PacBio’s Post

🚨 Gene Therapy Breakthrough for Sickle Cell Disease 🚨 With FDA approvals of innovative treatments like Casgevy, gene therapy is transforming sickle cell disease (SCD) care. Alongside, Beam Therapeutics’ base editing approach is advancing the field, offering precise solutions like BEAM-101 to activate fetal hemoglobin. As gene therapy evolves, when should we use CRISPR vs. base editing? Discover how KACTUS Bio’s AccuBase™ Base Editor and GMP Cas9 protein are shaping the future of gene editing. 🔗 https://bit.ly/3YnP5EM #GeneTherapy #SickleCell #CRISPR #BaseEditing #Biotech #KACTUS #Casgevy

Ensuring the quality, stability, consistency, and comparability of gene therapies is paramount for their success. Next-generation sequencing (NGS) is a crucial technique for assessing capsid content and detecting non-therapeutic capsids, which can influence the potency, purity, and safety of AAV gene therapy candidates and manufactured products. In her latest article, Claire Aldridge, PhD, CSO of Form Bio, explores how NGS is utilized to characterize DNA contaminants during various stages of AAV production and its impact on the regulatory approval process for AAV gene therapies. Dive into the full article: https://hubs.li/Q02y_ZbC0 #GeneTherapy #NGS #AAV #longreadsequencing

Are you working with recombinant adeno-associated vector (AAV)? Looking into viral vector genome (VG) titer, purity, potency, host and process-related impurities, viral capsid characterization (i.e., the ratio of empty to full capsids), and the identification of replication-competent viruses? If so, check out this article from Cell & Gene for perspectives on the benefits of ddPCR technology for this application and how ddPCR can fit into viral gene therapy workflows. #cellandgenetherapy #lifesciences #medicine #research #aav #viralvector

Are you working with recombinant adeno-associated vector (AAV)? Looking into viral vector genome (VG) titer, purity, potency, host and process-related impurities, viral capsid characterization (i.e., the ratio of empty to full capsids), and the identification of replication-competent viruses? If so, check out this article from Cell & Gene for perspectives on the benefits of ddPCR technology for this application and how ddPCR can fit into viral gene therapy workflows. #cellandgenetherapy #lifesciences #medicine #research #aav #viralvector

AI-inspired medical devices are revolutionizing cell and gene therapy by enhancing the efficiency and precision of therapeutic production. A recent example, Asimov’s AAV Edge System, which combines AI models, host cells, and genetic tools to create an end-to-end platform for gene therapy development. This system includes AI-designed promoters, optimized DNA sequences, and a high-performance bioreactor system. These advancements are making cell and gene therapies more accessible and effective, ultimately improving patient outcomes.

Shelved MLD gene therapy program has a new home! Jim Wilson, formerly of U. Penn, founded a new company, GEMMA Biotherapeutics, and licensed the Passage Bio MLD gene therapy IP which he helped invent. Those of you following MLD therapy developments may remember Passage Bio was one of several companies that were discussing clinical trial design with MLD Foundation when they had to scale back a few years ago due to external economic conditions. We are very excited to see this program find a new home and renewed traction. Dr. Wilson is a backbone of knowledge, inspiration, and hands on development of many active gene therapy programs. Gemma also acquired Passage's Krabbe and GM1 gangliosidosis program, both or which are in later stages of pre-clinical development. There are several additional gene therapy programs in pre-clinical stages at other companies, each offering differing vector and infusion approaches, different target patient populations, and differing inclusion criteria. Over the past three weeks we've met with two new promising international research programs, too! https://lnkd.in/g4hdfyV3 https://lnkd.in/grKhnzWy #FMLD #MLD #genetherapy #raredisease #PassageBio #GEMMAtherapeutics #JimWilson

🔍 A Critical Hurdle in Gene Therapy! Despite tremendous advancements, gene therapy faces a significant challenge: the human immune system. Individuals like Donavon Decker, who suffers from a genetic muscle disorder, enter trials knowing they might never qualify for a second dose due to severe immune reactions. Even after 25 years, this issue remains unresolved. At a recent gene therapy conference, experts explored promising strategies to tackle this obstacle, including innovative immune suppression methods and non-viral delivery alternatives. Will these breakthroughs enable repeated treatments and expand access? The journey to find out is ongoing. #GeneTherapy #HealthcareInnovation #MedicalResearch

Gene therapy treatment is currently very expensive, which usually limits its use to high-income countries like the United States. Another factor is that gene therapies may be approved for use in high-income countries because that's where the clinical studies were performed. Based on my current understanding, it looks like we still have a long way to go before gene therapies can be implemented in low- and middle-income countries. I hope the trajectory will be similar to genome sequencing - as technologies improve, costs and logistical barriers will drop dramatically which will allow for widespread adoption. https://lnkd.in/d_NxFp9k #sciencetranslationalmedicine

With improving technology, gene therapy promises safe and effective treatments for a wide variety of diseases. Nevertheless, proactive steps must be taken to ensure wider development and access. This Science Trans Medicine article reviews the translational gap for gene therapy in low and middle income countries. https://lnkd.in/ey3H_g2i

Discover how analysis of scRNA-seq data supports AAV gene therapy research, in our latest blog. You will learn how it helps with everything from identifying target cells to predicting treatment response and resistance! #genetherapy #scRNAseq #bioinformatics