UMass Chan Medical School has launched Rare Diseases, Real Stories, a six-episode podcast series. This is Catherine's story. It is available on all major streaming platforms.

Four faculty members were honored with chancellor's medals at the annual Convocation and Investiture ceremony.

These findings, published in Nature, point to potential new approaches to protect young infants from perinatal HIV transmission.

PhD candidate Bradley Class has received a NIH grant to further his research on neuron mutations and ataxia-telangiectasia, a rare disease characterized by impaired motor function, dilated blood vessels and a weakened immune system.

UMass Chan Medical School scientists Jun Xie, PhD, and Guangping Gao, PhD, have received $500,000 to help advance gene therapy.

UMass Chan researchers demonstrated the promise of a gene therapy to correct a gene mutation causing maple syrup urine disease.

UMass Chan Medical School has launched Rare Diseases, Real Stories, a six-episode podcast series. It is available on all major streaming platforms.

A family whose child has a rare neurological disease, SPG4, visited UMass Chan Medical School’s scientists to learn about development of a gene therapy for the disease. The family is raising funds to support the research in the Translational Institute for Molecular Therapeutics.

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.

UMass Chan Medical School has received $2.2 million from a nonprofit patient-advocacy organization to contract with Andelyn Biosciences to manufacture clinical grade AAV9-CSA vector to treat Cockayne syndrome.

The story of Raiden Pham, an Oregon toddler with an ultra-rare genetic disease was included on the Lunaprise Museum, a digital time capsule that went to the moon with the Odysseus spacecraft in February. Tommy and Linda Pham founded Raiden Science Foundation in 2021 to raise money to support research on a treatment at UMass Chan’s Translational Institute for Molecular Therapeutics.

Dean Flotte testified in support of policies that would encourage and accelerate gene therapy for rare diseases, in a Feb. 29 hearing before the House Committee on Energy and Commerce’s Subcommittee on Health.

Rare disease research news from UMass Chan Medical School

UMass Chan scientist receives $1.6 million from National Institute of General Medical Sciences to refine gene therapy vectors

Terence R. Flotte addresses challenges to commercialization of gene therapy treatments for rare diseases

Toloo Taghian awarded $3.2M to develop gene therapy for a rare genetic disease

UMass Chan licenses gene therapy for maple syrup urine disease to Plowshare Therapies

Robert H. Brown Jr. elected to American Academy of Arts & Sciences

Fen-Biao Gao named fellow of the American Association for the Advancement of Science

UMass Chan awarded $800K to advance ALS research

UMass Chan licenses gene therapy for rare genetic disorder to Raiden Science Foundation

Top story: Catherine’s story: Tay-Sachs disease

Chancellor Collins’ final Convocation address encourages optimism, determination

Gene therapy vector produced by UMass Chan used to deliver prophylactic therapy for HIV

PhD candidate Bradley Class receives NIH grant to study Louis-Bar syndrome

Origen Biotechnology funds research at UMass Chan to advance gene therapy technology

Gene therapy developed for maple syrup urine disease shows promise, new UMass Chan study reports

Listen now: Rare Diseases, Real Stories podcast series

Family with child affected by SPG4 visits UMass Chan researchers developing gene therapy

UMass Chan Medical School joins sponsored research agreement with Astellas Pharma

UMass Chan receives $2.2 million to fund gene therapy for Cockayne syndrome

Story of boy with ultra-rare UBA5 disorder being studied at UMass Chan goes to the moon

Dean Flotte testifies at congressional hearing in support of gene therapy development