Tagged News
Ionis Reports Positive Phase 3 Results for Olezarsen in Familial Chylomicronemia Syndrome
- Ionis Pharmaceuticals' olezarsen met primary and key secondary endpoints in Phase 3 BALANCE trial for familial chylomicronemia syndrome (FCS), demonstrating significant triglyceride reduction.
- The antisense therapy showed a favorable safety profile with no major adverse events reported, potentially offering a new treatment option for this rare genetic disorder.
- Ionis plans to submit regulatory applications in the coming months, positioning olezarsen to potentially become the first approved therapy specifically targeting FCS.
First-of-its-kind Gene Editing Treatment Saves Baby with Rare Genetic Disorder
- Doctors at Children's Hospital of Philadelphia successfully treated a baby with CPS1 deficiency using a custom-designed CRISPR base editing therapy, marking a groundbreaking advancement in personalized genetic medicine.
- The experimental treatment corrected a specific genetic mutation in KJ Muldoon's DNA, allowing his liver to properly process ammonia and preventing toxic buildup that threatens brain development and survival.
- Researchers believe this pioneering approach could transform treatment for millions with rare genetic disorders, demonstrating that personalized gene therapies can be developed relatively quickly and at costs comparable to conventional treatments.
GRI Bio Advances Phase 2a Trial of GRI-0621 for Idiopathic Pulmonary Fibrosis with Promising Interim Safety Data
- GRI Bio has completed enrollment for the interim analysis of its Phase 2a trial evaluating GRI-0621 in idiopathic pulmonary fibrosis patients, with 24 of 36 planned patients randomized.
- Early safety data from the first 12 patients shows GRI-0621 is well-tolerated with no significant changes in lipid profiles, consistent with previous studies of related compounds.
- The company has secured $13.9 million in funding since early 2024, extending its cash runway through the interim data readout expected in Q1 2025, with topline results anticipated in Q2 2025.
Highlighted Clinical Trials:
GRI Bio Operations, Inc.
Posted 4/24/2024
Roche's PiaSky Approved in EU as First Monthly Self-Administered Treatment for Paroxysmal Nocturnal Haemoglobinuria
- The European Commission has approved PiaSky (crovalimab), the first monthly subcutaneous treatment for paroxysmal nocturnal haemoglobinuria (PNH), offering patients the option to self-administer following training.
- PiaSky utilizes innovative recycling antibody technology developed by Chugai Pharmaceutical, allowing it to bind to C5 protein multiple times and remain effective longer with a small volume of medicine.
- Clinical trials demonstrated PiaSky's non-inferiority to eculizumab, the current standard of care, while potentially reducing treatment burden through less frequent administration and eliminating the need for regular clinic visits.
Highlighted Clinical Trials:
Hoffmann-La Roche
Posted 10/8/2020
Hoffmann-La Roche
Posted 11/17/2021
Hoffmann-La Roche
Posted 10/22/2021
Hoffmann-La Roche
Posted 9/30/2020
FDA Grants Orphan Drug and Rare Pediatric Disease Designations to ATSN-201 for X-Linked Retinoschisis
- Atsena Therapeutics' gene therapy ATSN-201 has received both Orphan Drug and Rare Pediatric Disease designations from the FDA for X-linked retinoschisis, a rare genetic disorder causing vision loss primarily in males.
- ATSN-201 utilizes a novel spreading capsid (AAV.SPR) to deliver functional copies of the RS1 gene to retinal photoreceptors while minimizing risks of foveal detachment, potentially addressing the root cause of the disease.
- Preliminary data from the Phase 1/2 LIGHTHOUSE trial has shown promising results, with two out of three patients experiencing extensive schisis resolution beginning at 8 weeks post-treatment.
NHS England Approves First CRISPR Gene-Editing Therapy for Beta Thalassaemia Treatment
- NHS England has approved Casgevy, the first CRISPR gene-editing therapy, for treating severe beta thalassaemia in patients aged 12 and older, making it among the first healthcare systems globally to offer this treatment.
- Clinical trials demonstrated that 93% of beta thalassaemia patients did not require blood transfusions for at least a year after receiving the one-time gene therapy, offering potential lifetime cure for a condition that typically requires transfusions every 3-5 weeks.
- The therapy will be available at seven specialist NHS centres within weeks, with an estimated 460 patients in England potentially eligible for the treatment that uses Nobel Prize-winning CRISPR technology to edit bone marrow stem cells.
Gene Therapy Pioneer Jim Wilson Departs UPenn to Launch Two New Companies
- Gene therapy pioneer Jim Wilson is leaving the University of Pennsylvania after 30 years to found two new companies, Gemma Biotherapeutics and Franklin Biolabs, spinning out the university's Gene Therapy Program.
- Gemma Bio will focus on rare disease research and has licensed three clinical-stage gene therapy candidates from Passage Bio for pediatric conditions including GM1 gangliosidosis and Krabbe disease.
- The move aims to accelerate gene therapy development and patient access during a biotech funding downturn, with Wilson's team having contributed to three FDA-approved AAV-based gene therapies and over 40 active programs in development.
EMA Grants Orphan Designations for Rare Disease Treatments in Pulmonary Fibrosis and B-Cell Lymphoma
- The European Medicines Agency granted orphan designation to a medicine for treating idiopathic pulmonary fibrosis on December 13, 2023, providing regulatory support for development.
- A second orphan designation was awarded on July 25, 2024, for treatment of primary large B-cell lymphoma of immune-privileged sites.
- Both designations enable developers to receive scientific and regulatory guidance from EMA while advancing toward marketing authorization applications.
- The orphan designations do not constitute approval for patient use, as all medicines must undergo full authorization before becoming available in the EU.
Riliprubart Shows Promise as First-in-Class Treatment for Chronic Inflammatory Demyelinating Polyneuropathy
- Sanofi's riliprubart demonstrated significant disease-controlling benefits across all patient cohorts in a Phase 2 study for chronic inflammatory demyelinating polyneuropathy (CIDP), including those who failed standard treatments.
- The complement C1s inhibitor showed sustained efficacy for up to 48 weeks, with 87-92% of participants experiencing improvement or disease stabilization after 24 weeks of treatment.
- Riliprubart reduced neurofilament light chain levels by 35% across all cohorts, suggesting potential reduction in nerve damage, while also improving patient-reported fatigue and quality of life outcomes.
Highlighted Clinical Trials:
Intellia's CRISPR Gene Therapy Shows Potential as Functional Cure for Hereditary Angioedema in Long-Term Study
- Intellia Therapeutics' NTLA-2002, an in vivo CRISPR-based gene editing therapy, demonstrated a 98% mean reduction in monthly hereditary angioedema attack rates across all patients, with follow-up extending beyond two years.
- Eight of ten patients remained completely attack-free following the initial 16-week observation period, with the longest attack-free duration reaching over 26 months and continuing.
- The single-dose treatment showed a favorable safety profile across all dose levels, with no serious adverse events reported, positioning NTLA-2002 as a potential functional cure for this rare genetic disease.
Highlighted Clinical Trials:
Intellia Therapeutics
Posted 12/10/2021