Showing 25 to 36 of 43 results
FDA Approves First Gene Therapy for RDEB, Showcasing Innovative Funding Model
The FDA approved ZEVASKYN, the first autologous cell-based gene therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB), a significant advancement driven by EB Research Partnership's innovative venture philanthropy model, offering hope to 500,000 people globally.
FDA Approves First Gene Therapy for RDEB, Showcasing Innovative Funding Model
The FDA approved ZEVASKYN, the first autologous cell-based gene therapy for Recessive Dystrophic Epidermolysis Bullosa (RDEB), a significant advancement driven by EB Research Partnership's innovative venture philanthropy model, offering hope to 500,000 people globally.
Progress
52% Bias Score
Gene Editing Offers Potential Cure for Newfoundland's 'Curse'
In Newfoundland, a genetic mutation causes arrhythmogenic right ventricular cardiomyopathy (ARVC), leading to sudden heart failure. Current treatment involves defibrillator implants, but researchers are using gene editing to correct the genetic defect in heart cells grown from patient samples, offer...
Gene Editing Offers Potential Cure for Newfoundland's 'Curse'
In Newfoundland, a genetic mutation causes arrhythmogenic right ventricular cardiomyopathy (ARVC), leading to sudden heart failure. Current treatment involves defibrillator implants, but researchers are using gene editing to correct the genetic defect in heart cells grown from patient samples, offer...
Progress
32% Bias Score
Drug Repositioning Offers Hope for Rare Disease Treatment
Professor Guillaume Canaud's successful repositioning of a cancer drug to treat Cloves syndrome, a rare disease, highlights the potential of drug repurposing to address the treatment gap for rare diseases, offering a faster and more cost-effective alternative to traditional drug development.
Drug Repositioning Offers Hope for Rare Disease Treatment
Professor Guillaume Canaud's successful repositioning of a cancer drug to treat Cloves syndrome, a rare disease, highlights the potential of drug repurposing to address the treatment gap for rare diseases, offering a faster and more cost-effective alternative to traditional drug development.
Progress
48% Bias Score
Gene Therapy Restores Sight in Children with Inherited Blindness
London scientists successfully treated childhood blindness caused by Leber Congenital Amaurosis (LCA) in four children (ages 1-2) from Turkey, Tunisia, and the USA using a groundbreaking gene therapy that involved a 60-minute laparoscopic surgery to inject healthy AIPL1 genes into their retinas, res...
Gene Therapy Restores Sight in Children with Inherited Blindness
London scientists successfully treated childhood blindness caused by Leber Congenital Amaurosis (LCA) in four children (ages 1-2) from Turkey, Tunisia, and the USA using a groundbreaking gene therapy that involved a 60-minute laparoscopic surgery to inject healthy AIPL1 genes into their retinas, res...
Progress
40% Bias Score
First Spanish Child Cured of Wiskott-Aldrich Syndrome via Gene Therapy
Five-year-old Javier, diagnosed with Wiskott-Aldrich syndrome at six months old, is the first child in Spain cured using gene therapy; this case highlights the potential of gene therapy and challenges of accessing innovative treatments for rare diseases affecting less than 1 in 250,000 male births.
First Spanish Child Cured of Wiskott-Aldrich Syndrome via Gene Therapy
Five-year-old Javier, diagnosed with Wiskott-Aldrich syndrome at six months old, is the first child in Spain cured using gene therapy; this case highlights the potential of gene therapy and challenges of accessing innovative treatments for rare diseases affecting less than 1 in 250,000 male births.
Progress
40% Bias Score
NHS to Offer £1.65m Gene Therapy for Sickle Cell Disease
England's NHS will offer the £1.65m gene-editing therapy Casgevy to about 50 sickle cell patients yearly, marking a major advance in treating this life-threatening blood disorder primarily affecting people of Black African and Black Caribbean heritage, offering a potential cure and improved quality ...
NHS to Offer £1.65m Gene Therapy for Sickle Cell Disease
England's NHS will offer the £1.65m gene-editing therapy Casgevy to about 50 sickle cell patients yearly, marking a major advance in treating this life-threatening blood disorder primarily affecting people of Black African and Black Caribbean heritage, offering a potential cure and improved quality ...
Progress
44% Bias Score
Bayer's 7% Revenue Growth in Spain Driven by Innovation and Investment
In 2024, Bayer's Spanish operations reported €822 million in revenue (a 7% increase), driven by pharmaceuticals, agriculture, and consumer health products; €145 million was invested, with €118 million dedicated to R&D and €27 million to efficiency improvements; Viralgen, a Bayer subsidiary specializ...
Bayer's 7% Revenue Growth in Spain Driven by Innovation and Investment
In 2024, Bayer's Spanish operations reported €822 million in revenue (a 7% increase), driven by pharmaceuticals, agriculture, and consumer health products; €145 million was invested, with €118 million dedicated to R&D and €27 million to efficiency improvements; Viralgen, a Bayer subsidiary specializ...
Progress
40% Bias Score
New Method to Measure PTEN Gene Activity May Advance Treatment of Developmental Disorders and Cancers
Researchers at Tel Aviv University developed a new method to precisely measure the activity of the PTEN gene, crucial for cellular growth, which may advance treatments for multiple developmental disorders and cancers; the study's findings were published in Nature Methods.
New Method to Measure PTEN Gene Activity May Advance Treatment of Developmental Disorders and Cancers
Researchers at Tel Aviv University developed a new method to precisely measure the activity of the PTEN gene, crucial for cellular growth, which may advance treatments for multiple developmental disorders and cancers; the study's findings were published in Nature Methods.
Progress
44% Bias Score
"Gene Therapy Breakthrough: Cell-Free Production Method Receives $5.2 Million in Funding"
"Moorfields Eye Hospital successfully used gene therapy to save the sight of four children with a rare blinding genetic condition; this success highlights the potential of Fuse Vectors, a Danish biotech startup, which just secured $5.2 million in funding to revolutionize gene therapy manufacturing t...
"Gene Therapy Breakthrough: Cell-Free Production Method Receives $5.2 Million in Funding"
"Moorfields Eye Hospital successfully used gene therapy to save the sight of four children with a rare blinding genetic condition; this success highlights the potential of Fuse Vectors, a Danish biotech startup, which just secured $5.2 million in funding to revolutionize gene therapy manufacturing t...
Progress
52% Bias Score
MSH3 Protein Identified as Driver of Huntington's Disease CAG Repeat Expansion
Research reveals that the DNA mismatch repair protein MSH3 drives CAG repeat expansion in Huntington's disease, offering hope for new treatments targeting the root cause of this devastating condition and potentially others with similar mechanisms.
MSH3 Protein Identified as Driver of Huntington's Disease CAG Repeat Expansion
Research reveals that the DNA mismatch repair protein MSH3 drives CAG repeat expansion in Huntington's disease, offering hope for new treatments targeting the root cause of this devastating condition and potentially others with similar mechanisms.
Progress
44% Bias Score
Spinal Cord Stimulation Improves Muscle Function in SMA Patients
A small study shows spinal cord stimulation temporarily improved muscle function in three adults with spinal muscular atrophy (SMA), offering hope for a new treatment for this progressive, incurable disease.
Spinal Cord Stimulation Improves Muscle Function in SMA Patients
A small study shows spinal cord stimulation temporarily improved muscle function in three adults with spinal muscular atrophy (SMA), offering hope for a new treatment for this progressive, incurable disease.
Progress
32% Bias Score
mvGPT: A Revolutionary Gene Editing Technology
Rice University researchers developed mvGPT, a compact gene editing technology enabling simultaneous gene editing, activation, and repression; successful in treating Wilson's disease, Type I diabetes, and Transthyretin amyloidosis.
mvGPT: A Revolutionary Gene Editing Technology
Rice University researchers developed mvGPT, a compact gene editing technology enabling simultaneous gene editing, activation, and repression; successful in treating Wilson's disease, Type I diabetes, and Transthyretin amyloidosis.
Progress
48% Bias Score
Showing 25 to 36 of 43 results