Editor | Med-Fine CapitalOn October 6, just before the annual European Society of Gene and Cell Therapy (ESGCT) conference and the biennial Spanish Society of Gene and Cell Therapy meeting, two scientific founders of Cogo Bio—Professor Jose-Carlos Segovia (Professor at CIEMAT-CIBERER Institute/FIIS-FJD, Madrid, Spain) and Professor Matthew Porteus (Professor at Stanford University, USA, and current President of ASGCT)—jointly organized a one-day symposium titled "Homology-Directed Repair (HDR): The Path Forward." The event attracted support and participation from scholars across multiple countries, including David Rawlings, University of Washington, Seattle, USA; Paula Cannon, University of Southern California, Los Angeles, USA; Ayal Hendel, Bar-Ilan University, Ramat-Gan, Israel; Justin Eyquem, University of California San Francisco, San Francisco, USA; Alessia Cavazza, University College London, London, UK; and Rasmus.Bak, Aarhus University, Aarhus, Denmark and other renowned scholars attended the conference. This seminar was held at the ESGCT venue in Seville, Spain, and was supported by BE pharma, Synthego,AstraZeneca, with sponsorship support from companies such as Touchlight.Homology-directed repair (HDR) technology is currently one of the most widely used methods for editing the human genome. This seminar focuses on the optimization and clinical application of HDR technology, laying the foundation for an in-depth discussion on how to advance and optimize HDR technology across all nuclease platforms. It also builds an important platform for academic exchange and technical cooperation in the HDR field, further promoting the development of this technology in clinical transformation and providing more possibilities for gene-editing treatments of related diseases. The latest clinical progress has proven that Synthego Corp has reached the global forefront in the clinical application of precise HDR-based gene insertion technology.Professor Matthew Porteus Shared at the Meeting
Professor Matthew Porteus shares research progress
At the conference, Professor Segovia, representing Synthego, announced the latest clinical data from the world's first patient in the DNGT001 clinical trial for pyruvate kinase deficiency, using the company’s self-developed full gene replacement technology platform. After a year-long follow-up, positive safety data has been obtained. To date, there have been three patients globally who have received HDR gene therapy (for different indications), with Synthego being the first to conduct a clinical trial for pyruvate kinase deficiency, establishing its position as a pioneer in the field with compelling data.Jose-Carlos SegoviaProfessor Shares Latest Advances and Clinical Data
Professor Segovia shares research progress and latest clinical data
The gene replacement technology platform of Synthego Corp can, based on knocking out pathogenic genes, utilize the principle of homologous recombination to simultaneously achieve precise in-situ Knock-in insertion of normal genes to restore and enhance gene function. This "two birds with one stone" technology has unique advantages in many disease areas, breaking through the limitations of conventional therapies' singularity, and can simultaneously...Achieving the elimination of lesions and restoration enhancement provides a better option for many diseases that are difficult to cure or have poor treatment outcomes with conventional therapies. Its application scope covers genetic disorders, cancer, cognitive impairment, and even anti-aging, representing the ideal goal of gene therapy and indicating the development direction of gene therapy.MedPower Health Fund led the angel round of financing for Synthego in 2022.Pyruvate Kinase Deficiency (PKD):
Pyruvate kinase deficiency is a rareAutosomal Recessive Genetic Disorder. It can lead to chronic hemolytic anemia in patients, which is the accelerated destruction of red blood cells. Mutations in the PKLR gene result in energy deficiency in red blood cells, mainly characterized by reduced PK enzyme activity, decreased ATP production, and the accumulation of upstream metabolites, causing energy deficiency in red blood cells, accelerating their death, leading to hemolytic anemia, and even death.。
Wuxi CoGent Biotechnology Co., Ltd. (Abbreviation: CoGent Biotech)Established in January 2022, the parent company is located in Wuxi, Jiangsu, with research and development centers established in Spain, Hong Kong, and Shanghai. The team members all come from world-renowned pharmaceutical companies such as Novartis, Roche, and WuXi AppTec, and include Nobel Prize team members and two national talents leading the team.
The company focuses on cell and gene therapy. Its independently developed complete gene replacement technology platform can accurately insert or replace genes, curing diseases at the genetic level. This "two birds with one stone" technology has unique advantages in many disease areas, breaking through the limitations of conventional therapies' singularity. It can simultaneously eliminate lesions and restore or enhance functions, covering a range of conditions including genetic disorders, cancer, cognitive impairment, and even anti-aging.
In addition to its applications in the field of disease, precise gene insertion can also allow individuals to safely gain new functions. In the future, it will have applications in agriculture, forestry, and even national defense. This is a technology full of imagination, and we look forward to people's lives becoming better because of our technology.
