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from www.nytimes.com
2 weeks ago
Medicine

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.

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.

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.

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.

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.

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.

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.

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.
moregene-therapy

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.
#sickle cell disease

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.

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.
moresickle cell disease

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.

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.
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