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.
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.
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.