Sven Kili, ISCT 2022 Annual Meeting co-chair/Courtesy of YouTube
The cell and gene therapy space is growing up and spawning myriad scientific gatherings, including two on the immediate horizon. On May 16, the 25th annual meeting of the American Society of Gene & Cell Therapy (ASGCT) will commence in Washington, DC. And this week in San Francisco, industry, investors, regulators, physicians and patients will convene at the International Society for Cell & Gene Therapy (ISCT)s annual conference the first to be held in person since 2019.
Sven Kili, CEO at Antion Biosciences and 2022 Annual Meeting co-chair told BioSpace that the conference will set the pace for the next 12 months in terms of the most intriguing technology, key investments and key moves.
We often talk jokingly, but it's actually quite serious, that this represents almost the G7 summit of cell and gene therapy, Kili said. Rather than being a place where partnerships and M&A activity is announced (though that may come after), ISCT is focused on really changing the landscape of the cell and gene therapy space.
A summit evokes the idea of key community leaders coming together to make important decisions, and the organizers of ISCT felt it important that every voice is at the table for every key discussion, as opposed to branching off into their own siloed manufacturing or cancer tracks.
At typical scientific meetings, You have a regulatory track and you have a manufacturing track and everyone toddles off to their own track where they sit in isolation and hear about the latest developments, Kili said. ISCT wanted to provide better educational value for its attendees. So, this years meeting will reflect the development pathway of a cell and gene therapy product.
When you develop a therapy, you sit down with a bunch of different people. You sit down with scientists, you sit down with clinicians, you sit down with patients; you might even have a commercial person and a regulatory person. You have a variety of people around the table," Kili said.
This years theme: Bench to Bedside to Benefit - Creating Value for Patients through CGT Translational Science, will integrate the full chorus of voices discussing four main topics: Innovation and R&D the cool new technology; translation into the clinic; late-stage clinical development; and launch and patient access.
Speaking of cool new technology, Geneva, Switzerland-based Antion will be presenting new data around its proprietary miCAR platform, which aims to overcome some of the current limitations in gene editing.
Gene editing, as incredible and as powerful as it is, has some major drawbacks when we try to make more than three or four changes to a cell. As we try to make more and more changes to cells, which we are going to need to do as we get into more complex tumors and solid tumors, gene editing really falls down, Kili said.
This is where Antion, which recently entered into a collaboration funding agreement with Allogene Therapeutics, comes in.
This miCAR platform allows us to silence up to six different genes and add a CAR or a TCR all in a single construct, in a single transduction step, he explained, adding that the technology enables Antion to create very highly modified cells that have very high efficiency and a very good recovery, all without exhausting the cells during the manufacturing process.
This week, Antion will present data that shows the functional silencing of six different genes with a CAR and the activation and activity of the chimeric antigen receptor.
Kili said to also anticipate announcements regarding technological advances in COVID-19 and Acute Respiratory Distress Syndrome (ARDS). Interest in each is understandably at a fever pitch. The second track will focus on overcoming some of the big challenges in early clinical work, such as patient enrollment and running remote clinical trials, while the third will look at the later stages of clinical development.
Its the fourth track, however, where Kili focused most of his thoughts.
This is kind of a coming of age for ISCT, he shared. ISCT, in the many years before, was focused on a lot of R&D work and clinical translation, but we've seen in the last few years more products come into the market and this is an area of creating really good launch excellence. How do you launch and provide therapies that are good value, that change patients lives, that make patients lives easier in the administration? All of these things are going to be critical to discuss.
On May 6, Kili will host a discussion entitled Are Cell & Gene Therapies a First World Only Luxury?
We sit in our first world countries, all very happy and we have access to many of these therapies. But what about patients in South America and Sub-Saharan Africa? What are we doing as an environment about making these therapies available to them? he asked. Kili has brought together panelists including the Institute for Cellular and Molecular Medicine in South Africa, Caring Cross in the United States, Universidad de los Andes in Chile and Novartis.
Looking ahead to the next five-to-10 years, I think we're going to understand the pathophysiology and we're going to understand the cause of particularly cancers like solid tumors, but also some immunology diseases. We're going to understand them on a much better molecular level, Kili predicted. At the moment, we tend to go in, take a cancer and say, these cancer cells have got a little flag that says hello, I'm a CD19, so we say, let's arm a cell and chuck it in there. But we forget that there's an entire orchestra going on in inside that space, and then we wonder why sometimes it doesn't work.
This stronger molecular science will then translate into better understanding and better refinement of the technologies, the cell-based therapies that we're using, so our ability to penetrate and survive those cells in solid tumors in that tumor microenvironment, Kili continued.
He envisions the space making more progress in solid tumors, as well as a progression away from cancer and into immunotherapies, using cell and gene-modified cell therapies for immune conditions, such as diabetes and Systemic lupus erythematosus (SLE).
In the manufacturing space, Kili said the key will be to learn how to usevectors better to create better in vivo therapies, be they gene-modified cell therapies or pure gene therapies. All of these aspects need to move forward, he said, to allow us to decrease the cost of goods for these therapies so that we can make them available to more people both in our very privileged first world, but also in low and middle-income countries.
In conclusion, Kili said, I think we're going to see a lot of very rapid changes and very rapid improvements.
ISCT kicks off Wednesday, May 4, with the excitement continuing through Saturday. BioSpace will have the highlights from both this conference andASGCT.
Excerpt from:
At ISCT This Week, Cell & Gene Therapy Grows Up - BioSpace
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- Sangamo Therapeutics to Regain Full Rights to Hemophilia A Gene Therapy Program Following Pfizers Decision to Cease Development of Giroctocogene... - January 6th, 2025
- JCR Pharmaceuticals and Modalis Therapeutics Announce Transition to the Next Phase of Joint Research Agreement for Development of Novel Gene Therapy -... - January 6th, 2025
- Gene therapy targets the retina to treat eye disease - Nature.com - January 6th, 2025
- Sangamos Stock Plummets as Pfizer Axes Hemophilia Gene Therapy Pact - BioSpace - January 6th, 2025
- How Increased Use of Gene Therapy Treatment for Sickle Cell Disease Could Affect the Federal Budget - Congressional Budget Office - January 6th, 2025
- The Future of Regulatory Processes in Cell and Gene Therapy - Pharmaceutical Executive - January 6th, 2025
- CGTLive's 2024 Pillars of Progress: Most-Watched Conference Interviews - CGTLive - January 6th, 2025
- Pfizer cuts losses on near-approval hemophilia gene therapy, adding to troubled Sangamo's woes - Fierce Biotech - January 6th, 2025
- JCR Pharmaceuticals and Modalis Advance Joint Gene Therapy Research - TipRanks - January 6th, 2025
- JCR and Modalis Advance Joint Gene Therapy Research - TipRanks - January 6th, 2025
- Novartis Gene Therapy Shows Promise in Treating SMA - Yahoo Finance - January 6th, 2025
- Gene Therapy Market to Hit Valuation of US$ 42.26 Billion By 2033 | Astute Analytica - Yahoo Finance - January 6th, 2025
- Novartis gene therapy helps children with rare muscle disorder in study - Reuters - January 6th, 2025
- Capricor Puts Rolling BLA for DMD Cardiomyopathy Cell Therapy Deramiocel in Front of the FDA - CGTLive - January 6th, 2025
- Positive data could expand use of Novartis gene therapy for SMA - Yahoo Finance - January 6th, 2025
- Sangamo spirals after Pfizer halts hemophilia A gene therapy partnership - MM+M Online - January 6th, 2025
- Cell Therapy and Gene Therapy CDMO Market to Reach USD 11.11 Billion by 2030 | Discover Growth Trends and Insights | Valuates Reports - PR Newswire - January 6th, 2025
- Struggling With Adoption, Sickle Cell Gene Therapy Manufacturers Embrace CMS Model - News & Insights - January 6th, 2025
- Sangamo Therapeutics to Regain Rights to Gene Therapy Program from Pfizer - Contract Pharma - January 6th, 2025
- Researchers Create Gene Therapy with Potential to Treat Peripheral Pain ... - December 28th, 2024
- How CRISPR Is Changing Cancer Research and Treatment - December 28th, 2024
- Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease - December 28th, 2024
- 100 cell and gene therapy leaders to watch in 2025 - December 28th, 2024
- Can a new gene therapy reverse heart failure? - Futurity - December 28th, 2024
- Sustained visual improvements in LHON patients treated with AAV gene therapy - Medical Xpress - December 28th, 2024
- Nebraska Medicine administers novel gene therapy to first hemophilia ... - December 28th, 2024
- Gene Therapy for Cardiomyopathies Presents Promising Alternative to Current Treatment - Managed Healthcare Executive - December 28th, 2024
- Stem Cell Transplantation Still the Main Treatment Option for Beta-Thalassemia - Medpage Today - December 28th, 2024
- Caribou Overhyped Gene-Therapy Testing, Investor Class Suit Says - Bloomberg Law - December 28th, 2024
- WuXi AppTec sells off cell and gene therapy operations in US, UK - FirstWord Pharma - December 28th, 2024
- Top 5 Print Publication Articles of 2024 - Managed Healthcare Executive - December 28th, 2024
- Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease - Medpage Today - December 28th, 2024
- UPenn gene therapy pioneers biotech gets $34 million in funding - The Philadelphia Inquirer - December 28th, 2024
- PHC Corporation to present LiCellGrow at Advanced Therapies Week 2025 - Drug Target Review - December 28th, 2024
- The Evolution of Cell & Gene Therapy: Development and Manufacturing Insights and the Role of CDMOs - Pharmaceutical Technology Magazine - December 28th, 2024
- Pig kidney transplants, new schizophrenia drug: Here are 5 of the biggest medical breakthroughs in 2024 - ABC News - December 28th, 2024
- Cell Therapy Manufacturing Trends And Advancements Continuing In 2025 - BioProcess Online - December 28th, 2024
- Can Gene Therapy Treat Chronic Pain? - LabRoots - December 28th, 2024
- Driving innovation: India's foray into gene and cell therapies - The Economic Times - December 28th, 2024
- Governor Hochul Celebrates the Opening Of New York's First Cell and Gene Therapy Hub at Roswell Park Comprehensive Cancer Center in Buffalo - PR Web - December 19th, 2024
- GenSight Biologics Provides Update on Regulatory Discussions and Financial Situation - Business Wire - December 19th, 2024
- Atsena completes dosing in part A of X-linked retinoschisis gene therapy trial - Healio - December 19th, 2024
- Astellas and Sangamo Therapeutics Announce Capsid License Agreement to Deliver Genomic Medicines for Neurological Diseases - StreetInsider.com - December 19th, 2024
- Ring Therapeutics lays off just under half of staff in 2nd wave of cuts this year, CEO set to step down - Fierce Biotech - December 19th, 2024
- Gov. Hochul celebrates opening of first cell and gene therapy hub in NYS - WIVB.com - News 4 - December 19th, 2024
- Muscular Dystrophy Association and Coalition to Cure - GlobeNewswire - December 19th, 2024
- Atsena Therapeutics Announces Dosing Completed in Part A of - GlobeNewswire - December 19th, 2024
- 'A milestone moment': Roswell Park celebrates opening New York's first cell and gene therapy hub - WKBW 7 News Buffalo - December 19th, 2024
- Gene therapy to prevent stillbirth and premature delivery developed - News-Medical.Net - December 19th, 2024
- Breaking through the blood-brain barrier - Science - December 19th, 2024
- Cell therapy weekly: partnerships for advancing cell and gene therapies - RegMedNet - December 19th, 2024
- Roswell Park Opens Cell, Gene Therapy Hub - WGRZ.com - December 19th, 2024
- Cartherics gets $300k grant to advance Cell and Gene Therapy development - ETHealthWorld - December 19th, 2024
- ELMCRx Solutions Offers Cell & Gene Therapy Support Through Partnership with Emerging Therapy Solutions (ETS) - Business Wire - December 19th, 2024
- Fueling the Future of Gene Therapies with Manufacturing Innovation, Upcoming Webinar Hosted by Xtalks - PR Web - December 19th, 2024
- Concinnity secures 3M Seed funding to advance AI-driven gene therapy safety - Tech.eu - December 19th, 2024
- Viral Vectors-Based Gene Therapy for Non-Human Primates Market to Reach Over USD 92.76 Million by 2034 - EIN News - December 19th, 2024
- The pharma industry's silence on RFK Jr., and efforts by parents to develop gene therapies for their children - STAT - December 19th, 2024
- Tenaya reports positive early data on heart gene therapy - Investing.com - December 19th, 2024
- Unraveling The Complexity Of Cell Therapy: Advancements And Challenges - Life Science Leader Magazine - November 27th, 2024
- Novartis wagers more than $1B on gene therapies for the nervous system - BioPharma Dive - November 27th, 2024
- Gene therapy for geographic atrophy in age-related macular degeneration: current insights - Nature.com - November 27th, 2024
- Novartis buys gene therapy startup Kate Therapeutics, joining pursuit of muscular dystrophy treatment - STAT - November 27th, 2024
- At MGB's gene therapy institute, effort to win first venture capital investments continues - The Business Journals - November 27th, 2024
- Neurogene reports death of Rett patient left in critical condition by high dose of gene therapy - Fierce Biotech - November 27th, 2024
- Alzheimer Disease Awareness Month 2024: Looking Back at a Year of Progress in Cell and Gene Therapy - CGTLive - November 27th, 2024
- Why This Gene-Therapy Companys Stock Is Rising 228% - Yahoo! Voices - November 27th, 2024
- How Minaris is Tackling the Scalability Challenge in Cell and Gene Therapy: A Conversation with CEO, Dr. Hiroto Bando - geneonline - November 27th, 2024
- RNA editing is the next frontier in gene therapy heres what you need to know - The Conversation - November 27th, 2024
- Assessment of gene therapy viral vectors in RPE cells - News-Medical.Net - November 27th, 2024
- Retinal organoids and RPE models for retinal gene therapy development - News-Medical.Net - November 27th, 2024
- China Vows to Bolster Gene Therapy Research in Key Biotech Hub - Bloomberg - November 27th, 2024
- Gene Therapy - Volume 31 Issue 11-12, November 2024 - Nature.com - November 27th, 2024
- Iovance Biotherapeutics Announces the Promotion of Raj Puri, M.D., Ph.D. to Chief Regulatory Officer - GlobeNewswire - November 27th, 2024
- Patient Dies in Gene Therapy Trial, But FDA Permits Neurogene to Proceed With Low Dose - MedCity News - November 27th, 2024
- New CRISPR system pauses genes, rather than turning them off permanently - Livescience.com - November 27th, 2024
- Liver-targeting gene therapy lowers mice whole-body SMA symptoms - SMA News Today - November 27th, 2024
- Bright breakthroughs: Real stories of beating rare disease - Science - November 27th, 2024
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