Carl Schoellhammer’s Post

The Charles River Laboratories CGT Summit last month was a great event bringing together stakeholders across the drug development landscape from biopharma to investors and other strategics. One of the coolest outputs of the meeting? the graphic below, which was developed live during the meeting. Great encapsulation of a great day of talks. #genetherapy #drugdiscovery #venturecapital

Cell & Gene Day as a Lead Up to ASGCT... I greatly enjoyed the talks and panels that were apart of Endpoints News' Cell & Gene Day yesterday. As Alliance for Regenerative Medicine CEO Tim Hunt put it, there is "palpable excitement" in the field again. I think we at DeciBio have seen similar. I appreciated Richard Wilson's succinct summation of the field requiring global collaboration among policy makers, drug developers, and regulators to make sure we are maximizing benefit to patients. The other critical, often overlooked point he made was in the benefit of having more arrows (e.g., modalities / technologies) in the quiver. Wrong mentality to think "We have B now so we don't need A"... Dead wrong. (That and his sentiment that the "death of AAV" is greatly exaggerated). Hear hear! Ultimately, the field is moving away from "if" and more towards "how", as Alberto Santagostino put it, keeping a reduction in disease burden as our common passion. #pharma #celltherapy #genetherapy #healthcare

Another day another RNA deal... Boehringer Ingelheim is teaming up with Ochre Bio to work on multiple targets for liver disease, including MASH. Rather than silencing certain factors and pathways, Ochre is taking a different approach to enhance the liver's ability to regenerate, and therefore, potentially treat liver disease. The liver has always been a favorite target due to the capacity for LNPs to achieve hepatic delivery. As such, there are no shortage of companies working on similar approaches for MASH and other liver diseases such as Alnylam, GSK, Arrowhead, Ionis, and Novo Nordisk (Dicerna). This includes several candidates in clinical trials. But Ochre is the only one taking a non-silencing approach. Boehringer Ingelheim, no stranger to partnerships in the RNA space, must have seen something they really liked! #genetherapy #RNA #drugdiscovery https://lnkd.in/ghavaK9c

AAV's Broadening Horizons... Nice news on a Friday for AskBio (Bayer) receiving fast track status for their AAV candidate for congestive heart failure. We're seeing more and more AAV programs targeting indications beyond the classical monogenic CNS or eye disorder, including Ask's AB-1002. The asset is presently in Phase II testing and is the only AAV asset in development for this type of indication. On the RNA front, there are several programs for various types of heart failure, including from the likes of Ionis and Cardior (all from our internal TheraTrack program tracker). Nice way to end the week and getting more and more excited for the upcoming American Society of Gene & Cell Therapy annual meeting to talk all things CGT. #pharma #genetherapy #drugdevelopment https://www.askbio.com/

AAV and Crispr paired gene editing... Interesting study out of Stanford University School of Medicine showing difficult to detect insertions of the viral vector genome at the target site when using AAV6 to deliver Crispr machinery. This is one area where AAV could potentially be quite useful as a delivery vehicle, but this latest study calls that into question. While only shown with AAV6, this will raise the question of whether other serotypes would be any better. The authors, however, do show strategies for preventing this, including cutting the vector genome after transduction. The other thing this study demonstrates and implicitly highlights is the need for more advanced analytics and methodologies for carrying out this work. These insertions are typically quite hard to detect and the authors do a nice job of developing advanced methods to enable streamlined readouts. The entire field of CGT could benefit from more advanced methods like these. #genetherapy #research #pharma https://lnkd.in/gcASApaE

Battle of the Biotherapies: AAV vs. RNA for Genetic Payloads... We looked at the landscape of indications in development for AAV and RNA-based medicines from our #TheraTrack platform. No surprise there are competitive programs in the same indication but just how many overlapping indications? ~30% of indications in development across RNA and AAV are shared across both modalities. The true number is probably a bit higher given the number of new programs exploding on the mRNA side that are undisclosed, but its not as significant as one would have perhaps thought. No surprise eye is squarely AAV's domain. Similarly, I&I type indications generally fall in the RNA bucket. Have a look and let us know what you think. How does AAV overcome its present challenges? How does RNA achieve better, more specific delivery? These are the most pressing questions in the field today. #genetherapy #mRNA #healthcare

Dual AAV strategies entering the clinic... iECURE received approval to start a clinical trial of their dual-AAV therapy for ornithine transcarbamylase deficiency in infants utilizing Precision Biosciences' ARCUS technology. This could be a monumental step forward for AAV-based therapies to overcome the cargo size limitations and other hurdles. Obviously, the field will be watching the potential immunogenicity and adverse events, particularly given this trial will be in infants. If the field can develop novel AAV with reduced serotypes, it could become a superior delivery vehicle for genetic medicines. But that's a big IF... This is an indication that also comes with quite a bit of competition. Ultragenyx and Poseida are both developing AAV-based therapies for this indication. You also have Arcturus developing an mRNA-based therapy that's currently being tested in adults (all in our own TheraTrack drug program trackers...). Either way, the multiple strategies for delivering genetic medicines should ultimately help patients. #genetherapy #drugdiscovery #research https://lnkd.in/gTBRMQjg

Delighted to get to participate in the BioTools Innovator west coast pitch event. This is a great program for new startups in the life science space to get to accelerate your vision. I'll be representing DeciBio as well as the BroadOak Capital Partners BioTools Venture Fund that I co-manage with Austin Duke. We are actively deploying capital and look for companies developing innovative "picks and shovels" to support cell and gene therapy and biologics development. If you're working on something in this space, we'd love to connect with you. I also don't mind the chance to spend a few days in San Diego #venturecapital #pharma #drugdiscovery

Allo. versus auto.? All wrong... My colleague Hannah Glazier and I were able to sit down with Peter Olagunju and discuss all things cell therapy covering the past, present, and what the future might hold. Full writeup here: https://lnkd.in/g_5uADyy Some key takeaways from what was an insightful discussion: Allo vs. auto. is all wrong: There will be room for both across different indication types and settings. Of course, auto. has a bit of a head start and with allo. its not so much the engineering challenge that auto faces but one of biology. Underinvestment in QC/QA: The field has focused on the process to optimize every bit of it but at the expense of next-gen. assays and analytical methods. Imagine if you could, in real time, pick out those CAR-Ts that actually recognized and killed cancer, for example? That right there would greatly shorten your expansion time, TAT, and improve potency and potentially reduce side effects. Making a global impact: We have to remember that patients outside of the U.S. and EU can also benefit from these breakthrough medicines and think about how we can effectively deliver them to geographies outside these territories where the burden of certain diseases is astronomically high. Altogether, we're grateful to Peter for sitting down with us and sharing his collective wisdom in cell therapy. #pharma #celltherapy #drugdiscovery

LNP - Lamentable Nano Payloads... Verve with surprising news that they are deprioritizing VERVE-101 after a significant (yet transient) adverse event in their clinical trial testing inactivation of PCSK9. The company will focus on a mirror asset, VERVE-102, that uses the same guide and editor, but a different LNP with GalNAc ligand. The field has been stymied time and again by adverse events tied to LNPs. Arrowhead Pharma and Alnylam each experienced it back in the day. Now Verve. While LNPs helped save the world during the pandemic, this highlights what a blunt instrument they still are (something we wrote about toward the end of the pandemic about the stunting that was inadvertently occurring to research into better delivery technologies). Genetic medicines, sadly, succeed or fail on the back of delivery. We need better delivery vehicles that are more precise, actually targeted, and can achieve extra-hepatic delivery. The field needs to prioritize developing better delivery vehicles so that genetic medicines and next-gen. therapies can truly flourish. #genetherapy #pharma #drugdevelopment https://lnkd.in/grM_Kdxm