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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...
Groundbreaking Intravenous Gene Therapy Offers New Hope for Metachromatic Leukodystrophy Patients
A recent study conducted by researchers from the TIDU GENOV at the Institut du Cerveau in Paris, France, presents promising results for the treatment of Metachromatic Leukodystrophy (MLD), a severe neurodegenerative disease. Led by Emilie Audouard with key...
Astellas’ Friedreich’s ataxia gene therapy cleared for clinical study after earlier version stumbled
After hitting a speed bump in its first attempt at developing a gene therapy for Friedreich’s ataxia, Astellas is ready to put a new version in the clinic. On Thursday, the Japanese drugmaker announced the FDA has cleared its IND for ASP2016. The company is hoping to...
Autolomous and BioCentriq Partner to Speed Up Cell Therapy Delivery
Collaboration leverages cutting-edge digital platform to streamline development and manufacturing activities and speed patient access to life-changing treatmentsLONDON and NEWARK, N.J., April 24, 2024 /PRNewswire/ -- Autolomous, the market-leading developer of...
Study reviews the role of a ribonucleic acid ‘MALAT1’ in hematological malignancies
Recent studies have shown that an abnormally high expression of MALAT1 in blood cancer cells could aid the chemotherapeutic resistance development through multiple intermolecular interactions. Although researchers first identified the role of MALAT1 in patients with...
FDA’s biologics chief Peter Marks previews accelerated approval guidance
The FDA’s Center for Biologics Evaluation and Research is planning to release some crucial guidance soon for CRISPR and gene therapy developers on platform technologies, accelerated approvals for rare diseases, and potentially in the “not-too-distant future,” a...
ImmunityBio’s ANKTIVA® Granted FDA Approval: Breakthrough IL-15 Receptor Agonist First-in-Class for BCG-Unresponsive Non-Muscle Invasive Bladder Cancer
The U.S Food and Drug Administration (FDA) has granted approval to ImmunityBio, Inc. (NASDAQ: IBRX) for their immunotherapy treatment, ANKTIVA® (N-803), in combination with Bacillus Calmette-Guérin (BCG), for patients with BCG-unresponsive non-muscle invasive bladder...
Ipsen and Skyhawk Therapeutics Announce Collaboration on RNA-Modulating Small Molecules for Rare Neurological Diseases
PARIS and BOSTON, April 22, 2024 – In a significant development in the biopharmaceutical industry, Ipsen and Skyhawk Therapeutics have entered into an exclusive global agreement to advance the discovery and development of novel small molecules that target RNA. These...
Duchenne UK and Parent Project Muscular Dystrophy Award $500,000 to Evaluate Safety and Tolerability of Muscle Progenitor Cells in Phase 1 Trial
WASHINGTON, April 23, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization, and the UK charity Duchenne UK, two leading organizations dedicated to ending Duchenne muscular dystrophy (Duchenne), are excited to announce the...
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