GEne therapy - HealthQuill

Pfizer Inc. announced that USFDA has accepted its biologics license application for treating bleeding disorders in adults through gene therapy.

Gene therapy slows Huntington’s Disease progression by 75%

HQ Team September 25, 2025: Scientists in the United Kingdom have found a novel gene therapy known as AMT-130 that has slowed the.

by HealthQuill
September 25, 2025
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Stanford University, along with other researchers, has developed a set of gene delivery systems that can reach different neural cell types in the human brain and spinal cord with exceptional accuracy — a tool which may lead to new gene therapies to treat Parkinson’s, Alzheimer’s and neurogenerative diseases.

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Scientists make gene ‘truck’ to unload genetic package in brain, spine

HQ Team May 30, 2025: Stanford University, along with other researchers, has developed a set of gene delivery systems that can reach different.

by HealthQuill
May 30, 2025
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The US drug regulator halted Rocket Pharmaceuticals Inc.’s investigational gene therapy for Danon disease after a patient died due to an infection, according to a company statement.

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FDA halts Rocket’s Danon disease mid-stage therapy as patient dies

HQ Team May 28, 2025: The US drug regulator halted Rocket Pharmaceuticals Inc.’s investigational gene therapy for Danon disease after a patient died.

by HealthQuill
May 28, 2025
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Fetus treated with risdiplam for rare genetic disorder, a medical first

HQ Team February 27, 2025: A study published in The New England Journal of Medicine (NEJM) reveals that administration of the oral drug.

by HealthQuill
February 27, 2025
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Regeneron Pharmaceuticals, Inc.’s investigational gene therapy improved hearing in 10 out of 11 children born with hearing loss because of gene mutation, according to a company statement.

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Regeneron’s trial therapy improves hearing in children with gene mutations

Regeneron Pharmaceuticals, Inc.’s investigational gene therapy improved hearing in 10 out of 11 children born with hearing loss because of gene mutation, according.

by HealthQuill
February 25, 2025
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Moderna Inc., a biotechnology company, and Merck announced that an experimental personalised cancer vaccine, based on the messenger RNA (mRNA) technology, has received Breakthrough Therapy Designation from the USFDA.

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Breakthrough system allows mRNA delivery through blood-brain barrier

February 18, 2025: Scientists at the Icahn School of Medicine at Mount Sinai have achieved a major breakthrough in brain therapy by developing.

by HealthQuill
February 18, 2025
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China’s YolTech Therapeutics, a biotechnology startup, has started its clinical trial of an investigational therapy for treating transfusion-dependent beta-thalassemia (TDT), a severe genetic blood disorder.

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China’s YolTech starts gene editing trial for treating a blood disorder

China’s YolTech Therapeutics, a biotechnology startup, has started its clinical trial of an investigational therapy for treating transfusion-dependent beta-thalassemia (TDT), a severe genetic.

by HealthQuill
January 23, 2025
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Pfizer Inc., got a nod from the US drug regulator for its drug Hympavzi to treat two types of a rare blood disorder in haemophilia patients.

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Pfizer’s drug to treat rare haemophilia disorder gets US regulator’s approval

Pfizer Inc., got a nod from the US drug regulator for its drug Hympavzi to treat two types of a rare blood disorder.

by HealthQuill
October 13, 2024
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Breakthrough gene therapy restores hearing in children with inherited deafness

HQ Team August15, 2024: A clinical trial has successfully restored hearing in five children suffering from inherited deafness by administering gene therapy to.