Tagged News
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
Atsena Therapeutics Reports Promising Results for XLRS Gene Therapy Using Novel Spreading Capsid
- Atsena Therapeutics' ATSN-201 gene therapy showed positive safety and early efficacy in the first cohort of XLRS patients, with two of three patients experiencing extensive schisis resolution beginning at 8 weeks post-treatment.
- The trial demonstrated clinical validation of AAV.SPR's ability to spread laterally beyond subretinal injection sites, achieving therapeutic effects without requiring risky foveal detachment procedures.
- Functional improvements were observed using microperimetry, with one patient showing improvements up to 14 dB and 38 loci improving by more than 7 dB, exceeding the FDA's threshold for clinical significance.
Highlighted Clinical Trials:
Atsena Therapeutics Inc.
Posted 8/22/2023
Vicore's Buloxibutid Shows Promising Lung Function Improvement in Phase 2a IPF Trial
- Vicore Pharma's buloxibutid demonstrated significant lung function improvement in IPF patients, with an average FVC increase of 216ml over 36 weeks compared to the typical 180ml decline in untreated patients.
- The angiotensin II type 2 receptor agonist was well-tolerated with no new drug-related adverse events reported during the 26-week treatment period, suggesting a favorable safety profile.
- Final data from the Phase 2a AIR trial will be presented as a late-breaking presentation at the 2024 American Thoracic Society International Conference, with Vicore planning to advance to a Phase 2b ASPIRE trial.
Global Phase 3 Trial Tests Efzofitimod as Steroid-Sparing Treatment for Sarcoidosis
- Royal Papworth Hospital is participating in the world's first advanced phase global trial for sarcoidosis, testing if the protein efzofitimod can reduce steroid dependence in patients.
- Sarcoidosis affects approximately 7,000 people in the UK, typically younger adults in their 30s and 40s, causing granulomas to form in the lungs and potentially other organs.
- The trial involves about 200 patients across Europe, North America, and Asia, aiming to improve quality of life by reducing long-term steroid side effects like weight gain, diabetes risk, and bone weakness.
Novartis Secures $1.3 Billion Deal with Chong Kun Dang for HDAC6 Inhibitor Targeting CMT Disease
- Novartis has signed a $1.3 billion licensing agreement with Korean biotech Chong Kun Dang Pharmaceutical for CKD-510, an HDAC6 inhibitor with FDA orphan drug designation for Charcot-Marie-Tooth disease.
- The deal includes an $80 million upfront payment plus $1.2 billion in potential milestone payments, with Novartis gaining exclusive global rights to develop and commercialize the drug outside Korea.
- This acquisition follows Novartis' recent procurement of DTX-1252, another CMT1A therapeutic candidate, signaling the company's strategic expansion into rare neurological disease treatments.
UK Approves World's First CRISPR Gene Therapy for Sickle Cell Disease and β-Thalassemia
- The UK has made a landmark decision to approve the world's first CRISPR-based gene therapy, developed by Vertex Pharmaceuticals, for treating sickle cell disease and β-thalassemia.
- This one-time treatment offers potential transformation of patient lives by addressing the genetic root cause of these blood disorders, marking a historic milestone in gene editing technology.
- Despite its therapeutic promise, concerns remain about the high cost of the treatment and questions about accessibility for patients in need, particularly in regions with high disease prevalence.
Chinese Base Editing Therapy Achieves First Clinical Cure of Beta Thalassaemia
- Shanghai-based CorrectSequence Therapeutics has successfully treated a transfusion-dependent beta thalassaemia patient using their novel base editing therapy CS-101, marking a world first for this technology.
- The adolescent patient, who previously required blood transfusions every two weeks, has maintained normal hemoglobin levels above 130 g/L for over two months without transfusions or adverse effects.
- The transformer Base Editing (tBE) technology offers more efficient hematopoietic reconstruction with fewer safety risks compared to other CRISPR-based gene-editing therapies for blood disorders.