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I Peace and Vita Therapeutics Partner to Develop Universal iPS Cells for FSHD Treatment

Gene Editing
  • I Peace and Vita Therapeutics have announced a joint development program to create universal induced pluripotent stem cells (iPSCs) for cell transplantation therapy, with an initial focus on facioscapulohumeral muscular dystrophy (FSHD).
  • The collaboration will produce GMP-grade iPS cells modified with proprietary gene editing technology to create hypoimmune cell lines that have depleted Class I and II HLA genes to prevent transplant rejection.
  • The manufactured master cell bank will be registered in the FDA Drug Master File, with I Peace retaining rights to supply both normal and gene-edited cells for therapeutic applications globally.
  • Universal cells are designed to suppress immune rejection due to HLA incompatibility, allowing transplantation without requiring compatibility matching with the recipient's HLA type.

Broken String Biosciences and BioLizard Partner to Develop AI-Powered SafeGuide for CRISPR Gene Editing Safety

Gene EditingAI
  • Broken String Biosciences and BioLizard have announced the development of SafeGuide, an AI tool designed to select safer guide RNAs for CRISPR gene editing applications.
  • The collaboration aims to replace current trial-and-error approaches that take one to two years and result in drug costs exceeding $4 million per patient.
  • The project is supported by a $935,000 grant from the competitive Eurostars-3 program, part of the European Partnership on Innovative SMEs.
  • SafeGuide will integrate Broken String's INDUCE-seq platform data with BioLizard's AI algorithms to predict high-efficacy, low-risk guide RNA designs.

CRISPR Gene Editing Enables Mice to Produce Diabetes Drug Exenatide for Sustained Weight Control

Gene Editing
  • Japanese researchers at the University of Osaka used CRISPR gene editing to engineer mouse liver cells to produce exenatide, a diabetes and obesity medication, eliminating the need for regular injections.
  • The genome-edited mice maintained therapeutic drug levels in their blood for over six months, showing sustained weight control and improved glucose metabolism after a single treatment.
  • The study demonstrates potential for transforming chronic disease management by turning the liver into a continuous drug production factory, though only 1% of liver cells successfully incorporated the gene.
  • This approach could address the challenge of frequent dosing required for GLP-1 receptor agonists like exenatide, which typically need weekly or daily injections to maintain therapeutic levels.

CRISPR Therapeutics' CTX310 Shows Promising Lipid Reduction Results in Phase 1 Trial

Gene Editing
  • CRISPR Therapeutics' CTX310, an in vivo ANGPTL3 knockout therapy, demonstrated peak reductions of up to 82% in triglycerides and 86% in LDL cholesterol at dose level four in Phase 1 trial results.
  • The therapy maintained a favorable safety profile with no discernible alterations in liver enzyme levels, consistent with previous research findings.
  • H.C. Wainwright reaffirmed its Buy rating and $65 price target for CRISPR Therapeutics following the supplemental update to the CTX310 trial findings.
  • Study limitations include baseline cholesterol control measurements, only one patient at the highest dose level, and illness variability that continue to limit comprehensive analysis.

AvenCell Secures $40 Million AMED Grant to Advance Dual-Antigen Allogeneic CAR-T Therapy for B-Cell Lymphomas

CAR-TGene Editing
  • AvenCell Japan has been awarded up to $40 million in non-dilutive funding from Japan's AMED to support worldwide development of AVC203, a dual-antigen allogeneic CAR-T therapy targeting CD19 and CD20 for B-cell lymphomas.
  • The company's proprietary allogeneic technology uses multiple gene editing steps to prevent immune rejection while maintaining donor T-cell fitness and potency, addressing key challenges that have limited previous allogeneic CAR-T approaches.
  • Early clinical data from AvenCell's AVC201 program in relapsed and refractory AML patients demonstrate robust cell expansion and persistence beyond the typical one-month rejection period where other allogeneic candidates have failed.
  • The funding will enable AvenCell to collaborate with Japanese key opinion leaders and stakeholders to advance what the company describes as a paradigm-shifting therapeutic for patients with high unmet medical needs in lymphomas and leukemias.

Laverock Therapeutics Raises £20M in Expanded Seed Funding, Reports Positive Data for Cancer Cell Therapy Programs

Gene EditingOncology
  • Laverock Therapeutics has expanded its seed funding round to more than £20 million, with the latest £6.5 million extension led by Calculus Capital and including participation from Eli Lilly and Company.
  • The company reported positive functional data for both its primary T-cell program LKV201 and macrophage program LKV301, demonstrating enhanced tumor control in hematological and solid tumor cancer models.
  • Laverock's programmable gene control platform uses recoded miRNAs to deliver tunable gene silencing, potentially transforming the efficacy, precision and safety of advanced cell therapies.
  • The funding will support lead product selection and optimization of preclinical programs while enabling partnerships to integrate Laverock's platform technologies into other therapeutic pipelines.

Perceptive Advisors Completes $7 Billion Acquisition of CRISPR Solutions Leader Synthego

Gene Editing
  • Perceptive Advisors successfully completed its acquisition of substantially all assets of CRISPR solutions provider Synthego on July 18, 2025, strengthening the company's financial position for expansion.
  • Synthego will maintain normal operations under new ownership, continuing to manufacture guide RNA in Redwood City, California, with all leadership and staff remaining in place.
  • The acquisition provides enhanced resources for Synthego to accelerate innovation and expand its CRISPR product portfolio, including recent launches of GMP SpCas9 and new gene editing enzymes.
  • Synthego's position as a leading American manufacturer of CRISPR components supports FDA-regulated therapeutic development without tariff-induced price increases for clients.

EVA Pharma Establishes Eighth Global Office in Hangzhou's BioPharma Town to Advance Cell and Gene Therapy Innovation

CAR-TGene Editing
  • EVA Pharma signed a strategic cooperation agreement with Qiantang District's BioPharma Town in Hangzhou, establishing its eighth global office to focus on pharmaceuticals, vaccines, and biologics R&D.
  • The partnership leverages Hangzhou's position as a global leader in Cell and Gene Therapy, with specialized GMP platforms and companies focused on mRNA vaccines, CAR-T therapy, and gene editing.
  • EVA Pharma will contribute its R&D expertise and regulatory knowledge to explore joint projects in vaccine technology, mRNA applications, and therapeutic biologics while accessing local capabilities.
  • The collaboration strengthens EVA Pharma's global expansion strategy, building on its existing portfolio across twelve therapeutic areas and operations in over 70 countries worldwide.

Former Pfizer CSO Mikael Dolsten Joins Two Biotech Boards, Advancing Gene Editing and Mast Cell Therapeutics

Gene EditingRare Disease
  • Dr. Mikael Dolsten, former Pfizer Chief Scientific Officer, has joined the boards of Arbor Biotechnologies and MC Sciences as both companies advance novel therapeutic platforms.
  • Dolsten's 16-year tenure at Pfizer included advancing over 150 drug candidates into clinical studies and leading regulatory approval of 36 medicines and vaccines.
  • Arbor Biotechnologies is progressing its lead gene editing program ABO-101 for primary hyperoxaluria type 1 into clinical trials.
  • MC Sciences is developing first-in-class mast cell-targeting therapeutics for diseases including chronic urticaria, systemic mastocytosis, and asthma.

First-in-Human iPSC-CAR-NK Cell Therapy Shows Promise for Autoimmune Disease Treatment

Gene Editing
  • Qihan Biotech's QN-139b represents the first-ever application of iPSC-CAR-NK cells in treating autoimmune disease, successfully inducing immune reset in a patient with refractory systemic sclerosis.
  • The dual-targeting therapy eliminates pathogenic B cells and plasma cells by targeting both CD19 and BCMA, incorporating nine gene edits and advanced safety features including a tEGFR safety switch.
  • After six months of treatment, the patient showed significant reduction in autoantibodies, dramatic improvement in skin scores, and histological evidence of B cell clearance and fibrosis suppression.
  • The groundbreaking clinical results were published in Cell, marking a potential breakthrough for treating severe autoimmune diseases with off-the-shelf cell therapies.

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