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“A lot of our programs internally have been having great results with the AAV vectors you all have been packaging. PackGene is rapidly becoming our go-to for AAV vectors! ”
Biogen Scientist
“I have spreaded the word of my satisfaction with your AAV vectors to many people in Biogen’s research labs and I hope we continue to be a regular customer in the future.”
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NeuExcell’s NXL-004 Secures FDA Orphan Drug Designation for Glioma Treatment
On December 7, 2023, Beijing time, NeuExcell Therapeutics announced that its proprietary AAV gene therapy product, NXL-004, designed for treating malignant glioma, has been awarded Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). This...
PackGene Biotech Receives Exclusive C+ Round Investment from SDIC Fund, and Hosts a CGT Industry Salon to Discuss Global Strategies
December 1, 2023, PackGene Biotech, a frontrunner in gene therapy delivery, has successfully closed a landmark C+ funding round, securing over 100 million RMB exclusively from SDIC Fund Management Co., Ltd. This funding, a remarkable accomplishment in the current...
Tauopathy’s Role in Aβ Oligomer Formation: New Insights from Transgenic Primate Models on Alzheimer’s Pathogenesis
Tauopathy, characterized by protein tau and Aβ oligomer accumulation, is a significant aspect of Alzheimer's disease. The causal relationship between these accumulations and neurodegeneration remains unclear despite their links to various cognitive and movement...
Targeted Lysosomal Degradation of Mutant Huntingtin by Engineered Intrabody Peptide SM3
Huntington's disease (HD) is a devastating neurodegenerative condition caused by a mutation in the Huntingtin gene (HTT). This leads to the production of a mutant form of the huntingtin protein (mHTT) with an extended polyglutamine (polyQ) repeat, which has a...
Researchers Unveil a Novel Strategy for Protein Miniaturization in the CRISPR-Cas System
In a groundbreaking study published in Nature Communications, a team of researchers from China have introduced an innovative approach to protein miniaturization within the gene-editing realm of the CRISPR-Cas system. The CRISPR-Cas system is celebrated for its...
PackGene Biotech and Kudo Biotechnology Partner to Offer Customized mRNA Manufacturing Services
Collaboration provides end-to-end coverage of mRNA drug and vaccine development and manufacturing workflow HOUSTON, TX and NEEDHAM, MA – PackGene Biotech (PackGene) and Kudo Biotechnology (Kudo Bio) have partnered to provide high-quality, customized messenger RNA...
Zolgensma’s Success in SMA Treatment Advocates for AAV-based Gene Therapy and Promises a Bright Future for Genetic Disorders
At the 2023 Muscular Dystrophy Association Clinical and Scientific Conference, Novartis presented new data highlighting the continued efficacy and durability of its gene therapy treatment, Zolgensma, in treating spinal muscular atrophy (SMA). Zolgensma has been shown...
International Rare Disease Day | PackGene GMP plasmid is DMF registered, helping drug applications and shining lights on rare disease therapy!
February 28th is the 16th International Rare Disease Day with the theme of "Shine lights on your life" . Rare diseases, also known as "orphan diseases", have a low incidence rate and multiple disease types. Most of them are chronic, lifelong, severe, and...
Cell & Gene Meeting on the MESA (ARM; Phoenix, AZ, Oct 7-9)
The 2024 Cell & Gene Meeting on the Mesa is the premier annual conference for industry leaders, focused on innovative research and overcoming commercial challenges in cell and gene therapy. The event features expert panels, networking opportunities, and over 100...
Socienty for Neuroscience (Chicago, IL, Oct 5-9)
Annually, scientists from across the globe gather to explore new concepts, present their research, and experience the pinnacle of their field. By attending, you can present your own research, network with fellow scientists, participate in sessions and events, and...
CASSS-CGTP ( Rockville, MD, Jun 11-13)
The Cell and Gene Therapy Products (CGTP) Symposium focuses on Manufacturing, Quality, and Regulatory Considerations, fostering an exchange of scientific ideas and interactions with regulators that shape evolving regulatory frameworks for these innovative products....
BIO International (San Diego, Jun 3-6, 2024)
Whether you're involved in pharmaceuticals, biotech, academia, non-profits, or government, or working as a researcher, business developer, or investor, BIO is the place to establish valuable connections. The Premier Access package includes our BIO One-on-One...
Webinar: Treating Autosomal Recessive Deafness 9 with AAV1-hOTOF Gene Therapy (4/24/2024 10:00am EST/ 7:00am PST)
Abstract:Discover a groundbreaking advancement in treating Autosomal Recessive Deafness 9 (ARD9) with Dr. Yilai Shu, a pioneer in gene therapy for hearing loss. This webinar delves into the innovative AAV1-hOTOF gene therapy trial, offering new hope for individuals...
ASGCT (Maryland, May 7-11, 2024)
Abstract:Welcome to ASGCT 2024, the American Society of Gene and Cell Therapy's flagship conference. Esteemed researchers, clinicians, and industry leaders converge at this influential event to navigate the forefront of gene and cell therapy. In the vibrant atmosphere...
American Academy of Neurology (Colorado, Apri 13-18, 2024)
Abstract:The American Academy of Neurology Conference 2024 in Denver, a pivotal gathering for the brightest minds in neurology. This esteemed event brings together experts, researchers, clinicians, and innovators from around the globe to explore the forefront of...
Advanced Therapies Congress (London, Mar 19-20, 2024)
Abstract:The Advanced Therapies Congress convenes leaders in cell and gene therapy development across diverse sectors: pharma, biotech, startups, researchers, clinicians, academics, HTAs, payers, and regulators. In 2024, our new Gene Editing Track highlights CRISPR...
Pfizer’s New Gene Therapy BEQVEZ™ Wins FDA Approval for Hemophilia B
In a notable advancement for hemophilia treatment, Pfizer Inc. (NYSE: PFE) has received U.S. Food and Drug Administration approval for BEQVEZ™ (fidanacogene elaparvovec-dzkt), a one-time gene therapy aimed at adults suffering from moderate to severe hemophilia B. This...
Astellas Teams Up with Poseida Therapeutics for Innovative CAR-T Cell Therapy Development
Astellas Pharma, through its subsidiary Xyphos Biosciences, has entered into a second collaborative agreement with Poseida Therapeutics to pioneer the development of off-the-shelf cell therapies for solid tumors. This follows their initial partnership in 2023, which...
Cytiva Introduces New Cell Manufacturing Lines for Viral Vectors
Viral vector-based gene therapies hold immense promise for treating a multitude of diseases. However, their widespread adoption has been hindered by inefficient manufacturing methods and the absence of established standardized processes, resulting in high costs and...
Top 10 Best-Selling Cell and Gene Therapies
When it comes to cell and gene therapy revenues, it’s feast or famine. The feast side is well covered in this A-List, which ranks the top 10 therapies according to sales figures from 2023. The famine side begins almost exactly where this A-List ends. Future A-Lists...
Cytiva Introduces ELEVECTA Cell Lines
Global life sciences company, Cytiva, is creating solutions to the challenges of viral vector-based gene therapies with its new ELEVECTA cell lines. According to Emmanuel Abate, President Genomic Medicine, Cytiva: “To make gene therapy a realistic option for more...
An electrifying discovery may help doctors deliver more effective gene therapies
Experimental treatment groups involving hepatocytes exposed to various doses of AAV, AAV* (AAV pre-treated with EP), or plasmids, with or without electric field pulse (EP) exposure (illustrations not drawn to scale). Credit: PLOS ONE (2024). DOI:...
Lilly scraps trials of Verzenio, Jardiance and a gene therapy from Prevail
As part of its quarterly cleaning, Eli Lilly removed a few trials from its pipeline, including late-stage studies of approved cancer drug Verzenio and its Boehringer Ingelheim-partnered drug Jardiance. The Indianapolis-based pharma giant also removed a Phase 2 trial...
Researchers explore new cell target for cystic fibrosis treatment
Schematic model of working hypothesis of how (A) pulmonary ionocytes and (B) club cells function in pHBE cell culture stimulated with forskolin plus IBMX. Credit: American Journal of Respiratory and Critical Care Medicine (2024). DOI: 10.1164/rccm.202309-1565OCA team...
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