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January 05, 2024
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Survey: Halting neurodegeneration chief concern in Huntington’s disease

Survey: Halting neurodegeneration chief concern in Huntington’s disease

According to results of a recent survey, more than 80% of U.S.-based neurologists agreed that a major unmet need in confronting Huntington’s disease is halting neurodegeneration prior to the onset of symptoms.

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January 05, 2024
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CMS issues permanent J-code for Vyjuvek

CMS issues permanent J-code for Vyjuvek

Vyjuvek, the first FDA-approved treatment for dystrophic epidermolysis bullosa, has received a permanent J-code from the Centers for Medicare and Medicaid Services, Krystal Biotech announced in a press release.

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January 02, 2024
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Vyjuvek receives orphan drug designation in Japan for epidermolysis bullosa

Vyjuvek receives orphan drug designation in Japan for epidermolysis bullosa

The Japanese Ministry of Health, Labour and Welfare has granted Vyjuvek an orphan drug designation for the treatment of dystrophic epidermolysis bullosa, Krystal Biotech announced in a press release.

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December 30, 2023
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ASH recap: ‘Transformative’ advance in sickle cell disease, ‘breakthrough’ in AML and ALL

This year’s ASH Annual Meeting featured several high-impact abstracts.

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December 28, 2023
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AAV-based gene therapies hold promise for treating CNS conditions

AAV-based gene therapies hold promise for treating CNS conditions

Meaningful progress is being made in the development of gene therapies for rare neurological diseases caused by single gene mutations.

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December 22, 2023
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Positive interim data shown in phase 1/2 trials of Huntington’s disease therapy

Positive interim data shown in phase 1/2 trials of Huntington’s disease therapy

A gene therapy company has announced updated interim data from ongoing United States and European phase 1/2 clinical trials of AMT-130 to treat Huntington’s disease.

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December 19, 2023
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Future of epilepsy treatment depends on overcoming obstacles to gene-targeting therapy

Future of epilepsy treatment depends on overcoming obstacles to gene-targeting therapy

ORLANDO — While effective medications for epilepsy exist, scientific advancement requires that future gene-targeting therapies or cures benefit the largest number of patients while overcoming several barriers, according to a presentation.

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December 18, 2023
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VIDEO: Updated data on hemophilia A gene therapies presented at ASH

VIDEO: Updated data on hemophilia A gene therapies presented at ASH

In this video, Lindsey A. George, MD, discussed updated clinical trial data around hemophilia gene therapy presented at ASH Annual Meeting and Exposition.

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December 18, 2023
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VIDEO: Factor VIII variant shows ‘unclear biochemical mechanism for improved function’

VIDEO: Factor VIII variant shows ‘unclear biochemical mechanism for improved function’

In this video interview with Healio, Lindsey A. George, MD, spoke about a session on factor VIII biochemistry presented at ASH Annual Meeting and Exposition.

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December 18, 2023
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VIDEO: Improving function of factor VIII variants for hemophilia A gene therapy

VIDEO: Improving function of factor VIII variants for hemophilia A gene therapy

In this video, Lindsey A. George, MD, talked about improving the function of factor VIII variants for hemophilia A gene therapy, which she presented in a session at ASH Annual Meeting and Exposition.

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