#gene-therapy

#gene-therapy

Tech innovations in October highlighted significant funding rounds for companies like Knave, Purespring, and Basecamp Research.

Automated cell therapy manufacturing with less stress and contamination through modular device.

uniQure plans to raise $75M through a public offering amid promising progress in gene therapies for Huntington's disease.

2023 highlights include breakthroughs in gene therapy for sickle-cell anemia and a new HIV prevention drug that shows remarkable efficacy.

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

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

2025 is poised to be a transformative year for neuroscience with breakthroughs in brain mapping and AI-led therapies.

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

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.

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

Kendric Cromer received the first gene therapy for sickle cell disease, representing a significant breakthrough in treatment.

Kendric Cromer starts a hopeful journey free from sickle cell disease thanks to new gene therapy.

Gene therapy shows promising results in treating rare genetic blood disorders like beta thalassemia and sickle cell disease.

Gene therapy at Children's Hospital of Philadelphia offers new hope for sickle cell disease, potentially being a life-changing treatment.

Commercially approved gene therapy offers hope to sickle cell patients like Kendric Cromer, potentially leading to a cure.

The rise of multimillion-dollar gene therapies is pushing employers to drop coverage, leaving families in desperate situations.

Gene therapy approved for rare disease dramatically improves quality of life for children.

High price of the therapy raises concerns about affordability and access.

Gene therapy advancements face challenges in re-dosing after initial treatment.

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

Parents refused to give up despite rare disease diagnosis, traveled for gene therapy trial, showing hope and perseverance.

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.

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

Opal Sandy, born deaf due to a genetic condition, can now hear unaided after groundbreaking gene therapy.

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.

Significant milestone in gene therapy for treating deafness.

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.