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Foundation to Fight H-ABC, University of Massachusetts Medical School and Yale University Initiate Gene Therapy Study Targeting Cure for Rare Disease

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ROCKVILLE, Md., Oct. 13, 2020 /PRNewswire/ — Foundation to Fight H-ABC, a non-profit organization dedicated to increasing awareness and driving development of a cure for the degenerative children’s disease, H-ABC, today announced a sponsored research agreement with the University of Massachusetts Medical School and Yale University to advance a targeted gene therapy for H-ABC.

“We have high hopes to quickly prove efficacy with this approach to move research forward and find a permanent cure for this devastating disease,” said Michele Sloan, Co-Founder, Foundation to Fight H-ABC.

H-ABC (hypomyelination with atrophy of the basal ganglia and cerebellum) belongs to a group of conditions called leukodystrophies, diseases that affect the white matter of the brain. These diseases disrupt the growth or maintenance of the myelin sheath, a protective layer that insulates nerve cells and allows for the transmission of messages between cells.

Caused by a mutation in the TUBB4A gene, H-ABC is a rare genetic disorder that affects certain parts of the brain—specifically the basal ganglia and the cerebellum, which control movement. H-ABC targets these important structures, reducing both their size and function. As a result, children who suffer from H-ABC often experience motor problems, cannot walk, talk, or sit on their own. Currently, there is no known cure for this disabling and life-threatening condition.

The teams of Dr. Guangping Gao (University of Massachusetts Medical School) and Dr. Karel Liem (Yale School of Medicine) will combine extensive expertise in the fields of Adeno-associated virus (AAV), a platform for gene delivery for the treatment of a variety of human diseases and H-ABC disease models, to develop AAV vectors to silence or outcompete the mutated TUBB4A gene.

“To date, AAV-based gene delivery system is the vector of choice for in vivo gene therapy of many currently untreatable rare diseases including H-ABC,” said Guangping Gao,

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E.U. Court Rules Against Hungary Law Targeting Soros-Funded University

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BUDAPEST — The European Court of Justice ruled on Tuesday that Hungary had violated E.U. rules by changing legislation in 2017 that effectively expelled an American university founded by the billionaire financier George Soros from the country.

“The conditions introduced by Hungary to enable foreign higher education institutions to carry out their activities in its territory are incompatible with E.U. law,” the court’s ruling said.

The decision was the latest effort by the European Union to curb growing authoritarianism by the government of Prime Minister Viktor Orban, but is unlikely to have much impact on the ground in Hungary.

The ruling leaves no room for appeal, and requires Mr. Orban’s government to change the legislation to come in line with E.U. laws. If Hungary does not amend the law, the European Commission, the bloc’s executive arm, could request that the high court impose fines on the Hungarian government until it does so.

But the expelled institution, Central European University, is unlikely to restore its former setup in Hungary. Michael Ignatieff, the university’s rector, has said that it will be anchored in Vienna, where it has moved the bulk of its operations.

“We cannot return to Hungary, because its prevailing laws don’t meet the requirements of academic freedom,” Mr. Soros said in a statement on Tuesday. “The Hungarian government continues to trample E.U. law, with the latest victim being the world-renowned University of Theatre and Arts,” he added, referring to a Hungarian university that was the site of student protests last months over the increasing influence of Mr. Orban’s government in the school’s affairs.

Andras Lederer, a program coordinator with the Hungarian Helsinki Committee, a human rights organization in Budapest, said the Central European University case highlighted the need for the European Commission to step in early and suspend legislation it