Weekly Highlights | Gene Therapy News Digest Issue 115

Aug 12, 2024 17:56 CST Updated 17:56

Lingyi Biotech

Gene Therapy Drug Developer

Industry Dynamics Overview

Focus on Hotspots

First Successful Gene Therapy for Adult Severe Thalassemia Patient in Guangzhou

In 2024, the Department of Hematology at Guangzhou First People's Hospital launched a clinical study on gene therapy for β-thalassemia (hereinafter referred to as "β-thalassemia"), providing gene therapy for the first adult patient with severe β-thalassemia in Guangzhou. Jiajia has since been freed from blood transfusion dependence. Currently, gene therapy is gradually becoming a new approach internationally for the "one-time treatment, permanent cure" of β-thalassemia.

Lingyi Biotech's AAV Gene Drug LY-M003 Granted FDA Orphan Drug and Pediatric Rare Disease Designation

On August 7, 2024, Lingyi Biotech Co., Ltd. (hereinafter referred to as "Lingyi Biotech") announced that its self-developed gene therapy drug LY-M003 injection received Orphan-drug Designation (ODD) from the U.S. FDA for the treatment of Wilson’s disease (WD).

DMD Therapy Significantly Improves Disease Indicators, Will Discuss Accelerated Approval Pathway with FDA

Recently, Avidity Biosciences announced positive data from the Phase 1/2 clinical trial EXPLORE44 of its investigational antibody-conjugated oligonucleotide therapy delpacibart zotadirsen (del-zota) in patients with Duchenne muscular dystrophy (DMD44) suitable for exon 44 skipping therapy. Del-zota significantly increased the production of dystrophin in patients, reaching up to 54% of normal levels. Additionally, del-zota reduced creatine kinase levels to near-normal, decreasing by over 80% compared to baseline. Avidity stated that it plans to engage in discussions with the U.S. FDA to explore the best pathway for seeking accelerated approval of this therapy.

Ruijin Hospital Successfully Completes the First Patient Dosing in Shanghai for the AAV Gene Therapy Clinical Study Targeting Parkinson's Disease

A Study on the Safety, Tolerance, and Efficacy of BBM-P002 Injection in Stereotactic Bilateral Putamen Treatment for Moderate to Advanced Primary Parkinson's Disease, Initiated by Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine (hereinafter referred to as "Ruijin Hospital"), Recently Completed the First Patient Dosing in Shanghai.

Innovation Breakthrough

Nature Sub-Journal: New Discovery in Alzheimer's Disease – Not Only Neurons, but Glial Cells Also Produce Aβ Toxic Protein and Promote Plaque Formation

On August 5, 2024, researchers from the Max Planck Institute for Multidisciplinary Sciences published a research paper titled: Oligodendrocytes produce amyloid-β and contribute to plaque formation alongside neurons in Alzheimer’s disease model mice in the Nature Neuroscience, a sub-journal of Nature.

This study shows that, in addition to neurons, a special type of glial cell in the brain—oligodendrocytes—also produces β-amyloid (Aβ) and works with neurons to promote plaque formation. This discovery may open new avenues for future Alzheimer's disease treatment.

Nature: Liver "Immune Rheostat" Suppresses CD8+ T Cell Immunity in Chronic Hepatitis B Virus Infection

Recently, researchers from the Technical University of Munich, Germany, published a research paper titled "A liver immune rheostat regulates CD8 T cell immunity in chronic HBV infection" in the international top academic journal Nature.

This study demonstrates that liver sinusoidal endothelial cells act as the liver's "immune rheostat," making it difficult for HBV-specific CD8+ T cells to be activated and leading to the loss of their effector functions. Modulating the liver's "immune rheostat" function by targeting the inhibitory adenylate cyclase-cAMP-PKA signaling axis holds promise for enhancing the effectiveness of treatments aimed at restoring HBV-specific CD8+ T cell responses in chronic HBV infection.

Mol Cancer | Jiang Xian/Zhang Zhuo from Southwest Medical University Reveal miRNA Regulatory Pathways Highly Expressed in Hepatocellular Carcinoma

On August 6, 2024, Xian Jiang and Zhuo Zhang from Southwest Medical University jointly published a research article titled "Circular RNA ACVR2A promotes the progression of hepatocellular carcinoma through mir-511-5p targeting PI3K-Akt signaling pathway" in Molecular Cancer (IF=28). The study found that circACVR2A can directly interact with miR-511-5p, acting as a miRNA sponge to regulate the expression of related proteins in the PI3K-Akt signaling pathway.

CDD | Wei Fang/Xu Xun et al. from Shanghai Jiao Tong University collaborate to discover a potential new therapeutic target for diabetic retinopathy

On July 30, 2024, Fang Wei, Xun Xu, and Huiming Li from Shanghai Jiao Tong University jointly communicated in an article titled "TIN2 modulates FOXO1 mitochondrial shuttling to enhance oxidative stress-induced apoptosis in retinal pigment epithelium under hyperglycemia," published online in Cell Death & Differentiation. The study observed a significant increase in oxidative stress-induced RPE cell apoptosis both in vivo and in vitro, along with elevated expression of the telomere protein TIN2. Further research confirmed that the increase in mitochondrially localized TIN2 induced by hyperglycemia impairs mitochondrial activity, weakens intrinsic antioxidant defenses, thereby leading to the activation of the mitochondria-dependent apoptotic pathway.

Capital Express

Forward Bioscience Completes Over 100 Million Yuan in A+ Round Financing to Develop Novel Gene Editing Therapies

Recently, CorrectSequence Bio announced the completion of an over 100-million-yuan Series A+ financing round. This round was led by Shanghai Guotou Futeng Capital, with follow-on investments from existing shareholders including Lianxin Capital, Boyu Capital, Lilly Asia Ventures, Vici Capital, and Sequoia China. According to a press release from CorrectSequence Bio, this funding will further accelerate the clinical research and commercialization of CS-101, support the clinical translation of its in vivo therapeutic pipeline, and advance its global strategy and the early market launch of its gene-editing products.

Nearly $2 Billion! Roche Invests in Alzheimer's Gene Therapy

On August 6, Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a company focused on gene therapy drugs, announced that it had reached a licensing agreement with Genentech, a member of the Roche Group, to develop intravenously administered genomic medicines for treating neurodegenerative diseases, including Alzheimer's disease.

$1.95 Billion! Roche Collaborates with Sangamo to Develop Genomic Medicines

On August 6, Sangamo Therapeutics announced that it had reached a licensing agreement with Genentech, a subsidiary of Roche, to collaborate on the development of intravenously administered genomic medicines for the treatment of certain neurodegenerative diseases.

References:

About Paigen Bio