Weekly Highlights | Gene Therapy News Digest Issue 123

Oct 21, 2024 17:47 CST Updated 17:47

Lingyi Biotech

Gene Therapy Drug Developer

MeiraGTx

Developer of Innovative Gene Therapy Products

Industry Dynamics Overview

Focus on Hotspots

Warm congratulations on the success of Lingyi Biotech's LY-M001 injection in treating adolescent patients with Type I Gaucher disease in the clinical study at Shanghai Children's Medical Center

Recently, a "Prospective, Single-Center, Open-Label, Single-Arm Clinical Study to Evaluate the Safety and Efficacy of a Single Intravenous Infusion of LY-M001 Injection in Adolescent Patients with Type I Gaucher Disease" has been successfully conducted and yielded therapeutic effects at the National Children's Medical Center, Shanghai Children's Medical Center affiliated with Shanghai Jiao Tong University School of Medicine.

Tianjin Releases Notice on Soliciting Opinions for "Several Measures to Support the Full-chain Innovation and Development of Biomedicine," Promoting the Research, Development, Transformation, and Clinical Application of Innovative Drugs Such as Cell and Gene Therapies and Innovative Medical Device Products

Recently, Tianjin released the "Several Opinions on the Full-chain Support for Biomedical Innovation and Development in Tianjin (Draft for Comments)" (hereinafter referred to as the "Draft for Comments") for public comments.

Beijing Releases "High-Quality Construction of Cell and Gene Therapy Industry Cluster Implementation Plan (2024-2026) (Draft for Comments)"

Recently, Beijing released the "Implementation Plan for High-Quality Construction of Cell and Gene Therapy Industry Cluster (2024-2026) (Draft for Comments)", which provides strong policy support for the high-quality development of Beijing's cell and gene therapy industry. It particularly emphasizes encouraging medical institutions to file for new cell and gene therapy medical service price programs at any time and actively serving the application of innovative cell and gene therapy drugs for inclusion in the national medical insurance drug list. The plan also aims to optimize the import approval workflow for clinically urgent cell and gene therapy products and enhance Beijing’s supply and security capacity for such urgently needed products.

Up 24%, Positive Clinical Results for Parkinson's Disease AAV Gene Therapy

On October 15, MeiraGTx announced the latest clinical data for its Parkinson's disease AAV-GAD gene therapy. The results showed that AAV-GAD met the primary endpoints of safety and tolerability at 26 weeks and demonstrated statistically significant improvements in key efficacy endpoints. Following the announcement, MeiraGTx’s stock price surged significantly, closing up 14.87%, with intraday gains reaching as high as 24%.

AAV Gene Replacement Therapy Effectively Restores Hearing in DFNB111 Deaf Mice

Recently, the team of Shu Yilai and He Yingzi from the Eye, Ear, Nose, and Throat Hospital of Fudan University published a research paper titled "Hearing restoration by gene replacement therapy for a multisite-expressed gene in a mouse model of human DFNB111 deafness" in The American Journal of Human Genetics (IF=8.7), exploring the possibility of AAV gene therapy for Mpzl2-related hearing loss.

Two China-produced mRNA drugs initiate Phase I clinical trials on the same day

Recently, WestGene Biotech initiated a Phase I clinical trial to evaluate the safety, tolerability, pharmacokinetics, immunogenicity, and preliminary antitumor activity of the EB virus mRNA vaccine WGc-043 in EB virus-positive patients with relapsed or refractory lymphoma.

Innovation Breakthrough

Chen Kai et al. used LNP to deliver Cas9 RNP, achieving highly efficient gene editing in the lungs and liver.

CRISPR-Cas9-based genome editing technology holds tremendous potential, and if safe and effective delivery methods can be developed, CRISPR therapy will offer broad treatment options for genetic diseases. Currently, viral vectors are the most commonly used delivery vehicles for in vivo CRISPR gene editing. However, viral vectors may be immunogenic, pose risks of viral genome integration, and due to the sustained expression of the gene editor, could lead to off-target DNA damage. If issues related to efficacy and toxicity can be overcome, non-viral vector alternatives may address these limitations and provide new options for the delivery of CRISPR gene editors.

Science: The World's First Atomic Editing Drug Discovery Paradigm! Directly Edit Single Atoms to Advance Drug Development

On October 3, 2024, a research team led by Professor Yoonsu Park from the Korea Advanced Institute of Science and Technology (KAIST) published a research paper titled "Photocatalytic furan-to-pyrrole conversion" in the prestigious international academic journal Science.

This study successfully developed a technology capable of easily editing and correcting oxygen atoms in furan compounds into nitrogen atoms, directly transforming them into pyrrole frameworks. This technology is expected to be widely used in the pharmaceutical field, providing a new paradigm for drug development and disease treatment.！

Science Sub-Journal: Base Editing Therapy for Rare Immune Deficiency Disorders

In a latest study published in Science Translational Medicine, a research team from the National Institute of Allergy and Infectious Diseases (NIAID) and Massachusetts General Hospital optimized the SpRY enzyme using adenine base editor ABE8e and applied it to HSPCs extracted from patients with X-CGD. As a result, the modified ABE8e-SpRY could access a range of targets in HSPCs and correct the CYBB mutation responsible for X-CGD.

Advances and Opportunities and Challenges in mRNA-Based CAR Immunocyte Therapy

Genetically engineered immune cells expressing chimeric antigen receptors (CARs) have become a hotspot in cancer immunotherapy. The clinical efficacy of CAR-T cell therapy has been validated in numerous practices. As a new class of therapeutic approach, mRNA-based treatments hold tremendous potential beyond mRNA vaccines. When these two fields intersect, a technology using mRNA to encode CAR-engineered immune cells emerges, representing a new frontier in the treatment of cancer and other diseases.

Capital Express

Two Chinese CGT Companies Complete Over 100 Million Yuan in Financing Each

With continuous breakthroughs in the research, development, and clinical progress of the cell and gene therapy (CGT) field, cell and gene therapies remain one of the hottest investment areas today. On October 21, both Xuanjing Bio and Shize Bio officially announced the completion of financing exceeding 100 million yuan.

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