Northern Minnesota boy is first American to receive groundbreaking treatment for muscular dystrophyColton Belluzzo received groundbreaking gene therapy for Duchenne muscular dystrophy, showing promise in slowing disease progression.
Patient dies following muscular dystrophy gene therapy, Sarepta reportsA patient died from acute liver injury while using Sarepta's Elevidys gene therapy for muscular dystrophy, causing a major stock market reaction.
Young Patient Dies After Receiving Pfizer Gene TherapyThe tragic death of a young boy during a Pfizer medical trial for Duchenne muscular dystrophy has raised concerns and questions among scientists and parents.
Minneapolis parents share journey of having two sons with rare muscle-wasting disorderThe Kasner family faces Duchenne muscular dystrophy with resilience and a strong support network, embracing love and life despite the challenges.
Northern Minnesota boy is first American to receive groundbreaking treatment for muscular dystrophyColton Belluzzo received groundbreaking gene therapy for Duchenne muscular dystrophy, showing promise in slowing disease progression.
Patient dies following muscular dystrophy gene therapy, Sarepta reportsA patient died from acute liver injury while using Sarepta's Elevidys gene therapy for muscular dystrophy, causing a major stock market reaction.
Young Patient Dies After Receiving Pfizer Gene TherapyThe tragic death of a young boy during a Pfizer medical trial for Duchenne muscular dystrophy has raised concerns and questions among scientists and parents.
Minneapolis parents share journey of having two sons with rare muscle-wasting disorderThe Kasner family faces Duchenne muscular dystrophy with resilience and a strong support network, embracing love and life despite the challenges.
Uncovering New Therapeutic Targets for Pediatric Epilepsy - News CenterResearchers have identified new mechanisms in Dravet syndrome that highlight novel therapeutic targets, particularly in intronic regions of the SCN1A gene.
Long Island patient gets breakthrough sickle cell treatment called a 'cure': 'I'm not in pain anymore'Lyfgenia gene therapy successfully cured sickle cell disease in a New Yorker, showcasing a major medical breakthrough.
Sickle cell gene therapies roll out slowlyFDA has approved the first genetic treatments for sickle cell disease, offering new hope to patients like Olaide Adekanbi.
First Sickle Cell Gene Therapy Patient, 12, Leaves HospitalKendric Cromer received the first gene therapy for sickle cell disease, representing a significant breakthrough in treatment.
Groundbreaking' potential cure for sickle cell in England approved for NHS useNHS approved a groundbreaking gene therapy for sickle cell disease, potentially curing eligible patients in England.
First Day of a New Life' for a Boy With Sickle CellKendric Cromer starts a hopeful journey free from sickle cell disease thanks to new gene therapy.
Gene Therapies Could Transform Treatment of Rare Blood Disorders - News CenterGene therapy shows promising results in treating rare genetic blood disorders like beta thalassemia and sickle cell disease.
Long Island patient gets breakthrough sickle cell treatment called a 'cure': 'I'm not in pain anymore'Lyfgenia gene therapy successfully cured sickle cell disease in a New Yorker, showcasing a major medical breakthrough.
Sickle cell gene therapies roll out slowlyFDA has approved the first genetic treatments for sickle cell disease, offering new hope to patients like Olaide Adekanbi.
First Sickle Cell Gene Therapy Patient, 12, Leaves HospitalKendric Cromer received the first gene therapy for sickle cell disease, representing a significant breakthrough in treatment.
Groundbreaking' potential cure for sickle cell in England approved for NHS useNHS approved a groundbreaking gene therapy for sickle cell disease, potentially curing eligible patients in England.
First Day of a New Life' for a Boy With Sickle CellKendric Cromer starts a hopeful journey free from sickle cell disease thanks to new gene therapy.
Gene Therapies Could Transform Treatment of Rare Blood Disorders - News CenterGene therapy shows promising results in treating rare genetic blood disorders like beta thalassemia and sickle cell disease.
Gene therapy experiment gives children 'life-changing' sight boostGene therapy has significantly improved the vision of toddlers born with severe childhood blindness, providing life-changing outcomes.
Doctors in London cure blindness in children with rare conditionDoctors in London have pioneered gene therapy to cure childhood blindness caused by a genetic condition, marking a significant advancement in medical science.
Mutated DNA Restored to Normal in Gene Therapy AdvanceA single infusion successfully corrected a mutated gene, marking a significant advancement in gene therapy.This precision targeting could revolutionize treatment for various genetic diseases.
From brain maps to living bridges: The innovations set to reshape life in 20252025 is poised to be a transformative year for neuroscience with breakthroughs in brain mapping and AI-led therapies.
NHS to offer life-changing' gene therapy for blood disorder thalassaemiaCasgevy gene therapy approved for severe thalassaemia on NHS in England, potentially offering a lifetime cure with significant benefits for patients.
Gene therapy experiment gives children 'life-changing' sight boostGene therapy has significantly improved the vision of toddlers born with severe childhood blindness, providing life-changing outcomes.
Doctors in London cure blindness in children with rare conditionDoctors in London have pioneered gene therapy to cure childhood blindness caused by a genetic condition, marking a significant advancement in medical science.
Mutated DNA Restored to Normal in Gene Therapy AdvanceA single infusion successfully corrected a mutated gene, marking a significant advancement in gene therapy.This precision targeting could revolutionize treatment for various genetic diseases.
From brain maps to living bridges: The innovations set to reshape life in 20252025 is poised to be a transformative year for neuroscience with breakthroughs in brain mapping and AI-led therapies.
NHS to offer life-changing' gene therapy for blood disorder thalassaemiaCasgevy gene therapy approved for severe thalassaemia on NHS in England, potentially offering a lifetime cure with significant benefits for patients.
Inside the secret island where wealthy people go to alter their DNARoatan is attracting ultra-wealthy individuals seeking experimental gene therapies due to its lack of regulations around such treatments.
Why women's brains are more resilient: it could be their 'silent' X chromosomeThe second X chromosome in females may guard against cognitive decline and aging.
The search for solutions to geographic atrophy of the retinaAge-related macular degeneration (AMD) leads to progressive vision loss, significantly impacting independence, but recent drug approvals offer hope for treatment.
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 blindnessCRISPR-based gene therapy improved vision in individuals with inherited blindness.
The search for solutions to geographic atrophy of the retinaAge-related macular degeneration (AMD) leads to progressive vision loss, significantly impacting independence, but recent drug approvals offer hope for treatment.
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 blindnessCRISPR-based gene therapy improved vision in individuals with inherited blindness.
Cash-Strapped Gene Therapy Firm Bluebird Bio Agrees to Acquisition by Private Equity - MedCity NewsBluebird Bio has agreed to sell itself to private equity firms Carlyle and SK Capital for $29 million to stabilize its gene therapy commercialization efforts.
In other news this week: Knave secures 225M, London-based Purespring raises 95M, VERBUND X Ventures invests in Ausrtia's Easelink, and more - Silicon CanalsTech 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 manufacturingAutomated cell therapy manufacturing with less stress and contamination through modular device.
Amsterdam-based biotech company uniQure sets this share price for 73M public offering: Know more - Silicon CanalsuniQure plans to raise $75M through a public offering amid promising progress in gene therapies for Huntington's disease.
Cash-Strapped Gene Therapy Firm Bluebird Bio Agrees to Acquisition by Private Equity - MedCity NewsBluebird Bio has agreed to sell itself to private equity firms Carlyle and SK Capital for $29 million to stabilize its gene therapy commercialization efforts.
In other news this week: Knave secures 225M, London-based Purespring raises 95M, VERBUND X Ventures invests in Ausrtia's Easelink, and more - Silicon CanalsTech 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 manufacturingAutomated cell therapy manufacturing with less stress and contamination through modular device.
Amsterdam-based biotech company uniQure sets this share price for 73M public offering: Know more - Silicon CanalsuniQure plans to raise $75M through a public offering amid promising progress in gene therapies for Huntington's disease.
Gene therapy experiment gives children 'life-changing' sight boost at London hospitalPioneering gene therapy restores sight in young children with a rare genetic condition.
The second revolution in gene therapy that has cured Javier, a child with a lethal error in the X chromosomeA small sign led to diagnosis of Wiskott-Aldrich syndrome, which was overcome through groundbreaking gene therapy.
Gene therapy experiment gives children 'life-changing' sight boost at London hospitalPioneering gene therapy restores sight in young children with a rare genetic condition.
The second revolution in gene therapy that has cured Javier, a child with a lethal error in the X chromosomeA small sign led to diagnosis of Wiskott-Aldrich syndrome, which was overcome through groundbreaking gene therapy.
The immune system can sabotage gene therapies - can scientists rein it in?Gene therapy advancements face challenges in re-dosing after initial treatment.
Growing success in new gene therapy for inherited deafness- Harvard GazetteSuccessful gene therapy trial for genetic deafness promising for future availability.
Promising treatment for recurrent respiratory papillomatosisPRGN-2012 shows promise as a gene therapy for recurrent respiratory papillomatosis, potentially reducing the need for multiple surgeries.
The immune system can sabotage gene therapies - can scientists rein it in?Gene therapy advancements face challenges in re-dosing after initial treatment.
Growing success in new gene therapy for inherited deafness- Harvard GazetteSuccessful gene therapy trial for genetic deafness promising for future availability.
Promising treatment for recurrent respiratory papillomatosisPRGN-2012 shows promise as a gene therapy for recurrent respiratory papillomatosis, potentially reducing the need for multiple surgeries.
Publisher Correction: Placenta-tropic VEGF mRNA lipid nanoparticles ameliorate murine pre-eclampsiaThe corrections clarify important data inaccuracies in a study on lipid nanoparticles for murine pre-eclampsia.
The Most Important Breakthroughs of 20242023 highlights include breakthroughs in gene therapy for sickle-cell anemia and a new HIV prevention drug that shows remarkable efficacy.
Correcting Genetic Spelling Errors With Next-Generation CrisprSam Berns' life epitomizes the urgent need for advancements in genetic treatments, particularly for disorders like progeria that code for rapid aging.
The Most Important Breakthroughs of 20242023 highlights include breakthroughs in gene therapy for sickle-cell anemia and a new HIV prevention drug that shows remarkable efficacy.
Correcting Genetic Spelling Errors With Next-Generation CrisprSam Berns' life epitomizes the urgent need for advancements in genetic treatments, particularly for disorders like progeria that code for rapid aging.
Strimvelis, the gene therapy rejected by the pharmaceutical industry that has saved AitanaGene therapy saved Aitana's life after she was diagnosed with a severe immune deficiency.The Strimvelis treatment faced discontinuation, emphasizing the challenge of sustaining innovative medical therapies.
More children gain hearing as gene therapy for profound deafness advancesGene therapy is offering children with genetic deafness the ability to hear normally without the need for hearing aids or cochlear implants.
Strimvelis, the gene therapy rejected by the pharmaceutical industry that has saved AitanaGene therapy saved Aitana's life after she was diagnosed with a severe immune deficiency.The Strimvelis treatment faced discontinuation, emphasizing the challenge of sustaining innovative medical therapies.
More children gain hearing as gene therapy for profound deafness advancesGene therapy is offering children with genetic deafness the ability to hear normally without the need for hearing aids or cochlear implants.
Scientists grow human brains in space to treat Alzheimer'sMicrogravity facilitates rapid growth of brain organoids, enabling critical research into treatments for Alzheimer's, Parkinson's, and spinal injuries.
Cures for rare diseases now exist. Employers don't want to payThe rise of multimillion-dollar gene therapies is pushing employers to drop coverage, leaving families in desperate situations.
Goldman Sachs Recommends These 3 Growth Stocks As Strong Buys in SeptemberGoldman 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 cutsBioMarin 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 TherapiesAAVs 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 ParasiteToxoplasma engineered to deliver proteins may offer treatment for brain disorders like Rett syndrome and neurodegenerative diseases.
Argentinian couple moves to US to allow their toddler to join gene therapy trialParents refused to give up despite rare disease diagnosis, traveled for gene therapy trial, showing hope and perseverance.
Gene therapy trial restores hearing in both ears for deaf childrenGene therapy trial restores hearing in deaf children, showing promise for future treatments.
Deaf baby hears for the first time after 'groundbreaking' gene therapy trialOpal Sandy, born deaf due to a genetic condition, can now hear unaided after groundbreaking gene therapy.
Great Ormond Street hoping to license gene therapy for bubble baby' syndromeGene 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.
UK toddler has hearing restored in world first gene therapy trialSignificant milestone in gene therapy for treating deafness.
Gene therapy trial restores hearing in both ears for deaf childrenGene therapy trial restores hearing in deaf children, showing promise for future treatments.
Deaf baby hears for the first time after 'groundbreaking' gene therapy trialOpal Sandy, born deaf due to a genetic condition, can now hear unaided after groundbreaking gene therapy.
Great Ormond Street hoping to license gene therapy for bubble baby' syndromeGene 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.
UK toddler has hearing restored in world first gene therapy trialSignificant milestone in gene therapy for treating deafness.
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.
Gene therapy trials treat inherited blindness and deafness DW 05/09/2024The article highlights the groundbreaking achievement of restoring hearing and vision through gene therapy in children with rare genetic mutations.
Daily briefing: 'Open-washing' plagues big AIOpen-source AI not as transparent as claimedLow-frequency vibrations trigger mouse sexual behaviorsImportance of research putting individuals at the heart of schizophrenia study