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
- Patient Dies of Acute Liver Failure After Treatment With Sareptas DMD Gene Therapy Elevidys - CGTLive - March 19th, 2025
- Patient dies following muscular dystrophy gene therapy, Sarepta reports - The Associated Press - March 19th, 2025
- Duchenne patient dies after receiving Sarepta gene therapy - March 19th, 2025
- Liver Failure-Associated Death Reported in Patient Treated With Sarepta Gene Therapy Elevidys - MedCity News - March 19th, 2025
- DoD grant funds Hollings researcher's idea to pursue gene therapy for cancer - Medical University of South Carolina - March 19th, 2025
- Recon: Sarepta reports death of teen who received Duchenne gene therapy; Novartis to slash 427 jobs while revamping cardiovascular business -... - March 19th, 2025
- Data Gaps Leave Long-Term Impact of Ex Vivo Gene Therapy in DMD Uncertain - AJMC.com Managed Markets Network - March 19th, 2025
- CHO Plus Obtains U.S. Patent for Improved Production of Viral Vectors for Gene Therapy - Business Wire - March 19th, 2025
- Sarepta Shares Fall on Report of Patient Death After Gene Therapy - Bloomberg - March 19th, 2025
- Hologen AI commits up to $430M to help take MeiraGTx's Parkinson's gene therapy through phase 3 and beyond - Fierce Biotech - March 19th, 2025
- Duchenne patient on Sareptas gene therapy dies - The Business Journals - March 19th, 2025
- Im Unstoppable: New gene therapy cures first New Yorker of sickle cell anemia - PIX11 New York News - March 19th, 2025
- Boost in cancer treatment: PGI working on lab for stem cell, gene therapies - The Times of India - March 19th, 2025
- Man Cured Of Sickle Cell Disease In New York Thanks To New Gene Therapy - Forbes - March 19th, 2025
- Sarepta says teen died after its gene therapy treatment By Reuters - Investing.com - March 19th, 2025
- Innovative Gene Therapy Approach Drives Buy Rating for Insmed in DMD Treatment - TipRanks - March 19th, 2025
- Sarepta says patient dies after treatment with gene therapy - TradingView - March 19th, 2025
- Sarepta tumbles after patient dies following gene therapy treatment - TradingView - March 19th, 2025
- MeiraGTx teams with cryptic AI startup, co-founded by Eric Schmidt, to advance Parkinson's gene therapy - Endpoints News - March 19th, 2025
- Sickle cell anemia patient reunites with Long Island doctors whose gene therapy treatments made him symptom-free - Newsday - March 19th, 2025
- Extracellular vesicles for the delivery of gene therapy - Nature.com - March 9th, 2025
- Around the Helix: Cell and Gene Therapy Company Updates March 5, 2025 - CGTLive - March 9th, 2025
- Inside the secret island where wealthy people go to alter their DNA - Daily Mail - March 9th, 2025
- Regenerons Gene Therapy DB-OTO Trial Shows Promising Hearing Improvement - The Hearing Review - March 9th, 2025
- Global Cell and Gene Therapy Manufacturing Market to Reach ~USD 10 Billion by 2032 | DelveInsight - GlobeNewswire - March 9th, 2025
- College Station gene therapy company partners with nonprofit to develop treatments for rare diseases - KBTX - March 9th, 2025
- World Hearing Day 2025: Looking Back at Progress in Gene Therapy - CGTLive - March 9th, 2025
- Reflecting on a milestone year for cell and gene therapies - Pharmaceutical Technology - March 9th, 2025
- Q&A: Whats Next for Hemophilia Gene Therapy? | Newswise - Newswise - March 9th, 2025
- 'Llife-changing' gene therapy in London partially restores CT child's sight - CT Insider - March 9th, 2025
- The Genesis of Cell Therapy: Bridging Traditional Pharmacology and Gene Therapy - Technology Networks - March 9th, 2025
- Regenxbio at TD Cowen Conference: Gene Therapy Advancements - Investing.com - March 9th, 2025
- Anova Announces First Patient Enrolled to Phase 1/2a Study of DB107 for the Treatment of High-Grade Gliomas - Business Wire - March 9th, 2025
- Apertura Gene Therapy Supports the Broad Institute in Development of Gene Therapy for Prion Disease Using Engineered AAV Capsid Targeting TfR1 for CNS... - March 9th, 2025
- Gene therapy research offers hope for people with chronic kidney disease - Medical Xpress - January 6th, 2025
- 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