#gene-therapy

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#sickle-cell-disease

First Sickle Cell Gene Therapy Patient, 12, Leaves Hospital

Kendric Cromer received the first gene therapy for sickle cell disease, representing a significant breakthrough in treatment.

Sickle cell gene therapies roll out slowly

FDA has approved the first genetic treatments for sickle cell disease, offering new hope to patients like Olaide Adekanbi.
from www.nytimes.com
1 month ago

First Day of a New Life' for a Boy With Sickle Cell

Kendric Cromer starts a hopeful journey free from sickle cell disease thanks to new gene therapy.
from Cbsnews
1 month ago

New Jersey college student suffering from sickle cell disease has newfound hope with gene therapy at CHOP

Gene therapy at Children's Hospital of Philadelphia offers new hope for sickle cell disease, potentially being a life-changing treatment.

Gene Therapies Could Transform Treatment of Rare Blood Disorders - News Center

Gene therapy shows promising results in treating rare genetic blood disorders like beta thalassemia and sickle cell disease.

First Patient Begins Newly Approved Sickle Cell Gene Therapy

Commercially approved gene therapy offers hope to sickle cell patients like Kendric Cromer, potentially leading to a cure.
from www.nytimes.com
2 weeks ago

First Sickle Cell Gene Therapy Patient, 12, Leaves Hospital

Kendric Cromer received the first gene therapy for sickle cell disease, representing a significant breakthrough in treatment.

Sickle cell gene therapies roll out slowly

FDA has approved the first genetic treatments for sickle cell disease, offering new hope to patients like Olaide Adekanbi.

First Day of a New Life' for a Boy With Sickle Cell

Kendric Cromer starts a hopeful journey free from sickle cell disease thanks to new gene therapy.

New Jersey college student suffering from sickle cell disease has newfound hope with gene therapy at CHOP

Gene therapy at Children's Hospital of Philadelphia offers new hope for sickle cell disease, potentially being a life-changing treatment.

Gene Therapies Could Transform Treatment of Rare Blood Disorders - News Center

Gene therapy shows promising results in treating rare genetic blood disorders like beta thalassemia and sickle cell disease.

First Patient Begins Newly Approved Sickle Cell Gene Therapy

Commercially approved gene therapy offers hope to sickle cell patients like Kendric Cromer, potentially leading to a cure.
moresickle-cell-disease
#biotechnology

In other news this week: Knave secures 225M, London-based Purespring raises 95M, VERBUND X Ventures invests in Ausrtia's Easelink, and more - Silicon Canals

Tech innovations in October highlighted significant funding rounds for companies like Knave, Purespring, and Basecamp Research.

Swiss startup creates modular device for on-demand cell therapy manufacturing

Automated cell therapy manufacturing with less stress and contamination through modular device.

In other news this week: Knave secures 225M, London-based Purespring raises 95M, VERBUND X Ventures invests in Ausrtia's Easelink, and more - Silicon Canals

Tech innovations in October highlighted significant funding rounds for companies like Knave, Purespring, and Basecamp Research.

Swiss startup creates modular device for on-demand cell therapy manufacturing

Automated cell therapy manufacturing with less stress and contamination through modular device.
morebiotechnology
#healthcare-costs

Cures for rare diseases now exist. Employers don't want to pay

The rise of multimillion-dollar gene therapies is pushing employers to drop coverage, leaving families in desperate situations.

Lifesaving gene therapy for kids is world's priciest drug at $4.25M

Gene therapy approved for rare disease dramatically improves quality of life for children.
High price of the therapy raises concerns about affordability and access.

Cures for rare diseases now exist. Employers don't want to pay

The rise of multimillion-dollar gene therapies is pushing employers to drop coverage, leaving families in desperate situations.

Lifesaving gene therapy for kids is world's priciest drug at $4.25M

Gene therapy approved for rare disease dramatically improves quality of life for children.
High price of the therapy raises concerns about affordability and access.
morehealthcare-costs
#vision-loss

Trinity College Dublin scientists make breakthrough on gene therapy for eye disease described as 'silent thief of sight'

Gene therapy shows promise in protecting vision by improving retinal cell function in glaucoma patients.

CRISPR therapy restores some vision to people with blindness

CRISPR-based gene therapy improved vision in individuals with inherited blindness.

Trinity College Dublin scientists make breakthrough on gene therapy for eye disease described as 'silent thief of sight'

Gene therapy shows promise in protecting vision by improving retinal cell function in glaucoma patients.

CRISPR therapy restores some vision to people with blindness

CRISPR-based gene therapy improved vision in individuals with inherited blindness.
morevision-loss

Goldman Sachs Recommends These 3 Growth Stocks As Strong Buys in September

Goldman Sachs identifies three compelling growth stocks worth considering, signaling a shift towards diverse sectors amidst a technology market rotation.

BioMarin continues wave of California biotech layoffs with 128 more cuts

BioMarin Pharmaceutical is laying off 225 employees as part of an organizational redesign due to financial pressures from gene therapy projects.

New Gene Delivery Method Paves the Way for Advanced Brain Therapies

AAVs have potential for gene therapy but struggle to cross the blood-brain barrier, complicating treatments for brain disorders.

How Do You Get Drugs to the Brain? Maybe Try a Parasite

Toxoplasma engineered to deliver proteins may offer treatment for brain disorders like Rett syndrome and neurodegenerative diseases.

NHS to offer life-changing' gene therapy for blood disorder thalassaemia

Casgevy gene therapy approved for severe thalassaemia on NHS in England, potentially offering a lifetime cure with significant benefits for patients.
#clinical-trials
from Nature
5 months ago
OMG science

The immune system can sabotage gene therapies - can scientists rein it in?

Gene therapy advancements face challenges in re-dosing after initial treatment.

More children gain hearing as gene therapy for profound deafness advances

Gene therapy is offering children with genetic deafness the ability to hear normally without the need for hearing aids or cochlear implants.

The immune system can sabotage gene therapies - can scientists rein it in?

Gene therapy advancements face challenges in re-dosing after initial treatment.

More children gain hearing as gene therapy for profound deafness advances

Gene therapy is offering children with genetic deafness the ability to hear normally without the need for hearing aids or cochlear implants.
moreclinical-trials
#genetic-disorder

Argentinian couple moves to US to allow their toddler to join gene therapy trial

Parents refused to give up despite rare disease diagnosis, traveled for gene therapy trial, showing hope and perseverance.

Angiodema: Gene therapy treats painful hereditary disorder

Gene therapy has been successful in treating angiodema, a genetic disorder that causes painful swelling attacks.
The single-dose gene therapy using Crispr-Cas9 showed potential as a permanent cure for angiodema and has the potential to be a treatment for other genetic conditions.

Argentinian couple moves to US to allow their toddler to join gene therapy trial

Parents refused to give up despite rare disease diagnosis, traveled for gene therapy trial, showing hope and perseverance.

Angiodema: Gene therapy treats painful hereditary disorder

Gene therapy has been successful in treating angiodema, a genetic disorder that causes painful swelling attacks.
The single-dose gene therapy using Crispr-Cas9 showed potential as a permanent cure for angiodema and has the potential to be a treatment for other genetic conditions.
moregenetic-disorder
#medical-breakthrough

Great Ormond Street hoping to license gene therapy for bubble baby' syndrome

Gene therapy trial had high success but was dropped by pharmaceutical company, prompting Great Ormond Street hospital to license the therapy non-profit for wider access.

Deaf baby hears for the first time after 'groundbreaking' gene therapy trial

Opal Sandy, born deaf due to a genetic condition, can now hear unaided after groundbreaking gene therapy.

UK toddler has hearing restored in world first gene therapy trial

Significant milestone in gene therapy for treating deafness.

Gene therapy hailed as medical magic wand' for hereditary swelling disorder

A groundbreaking gene therapy has provided a permanent cure for patients with hereditary angioedema, a rare and debilitating disease.
Patients in the first human trial of the therapy experienced a dramatic improvement in their symptoms and were able to come off long-term medication.

Gene therapy trial restores hearing in both ears for deaf children

Gene therapy trial restores hearing in deaf children, showing promise for future treatments.

Great Ormond Street hoping to license gene therapy for bubble baby' syndrome

Gene therapy trial had high success but was dropped by pharmaceutical company, prompting Great Ormond Street hospital to license the therapy non-profit for wider access.

Deaf baby hears for the first time after 'groundbreaking' gene therapy trial

Opal Sandy, born deaf due to a genetic condition, can now hear unaided after groundbreaking gene therapy.

UK toddler has hearing restored in world first gene therapy trial

Significant milestone in gene therapy for treating deafness.

Gene therapy hailed as medical magic wand' for hereditary swelling disorder

A groundbreaking gene therapy has provided a permanent cure for patients with hereditary angioedema, a rare and debilitating disease.
Patients in the first human trial of the therapy experienced a dramatic improvement in their symptoms and were able to come off long-term medication.

Gene therapy trial restores hearing in both ears for deaf children

Gene therapy trial restores hearing in deaf children, showing promise for future treatments.
moremedical-breakthrough

Breakthrough therapies at Stanford are saving lives. Can we afford them?

Cell and gene therapies offer promising medical solutions but come with a hefty price tag, raising concerns about availability and affordability.

Young Patient Dies After Receiving Pfizer Gene Therapy

The tragic death of a young boy during a Pfizer medical trial for Duchenne muscular dystrophy has raised concerns and questions among scientists and parents.

Gene therapy trials treat inherited blindness and deafness DW 05/09/2024

The article highlights the groundbreaking achievement of restoring hearing and vision through gene therapy in children with rare genetic mutations.

Stealthy stem cells to treat disease

Researchers can create specialized cell types from stem cells to treat diseases.
Immune-edited stem cells, via gene therapy like CRISPR-Cas, could lead to mass-produced cell therapies without immune suppression.

Vertex's CRISPR Gene Therapy Lands Another FDA Nod in a Rare Blood Disease

Vertex Pharmaceuticals and CRISPR Therapeutics' gene therapy, Casgevy, has received FDA approval as a treatment for beta thalassemia.
The therapy provides a one-time treatment option for transfusion-dependent beta thalassemia patients age 12 and older.

In a World First, a Patient's Antibody Cells Were Just Genetically Engineered

Seattle-based biotech company, Immusoft, has genetically programmed a patient's B cells to treat a genetic disorder known as mucopolysaccharidosis type I (MPS I).
The patient's B cells are engineered to produce an essential enzyme that his body doesn't produce, eliminating the need for regular infusions of the enzyme.

New sickle cell treatment has roots at Children's Hospital of Philadelphia

The FDA has approved the first gene therapy treatments for sickle cell disease, a blood disorder that primarily affects people of color.
The gene therapy was tested at Children's Hospital of Philadelphia and has shown promising results in patients.

'It's Transformed My Life': FDA Approves First Gene-Editing Treatment for Illness | KQED

The CRISPR treatment for sickle cell disease involves editing a gene with CRISPR and infusing the modified cells back into patients.
The therapy, Casgevy, is designed to be a one-time treatment that alleviates symptoms for a lifetime.
#gene therapy

New Sickle Cell Therapies Will Be Out of Reach Where They Are Needed Most

FDA approves groundbreaking gene therapies for sickle cell disease
Access to these therapies remains a challenge for patients in countries where sickle cell is prevalent

The First Crispr Medicine Is Now Approved in the US

Casgevy uses Crispr technology to modify patients' cells and produce healthy hemoglobin.
Casgevy has shown promising results in a clinical trial, with 29 out of 31 patients being free of pain crises for at least a year.

World's first gene therapy for sickle cell and thalassemia approved in the U.K.

The UK has authorized the first gene therapy treatment for sickle cell disease, offering potential relief to thousands of patients.
Casgevy, made by Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine licensed using the gene editing tool.
Gene therapy offers a potential cure for sickle cell disease and thalassemia, which have previously only been manageable through difficult treatments.

The First Crispr Medicine Just Got Approved

Sickle cell disease and beta-thalassemia are painful, lifelong conditions that can be fatal.
Casgevy, a gene therapy treatment, aims to restore working hemoglobin in the body.
Trials have shown that Casgevy can reduce severe pain crises and the need for blood transfusions in patients with these conditions.

A Single Infusion of a Gene-Editing Treatment Lowered High Cholesterol

Researchers have shown that a single infusion of a gene-editing treatment can reduce cholesterol levels in people.
The treatment aims to permanently lower cholesterol by using Crispr to edit a gene in the liver, and initial results show significant reductions in LDL cholesterol.
Gene editing could provide a lasting option for treating hereditary high cholesterol, replacing the need for long-term medication.

New Sickle Cell Therapies Will Be Out of Reach Where They Are Needed Most

FDA approves groundbreaking gene therapies for sickle cell disease
Access to these therapies remains a challenge for patients in countries where sickle cell is prevalent

The First Crispr Medicine Is Now Approved in the US

Casgevy uses Crispr technology to modify patients' cells and produce healthy hemoglobin.
Casgevy has shown promising results in a clinical trial, with 29 out of 31 patients being free of pain crises for at least a year.

World's first gene therapy for sickle cell and thalassemia approved in the U.K.

The UK has authorized the first gene therapy treatment for sickle cell disease, offering potential relief to thousands of patients.
Casgevy, made by Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine licensed using the gene editing tool.
Gene therapy offers a potential cure for sickle cell disease and thalassemia, which have previously only been manageable through difficult treatments.

The First Crispr Medicine Just Got Approved

Sickle cell disease and beta-thalassemia are painful, lifelong conditions that can be fatal.
Casgevy, a gene therapy treatment, aims to restore working hemoglobin in the body.
Trials have shown that Casgevy can reduce severe pain crises and the need for blood transfusions in patients with these conditions.

A Single Infusion of a Gene-Editing Treatment Lowered High Cholesterol

Researchers have shown that a single infusion of a gene-editing treatment can reduce cholesterol levels in people.
The treatment aims to permanently lower cholesterol by using Crispr to edit a gene in the liver, and initial results show significant reductions in LDL cholesterol.
Gene editing could provide a lasting option for treating hereditary high cholesterol, replacing the need for long-term medication.
moregene therapy

FDA Approves First CRISPR Treatment in U.S.

The U.S. FDA has approved the first gene therapy treatment based on CRISPR technology.
The treatment, called exa-cel, is approved for sickle cell disease and may also be approved for beta thalassemia.

FDA approves first CRISPR gene editing treatment that may cure sickle cell disease

Regulators have approved two new gene therapies for sickle cell disease that could potentially cure the inherited blood disorder.
The treatments are the first to be approved based on CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020.

FDA approves CRISPR-based therapy for sickle cell disease

The FDA has approved the first therapy using CRISPR gene-editing technology to treat sickle cell disease.
The technology has the potential to be applied to other blood disorders, certain cancers, and infectious diseases.

Breakthrough Gene Treatments For Sickle Cell Disease Approved

Regulators have approved two new gene therapies for sickle cell disease, offering hope for a cure for the painful blood disorder
The treatments are designed for patients 12 and older with severe forms of the disease, and one of them is the first therapy based on CRISPR gene editing

Possible Therapy for Sickle Cell Won't Cure Its Racist Legacy - Non Profit News | Nonprofit Quarterly

The FDA is reviewing an experimental gene therapy treatment for sickle cell disease.
Racism has significantly affected the healthcare and outcomes of sickle cell disease patients.

Casgevy: UK approves gene-editing drug for blood disorders

The UK has approved a gene therapy using the Crispr gene-editing tool to treat sickle cell disease and beta thalassemia, a first in the world.
The treatment involves removing stem cells from a patient's bone marrow and using Crispr to disable the faulty gene, allowing the body to produce functioning haemoglobin.
Trials have shown promising results, with the majority of patients experiencing relief from symptoms and no longer needing blood transfusions.

CRISPR Therapy Gets U.K. Approval, the First in the World

Britain's medicines regulator has approved the world's first gene therapy for sickle cell disease, offering hope to thousands of patients.
Casgevy is the first treatment licensed using the gene editing tool CRISPR, and it targets the problematic gene in a patient's bone marrow stem cells.
Both sickle cell disease and thalassemia are caused by genetic mutations that affect the production of hemoglobin in red blood cells.

Growing success in new gene therapy for inherited deafness- Harvard Gazette

Successful gene therapy trial for genetic deafness promising for future availability.

Daily briefing: 'Open-washing' plagues big AI

Open-source AI not as transparent as claimed
Low-frequency vibrations trigger mouse sexual behaviors
Importance of research putting individuals at the heart of schizophrenia study
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