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#crispr

The Most Important Breakthroughs of 2024

2023 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 Crispr

Sam Berns' life epitomizes the urgent need for advancements in genetic treatments, particularly for disorders like progeria that code for rapid aging.

CRISPR therapy restores some vision to people with blindness

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

The Most Important Breakthroughs of 2024

2023 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 Crispr

Sam Berns' life epitomizes the urgent need for advancements in genetic treatments, particularly for disorders like progeria that code for rapid aging.

CRISPR therapy restores some vision to people with blindness

CRISPR-based gene therapy improved vision in individuals with inherited blindness.
morecrispr
#medical-innovation

From brain maps to living bridges: The innovations set to reshape life in 2025

2025 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 thalassaemia

Casgevy gene therapy approved for severe thalassaemia on NHS in England, potentially offering a lifetime cure with significant benefits for patients.

From brain maps to living bridges: The innovations set to reshape life in 2025

2025 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 thalassaemia

Casgevy gene therapy approved for severe thalassaemia on NHS in England, potentially offering a lifetime cure with significant benefits for patients.
moremedical-innovation
#pediatric-medicine

Strimvelis, the gene therapy rejected by the pharmaceutical industry that has saved Aitana

Gene 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 advances

Gene 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 Aitana

Gene 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 advances

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

Scientists grow human brains in space to treat Alzheimer's

Microgravity facilitates rapid growth of brain organoids, enabling critical research into treatments for Alzheimer's, Parkinson's, and spinal injuries.
#sickle-cell-disease

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 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.

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.

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.

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.

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.

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 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.

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.

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.

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.

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

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.

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.
#medical-research

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 Gazette

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

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 Gazette

Successful gene therapy trial for genetic deafness promising for future availability.
moremedical-research
#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

Gene therapy trial restores hearing in both ears for deaf children

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

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.

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.

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.

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.

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.

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.
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.

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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