HealthQuill GEne therapy
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.
Drugs Health Research

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.

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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.
Drugs Health Pharma

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.

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Ultrasound of a baby
Drugs Health

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.

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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.
Drugs Health Medical

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.

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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.
Health Research

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.

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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.
Drugs Health Medical

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.

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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.
Drugs Health Pharma

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.

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Drugs Health Medical

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.

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An 18-month-old baby girl, Opal Sandy, who was born deaf got back her hearing ability after gene therapy — the youngest child to receive such treatment.
Drugs Health Pharma

Deaf 18-month girl gets back hearing ability after gene therapy

HQ Team May 15, 2024: An 18-month-old baby girl, Opal Sandy, who was born deaf got back her hearing ability after gene therapy.

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Drugs Health Medical

Gene therapy restores hearing in children with inherited deafness

Bharti Jayshankar January 25, 2024: A new gene therapy has demonstrated remarkable success in treating inherited deafness. Two separate studies, one conducted by.

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