ITTBioMed’s Post

Recently, the FDA has approved an increasing number of recombinant adeno-associated virus (rAAV)-based gene therapies for a number of different conditions, including genetic diseases that result in blindness, muscular dystrophy, and blood disorders. However, manufacturing rAAV-based gene therapies is still a challenge, greatly limiting the potential for new therapies. This could change though, as researchers have developed a mechanistic model to optimize rAAV production for gene therapy. By providing a virtual representation, researchers can predict rAAV productivity for different process parameters and baculovirus constructs, offering a solution to current bottlenecks. Solutions like this could greatly expand the potential of rAAV-based gene therapy. #GeneTherapy #rAAV

Figuring out what cell types AAV vectors infect is crucial to optimize the right vectors and finding the right dosage. Read our blog on how single-cell sequencing can help map out AAV tropism.

Capitalizing on research by the Respiratory Gene Therapy Consort jum (GTC), AlveoGene is working first on a treatment for alpha-1 antitrypsin deficiency (AATD), a degenerative condition that can lead to early onset emphysema. The company plans to leverage the #IngenuiTy platform developed by the GTC for the therapy. With the FDA clearance of Grifols at-home AlphaID test in November 2022, the risk of AATD can easily be assessed by US patients, creating a significant opportunity for AlveoGene to fast-track the development of its first candidate AVG-001, a novel, inhaled gene therapy that promises to offer lasting efficacy. https://lnkd.in/gtbViAKK

Adeno-associated viral (AAV) vectors are the most common vehicle for gene therapy biodistribution. That statement is true for the dozen or so #genetherapy platforms accepted to US/UK/EU markets, as well as for novel gene therapy platforms studied in clinical trials and preclinical research. For drug developers currently working on AAV, primary goals are: ▶️ high specificity for target cell types ▶️ low off-target activity ▶️ high transduction efficiency Read this blog to learn how our services on single-cell biodistribution and #AAV transgene detection can provide the high-grade readouts necessary to reach those goals: https://hubs.ly/Q02btRjt0 ---

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

Hemophilia Breakthrough: New Gene Therapies Could Offer a Cure Researchers are developing new gene therapies for hemophilia A and B that are more efficient and less expensive than current therapies: A new hope for a cure Hemophilia A and B are genetic bleeding disorders that affect males. They are caused by mutations in the genes that code for factor VIII and factor IX, respectively, […] #MyHemophiliaLife #Hemophilia #HemophiliaNews #HemophiliaDisease #HemophiliaTreatment #HemophiliaSymptoms #HemophiliaHistory #HemophiliaResearch #HemophiliaHelp

This is an exciting approach for regulation of expression in gene therapy applications.

Nice overview on non-viral delivery systems for gene therapy Non-viral vectors are emerging as a promising approach for gene therapy. Unlike viral vectors, they do not carry the same potential safety risks associated with viral infections. Non-viral vectors can be formulated with various materials such as lipids, polymers, peptides, and inorganic nanoparticles. They have the potential to carry large gene sequences, lower immune responses, and enable large-scale production. However, non-viral vectors face several challenges that limit their efficacy. One significant challenge is the low efficiency of cellular uptake, which means that only a tiny fraction of the administered vector reaches the target cells. Another challenge is the clearance of the vector by immune surveillance, which recognizes the vector as a foreign entity and clears it from the body. Additionally, non-viral vectors can be degraded within the body before they reach their intended target. Despite these challenges, there appears to be continuos progress in developing non-viral vectors for gene therapy.

The translational gap for gene therapies in the developing world - stakeholders, strategies and way forward - https://lnkd.in/d9JRxb6c

Interesting results from Fractyl Health using AAV as a gene therapy solution for increasing GLP-1. I will be very curious to see what this does for AAV therapies. Most of the AAV technologies I have seen were primarily "orphan medicine." The Ozempic and Wegovy GLP-1 craze has shown what Americans are willing to go through and spend to shed some pounds (aside from diabetes treatment). Now think about the scientific and market impact of a single shot, long-lasting version... Could Fractyl Health be on the precipice of the first widespread AAV Gene Therapy? Could vanity be the key driver for the first widespread drug to edit human DNA? I am gearing up for a fun ride, especially since Wyatt light scattering solutions are useful for both AAV and GLP-1 characterization. #genetherapy #aav #GLP-1 #Lightscattering #Wyatttechnology #Waters Waters | Wyatt Technology Waters Corporation https://lnkd.in/gfc58BAz