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Home » Topics » Disease categories and therapies » Genetic/congenital

Genetic/congenital
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Ocular

AAV8-RK-hBBS10 gene therapy restores vision in Bardet-Biedl syndrome type 10 model

May 29, 2025
No Comments
Bardet-Biedl syndrome (BBS) is a group of rare autosomal recessive ciliopathies characterized by dysfunction of primary cilia, which affects multiple organ systems and leads to early-onset obesity, progressive retinal degeneration resulting in vision loss or blindness, and renal abnormalities that may progress to renal failure. Mutations in the BBS10 gene are the second most prevalent cause of BBS, accounting for over 20% of cases.
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Heart scientific overlay
Cardiovascular

Frontera’s FT-017 exerts robust activity in hypertrophic cardiomyopathy

May 27, 2025
No Comments
Frontera Therapeutics Inc. has developed FT-017, an adenovirus-associated vector(AAV)-based gene therapy that carries a human MYBPC3 optimized codon, for the treatment of hypertrophic cardiomyopathy (HCM).
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DNA data illustration
Genetic/congenital

Latus Bio’s LTS-101 is a potential therapeutic approach for Batten disease

May 26, 2025
No Comments
Latus Bio Inc. is developing a new gene therapy, LTS-101, for the treatment of neuronal ceroid lipofuscinosis type2 (CLN2), a form of Batten disease characterized by deficiency in the tripeptidyl peptidase 1 (TPP1) protein that leads to lysosomal dysfunction and neurodegeneration.
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Sickle cell illustration
Cancer

Study probes link between sickle cell disease and kidney cancer

May 26, 2025
By Anette Breindl
No Comments
Individuals with both sickle cell disease (SCD) and sickle cell trait are at higher risk than others of developing renal medullary cancer (RMC), the rarest and deadliest subtype of kidney cancer. Researchers at MD Anderson Cancer Center have identified the molecular mechanisms behind the increased risk, gaining new insights into antitumor immunity more generally and, potentially, new ways to treat RMC, and possibly other tumors as well.

SCD “has been studied for 30 years, but 95% of the effort [has been] working on the red blood cells … how red blood cells contribute to hypoxia and then reduce oxygen supply,” Chunru Lin told BioWorld.
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Neurology/psychiatric

Latus Bio presents novel therapy for Huntington’s disease

May 23, 2025
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Researchers from Latus Bio Inc. have developed a novel adeno-associated virus (AAV) therapy for treating Huntington’s disease.
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Art concept for mouse model
Drug design, drug delivery & technologies

From mice to big animal models in gene therapy for rare diseases

May 23, 2025
By Mar de Miguel
No Comments
The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare disorder affecting brain development for which a mouse model has finally been developed. Other times, small and large models exist, but an effective treatment remains elusive, as is the case with Krabbe disease, a fatal disease in children that could be prevented with the advances in gene therapy.
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DNA double helix under a magnifying glass
Drug design, drug delivery & technologies

Base and prime editions to achieve genetic cure

May 21, 2025
By Mar de Miguel
No Comments
Since the development of the base and prime editing technique by David Liu at the Broad Institute, their applications in biomedicine have continued to grow, reaching 17 clinical trials for base editing and one clinical assay for prime editing. The 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) marked a historic milestone this year by presenting the first case of treatment with base editors of a baby with a deadly metabolic disease.
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Children’s Hospital of Philadelphia
ASGCT 2025

First bespoke gene editing therapy treats rare metabolic disease

May 20, 2025
By Anette Breindl
No Comments
Using a customized gene editing therapy, researchers at the Children’s Hospital of Philadelphia have reported success in treating an infant with a severe metabolic disorder. Kiran Musunuru, Barry J. Gertz Professor for Translational Research in the University of Pennsylvania’s Perelman School of Medicine, presented the case at the American Society of Gene and Cell Therapy’s 2025 annual meeting. The case study was simultaneously published in The New England Journal of Medicine.
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Art concept for gene therapy research
Neurology/psychiatric

Caspida’s CAP-004 exhibits potential as a best-in-class therapy for Friedreich’s ataxia

May 16, 2025
No Comments
Capsida Biotherapeutics Inc. has reported development of a systematically administered capsid, CAP-004.
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Illustration of human brain and dna
Neurology/psychiatric

Quralis signs agreements to further fragile X syndrome research

May 16, 2025
No Comments
Quralis Corp. has entered into a number of agreements with the aim of advancing the treatment of fragile X syndrome, a genetic condition caused by a mutation of a single gene – fragile X messenger ribonucleoprotein 1 (FMR1) – on the X chromosome.
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