AAV

Life sciences companies are set to compete in a Dragon’s Den session at the BIA’s BioProcessUK event this month.   Taking place on day two of BIA BioProcess UK, the Dragon’s Den special session is focused on next generation analytical technologies and will see six companies compete for an audience vote.   Chairing the session will be […]

In the In Conversation With series, a part of the free DDW podcast, DDW speaks with members of the drug discovery industry about their work and how it helps turn science into business.    In this episode, Megan Thomas is in conversation with Dr Jude Samulski, Co-founder and Chief Scientific Officer at AskBio. Dr. Samulski’s graduate work demonstrated the […]

Earlier this year, DDW hosted a webinar, ‘A comprehensive guide to using adeno-associated virus (AAV) vectors in gene therapy’, supported by Azenta Life Sciences. The event aimed to help the audience understand the AAV workflow, from upstream methods for AAV production and validation to downstream analysis of the packaged product for enhanced therapeutic development.  AAV […]

Evox Therapeutics has agreed a research collaboration and option agreement with the Icahn School of Medicine at Mount Sinai (Icahn Mount Sinai) in New York, NY, US. The collaboration will work on developing exosome-encapsulated AAV (exoAAV) vectors as a novel gene delivery technology aimed at improving treatments for heart disease. Together, Evox and Icahn Mount […]

Join DDW for this exclusive free event, A Comprehensive Guide to Using Adeno-Associated Virus (AAV) Vectors in Gene Therapy, which is supported by Azenta Life Sciences.  AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers. This webinar will help you understand the […]

Join DDW for this exclusive free event, A Comprehensive Guide to Using Adeno-Associated Virus (AAV) Vectors in Gene Therapy, which is supported by Azenta Life Sciences.  AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers. This webinar will help you understand the […]

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD. It is approved in ambulatory paediatric patients aged […]

Join DDW for this exclusive free event, A Comprehensive Guide to Using Adeno-Associated Virus (AAV) Vectors in Gene Therapy, which is supported by Azenta Life Sciences.  AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers. This webinar will help you understand the […]

Evox Therapeutics has purchased Codiak Biosciences engEx-AAV technology platform, including all intellectual property rights, and exclusive access to certain engineering and manufacturing rights. The technology enables active loading of adeno-associated virus (AAV) into exosomes followed by their release when the AAV-loaded exosomes are delivered into recipient cells. This has been demonstrated to be an effective […]

AAV is optimal for gene therapy due to its efficiency and safety in humans; however, AAV processes can present obstacles for researchers. In this webinar, supported by Azenta Life Sciences, we’ll help you understand the AAV workflow, from upstream methods for AAV production and validation to downstream analysis of the packaged product for enhanced therapeutic […]

Gene delivery technologies company VectorBuilder has opened a new office in Edinburgh, hoping to bolster its presence in the United Kingdom. The new location is part of Edinburgh Technopole, a Pioneer Group in Midlothian Science Zone. “When it comes to published scientific research, the UK is ranked third in the world with nearly 200,000 citable […]

Sania Therapeutics, a new company focused on developing genetic medicines for neural circuit dysfunction, has launched at the American Society of Gene & Cell Therapy (ASGCT) conference. The company’s approach combines adeno-associated viruses (AAV) in human neural circuits with tuneable conditionally-activated ion channels. This corrects dysfunctional neural excitation. Sania’s technology platforms were first developed at […]

New South African company NewBiologix has launched with the aim of developing a proprietary and breakthrough platform for the advanced engineering of cell lines used to manufacture gene and cell therapies. The company’s DNA-based platform is being developed to address manufacturing challenges associated with the production of recombinant adeno-associated virus (rAAV) vectors used to produce […]

The European Medicines Agency (EMA) has granted PRIME (Priority Medicines) status to the gene therapy, GNT-0003, currently in clinical trials for Crigler-Najjar syndrome, a rare liver disease.   This status, granted only to drug candidates with major therapeutic potential, follows the successful completion of the first phases of the clinical trials.  The manufacturer, Genethon, is a […]

Svea Cheeseman, Refeyn, explains why better efficiency is needed to advance the production of viral vectors for use in gene therapies. Adeno-associated viruses (AAVs) are a promising, widely used vector for delivering gene therapies. As demand for AAV gene therapies increases, it is becoming essential to maximise the efficiency and accuracy of AAV vector production. […]

The latest episode of the DDW Highlights podcast is now available to listen to below. DDW’s Megan Thomas narrates five key stories of the week to keep DDW subscribers up-to-date on the latest industry updates.  Cell and gene therapies have been making the news this week, as we launch our very own guide to global innovation in CGT with the Winter […]