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Decibel, Principal Scientist
“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
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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...
Promising Early Results for Gene Therapy DB-OTO in Treating Genetic Deafness
Regeneron Pharmaceuticals, Inc. recently shared encouraging early results from the ongoing Phase I/II CHORD trial of DB-OTO, a gene therapy aimed at treating genetic deafness caused by variants of the otoferlin gene. The data was presented at the American Society of...
Groundbreaking CoCas9 Enzyme Unveiled: A Compact and Efficient CRISPR Tool for Genetic Therapy
Introduction: A revolutionary stride has been made in the field of genetic engineering, as researchers from the University of Trento introduce a novel CRISPR enzyme, CoCas9, which promises to enhance the delivery and efficacy of in vivo gene therapy using...
Avidity Biosciences Receives FDA Breakthrough Therapy Designation for Delpacibart Etedesiran (AOC 1001) for Treatment of Myotonic Dystrophy Type 1
Avidity initiating global Phase 3 HARBOR™ study for delpacibart etedesiran this quarter Delpacibart etedesiran data from MARINA-OLE™ showed reversal of disease progression in multiple functional measures in DM1 compared to END-DM1 natural history data SAN DIEGO, May...
NANITE AWARDED $1.8M TO DEVELOP AI-DRIVEN GENE THERAPY FOR HIV
Nanite Receives Funding to Employ SAYERTM Platform to Develop Non-Viral Delivery Vehicles to Express Neutralizing Antibodies In Vivo from the Bill & Melinda Gates Foundation BOSTON, May 8, 2024 /PRNewswire/ -- Nanite, Inc. today announced a $1.8M grant from the...
ASGCT2024: FDA’s Marks on Accelerated Approval, Gene Therapy Costs and More
“$4.25 million!!!” That’s the email Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER), said he received (give or take an exclamation point) from Commissioner Robert Califf when Orchard Therapeutics revealed the price tag of its...
Alzheimer’s Type Identified That Involves Gene Variant Carried by 2% of Population
Researchers headed by a team at the dementia neurobiology group, Sant Pau Research Institute, have found that over 95% of individuals over 65 years of age who have two copies of the APOE4 gene—APOE4 homozygotes—show biological characteristics of Alzheimer’s disease...
Caring Cross and ImmunoAdoptive Cell Therapy (ImmunoACT) Announce Agreement to Commercialize TriCAR-T Cell Immunotherapy for Leukemia and Lymphoma
Agreement allows ImmunoACT to develop and commercialize a novel TriCAR-T cell therapy for leukemia and lymphoma designed to decrease lapses seen in current single CAR-T cell therapies and improve outcomes GAITHERSBURG, Md. and MUMBAI, India, May 7, 2024 /PRNewswire/...
Precision BioSciences Announces Receipt of FDA Fast Track Designation for ECUR-506, iECURE’s Program for the Treatment of Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency
DURHAM, N.C.--(BUSINESS WIRE)-- Precision BioSciences, Inc., (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion, elimination,...
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