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Gene Therapy

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April 12, 2024
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Patient dosing continues in clinical trial of novel gene therapy for Wilson’s disease

Patient dosing continues in clinical trial of novel gene therapy for Wilson’s disease

A European clinical stage biotech company announced dosing of the first patient in cohort 2 of its ongoing phase 1/2 GATEWAY clinical trial of lead candidate VTX-801 to treat Wilson’s disease.

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March 28, 2024
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Opus Genetics completes dosing in first cohort of Leber congenital amaurosis trial

Opus Genetics completes dosing in first cohort of Leber congenital amaurosis trial

Dosing is complete in the first cohort of the phase 1/2 clinical trial of OPGx-LCA5 for the treatment of Leber congenital amaurosis, according to a press release from Opus Genetics.

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March 28, 2024
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The Latest News and Notes, plus Conversation with Wiley A. Chambers, MD

The Latest News and Notes, plus Conversation with Wiley A. Chambers, MD

In this episode, John A. Hovanesian, MD, FACS, and Jim Mazzo discuss the latest news in eye care and chat with Wiley A. Chambers, MD, about his career in the FDA and his plans for retirement.

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March 19, 2024
2 min read
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FDA approves Lenmeldy for early juvenile metachromatic leukodystrophy

FDA approves Lenmeldy for early juvenile metachromatic leukodystrophy

The FDA has approved Lenmeldy as the first gene therapy to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy.

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March 12, 2024
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VIDEO: Searching for new mechanisms of action for diabetic eye disease treatments

VIDEO: Searching for new mechanisms of action for diabetic eye disease treatments

In this video, Diana Do, MD, discusses the impact of socioeconomic disparities on patients with diabetic eye diseases.

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February 28, 2024
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VIDEO: Several treatments in the pipeline for geographic atrophy

VIDEO: Several treatments in the pipeline for geographic atrophy

Durga S. Borkar, MD, MMCi, assistant professor of ophthalmology and director of clinical data science Duke University Eye Center touches on geographic atrophy therapies currently in the pipeline and how they will contribute to the future of treatment.

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February 25, 2024
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Researchers predict ‘friction’ between payers, physicians, patients over cost of B-VEC

Researchers predict ‘friction’ between payers, physicians, patients over cost of B-VEC

The FDA’s broad indication for the use of beremagene geperpavec therapy for the treatment of dystrophic epidermolysis bullosa may have large financial implications for payers, according to a study.

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February 23, 2024
2 min read
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Topical gene therapy improves ocular complications of dystrophic epidermolysis bullosa

Topical gene therapy improves ocular complications of dystrophic epidermolysis bullosa

Ophthalmic application of beremagene geperpavec was successful in treating recurrent cicatrizing conjunctivitis in one patient with dystrophic epidermolysis bullosa, according to a study.

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February 16, 2024
3 min read
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ASH president aims to strengthen hematology workforce, address global challenges

ASH president aims to strengthen hematology workforce, address global challenges

Mohandas Narla, DSc, may hold the title of American Society of Hematology president, but his focus for the next several months will extend far beyond this country’s borders.

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January 09, 2024
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FDA official says CAR-T ‘incredibly beneficial’ despite secondary malignancy risk

FDA official says CAR-T ‘incredibly beneficial’ despite secondary malignancy risk

Benefits of chimeric antigen receptor T-cell therapy far outweigh the risks involved, including secondary T-cell malignancies, according to Peter Marks, MD, PhD, director of the FDA’s Center for Biologic Evaluation and Research.

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