genetic disorders Archives

News and Trends 10 Feb 2023

Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment

Rocket Pharmaceuticals, Inc says the U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to RP-A501. RP-A501 is Rocket Pharmaceuticals’ investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon disease, a devastating and fatal genetic cardiac disease for which there are no disease-altering therapies available. RMAT designation was […]

February 10, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 3 Feb 2023

AI boosts genome editing

Researchers at the University of Zurich have developed a new tool that uses artificial intelligence to predict the efficacy of various genome-editing repair options. Unintentional errors in the correction of DNA mutations of genetic diseases can thus be reduced. Genome editing technologies offer great opportunities for treating genetic diseases. Methods such as the widely used […]

February 3, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 26 Jan 2023

Milestone for gene-edited therapy for sickle cell disease

Vertex Pharmaceuticals (Europe) and CRISPR Therapeutics have announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application of exa-cel for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The submission is supported by two global phase 3 studies investigating exa-cel as a potential one-time therapy for people with […]

January 26, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 9 Jan 2023

Selecta and Astellas sign LOPD treatment agreement

Selecta Biosciences, Inc. and Astellas Pharma Inc. have announced an exclusive licensing and development agreement for IdeXork (Xork). Xork is being studied as a potential next generation immunoglobulin G (IgG) protease that will be developed by Astellas for use with AT845, an investigational, adeno-associated virus (AAV)-based treatment for late-onset Pompe disease (LOPD) in adults. Pompe […]

January 9, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 14 Dec 2022

NMD Pharma cleared for spinal muscular atrophy trial

NMD Pharma A/S has received clearance for its investigational new drug (IND) application from the US Food and Drug Administration (FDA) to advance NMD670 into a phase II clinical study in patients of spinal muscular atrophy (SMA) type 3. NMD Pharma is a Danish clinical stage biotech company developing first-in-class, small molecule ClC-1 inhibitors for […]

December 14, 2022 - 2 minutesmins - By Jim Cornall

News and Trends 8 Dec 2022

Metrion and KCNC1 Foundation announce collaboration to identify therapeutic

Metrion Biosciences Limited, and The KCNC1 Foundation are to collaborate to try and find a therapeutic for children. The KCNC1 Foundation is a not-for-profit organization focused on the development of a treatment for an ultra-rare genetic disorder. Variants of the KCNC1 gene impact Kv3.1 potassium channel function, resulting in neurodevelopmental disorders, which can include progressive […]

December 8, 2022 - 2 minutesmins - By Liza Laws

News and Trends 17 Nov 2022

New mitochondria study could pave way for disease treatments

Mitochondria, the ‘powerhouses of the cell,’ are important cellular structures that are vital for their role of generating energy. They constantly fuse together and split apart. They contain a small amount of genetic information, mitochondrial DNA (mtDNA). The mtDNA, organized into dot-like structures called nucleoids, also moves around inside the mitochondria. The method of distribution […]

November 17, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 16 Nov 2022

Enzymes could be key to understanding ‘origin of DNA errors’

Enzymes, critical to controlling how cells replicate in the human body, could be the very ingredient that encourages DNA to spontaneously mutate – causing potentially permanent genetic errors, according to new research from the University of Surrey in the U.K. Using quantum chemical calculations, researchers from Surrey’s Quantum Biology Doctoral Training Centre have found that […]

November 16, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 10 Aug 2022

Aro Biotherapeutics gets FDA orphan drug designation for treatment of Pompe Disease

Aro Biotherapeutics says the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ABX1100, an investigational Centyrin-siRNA conjugate that targets the Gys1 gene in the muscle, for the treatment of Pompe disease. “We are pleased to have received this designation and are gratified by the FDA’s recognition of the potential of ABX1100 […]

August 10, 2022 - 2 minutesmins - By Jim Cornall

News and Trends 27 Jun 2022

Trial for adults with late onset Pompe disease halted due to serious adverse event

The US Food and Drug Administration (FDA) has placed a clinical hold on a trial being run for Astrellas Pharma Inc after one of the participants had a serious adverse event (SAE) of peripheral sensory neuropathy. Astrellas made the announcement after the trial, which is evaluating AT845, an investigational adeno-associated virus (AAV) gene replacement in […]

June 27, 2022 - 3 minutesmins - By Liza Laws

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genetic disorders

Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment

AI boosts genome editing

Milestone for gene-edited therapy for sickle cell disease

Selecta and Astellas sign LOPD treatment agreement

NMD Pharma cleared for spinal muscular atrophy trial

Metrion and KCNC1 Foundation announce collaboration to identify therapeutic

New mitochondria study could pave way for disease treatments

Enzymes could be key to understanding ‘origin of DNA errors’

Aro Biotherapeutics gets FDA orphan drug designation for treatment of Pompe Disease

Trial for adults with late onset Pompe disease halted due to serious adverse event

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Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

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New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

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Advertise with us

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