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
- 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
- Sarepta Therapeutics Announces Global Licensing and Collaboration Agreement with Arrowhead Pharmaceuticals for Multiple Clinical and Preclinical siRNA... - November 27th, 2024
- A Year of DMD Gene Therapy Trial Failures - AJMC.com Managed Markets Network - November 3rd, 2024
- Hemophilia B: Gene Therapy Shows Promise - Medscape - November 3rd, 2024
- Around the Helix: Cell and Gene Therapy Company Updates October 30, 2024 - CGTLive - November 3rd, 2024
- 2024 PharmaVoice 100s: Cell and Gene Therapy Pioneers - PharmaVoice - November 3rd, 2024
- Cell therapy weekly: support for commercialization of complex therapies - RegMedNet - November 3rd, 2024
- Lexeo shares early data on Alzheimers gene therapy - Endpoints News - November 3rd, 2024
- Medicaid Aiming to Improve Patient Access to High-Cost Therapies - AJMC.com Managed Markets Network - November 3rd, 2024
- The Significance of Gene Therapy in Neuromuscular Medicine at the 2025 MDA Conference: Paul Melmeyer, MPP - Neurology Live - November 3rd, 2024
- OHSU researchers identify gene that could be key to future HIV vaccine - OHSU News - November 3rd, 2024
- Purespring gene therapy reduces kidney scarring in mice and is stably expressed in pigs - Fierce Biotech - November 3rd, 2024
- Data Roundup: October 2024 Features Update for TCR-Based Autologous Cell Therapy in Melanoma, the First Clinical Demonstration of Therapeutic RNA... - November 3rd, 2024
- NewBiologix Launches Xcell to Accelerate, Optimize, and Scale Gene and Cell Therapy Production - Business Wire - November 3rd, 2024
- Vertex Pharmaceuticals and CRISPR Therapeutics Casgevy: the 200 Best Inventions of 2024 - TIME - November 3rd, 2024
- Addressing gene and cell therapy commercialization challenges - TechTarget - November 3rd, 2024
- University of Pennsylvania gene therapy spinout Interius BioTherapeutics doses patient, achieves CAR therapy first - The Business Journals - November 3rd, 2024
- Roche will aim to tackle gene therapy challenges through Dyno deal - The Pharma Letter - November 3rd, 2024
- Behind the Breakthroughs: How to Turn $1,000,000 CAR Ts into Real Medicines - Inside Precision Medicine - November 3rd, 2024
- Terumo automates manufacturing to expand cell & gene therapies - European Pharmaceutical Manufacturer - November 3rd, 2024
- 12-Year-Old Leaves Washington DC Hospital As The First Patient To Receive Approved Gene Therapy For Sickle Cell Disease - AfroTech - November 3rd, 2024
- Lexeo Therapeutics Announces Positive Interim Data for - GlobeNewswire - November 3rd, 2024
- New FDA designations granted to NCATS for rare disease therapies. - NCBI - October 22nd, 2024
- $1.8 Million Awarded to Study the Durability of Gene Therapy - University of Arkansas Newswire - October 22nd, 2024
- By the numbers: US leads charge of cell and gene therapies - BioWorld Online - October 22nd, 2024
- University of Arkansas Researcher Awarded $1.8M for Gene Therapy Study - Arkansas Business - October 22nd, 2024
- Cellectis to Present Data on TALE-Base Editors and Non-Viral Gene Therapy at the ESGCT 31st Annual Congress - StockTitan - October 22nd, 2024
- Around the Helix: Cell and Gene Therapy Company Updates October 16, 2024 - CGTLive - October 22nd, 2024
- Japan mulls ways to boost cell, gene therapy approvals - BioWorld Online - October 22nd, 2024
- A New Type of Gene Therapy Shows Promise for Treating Retinitis Pigmentosa - Managed Healthcare Executive - October 22nd, 2024
- Buy, Sell, Hold: Cell and Gene Therapy - Part 2 - BioPharm International - October 22nd, 2024
- When a Miracle Cure Is Left on the Shelf - Bloomberg - October 22nd, 2024
- Genethon to Showcase the Latest Advances in Gene Therapies for Multiple Diseases at the ESGCT 31 - Business Wire - October 22nd, 2024
- MeiraGTx's gene therapy improves motor function and quality of life in phase 2 Parkinson's trial - Fierce Biotech - October 22nd, 2024
- 5 Sickle Cell Therapies to Watch Following Pfizers Oxbryta Exit - BioSpace - October 22nd, 2024
- Fiocruz and GEMMABio announce partnership for the development of gene therapies - Fiocruz - October 22nd, 2024
- JPMA on Japans Biotech Industry: Cancer, Cardiovascular, and Aging Lead Diseases; Antibody, Cell, and Gene Therapies Top the Innovation List -... - October 22nd, 2024
- Cell and Gene Therapy Clinical Trial Market is expected to reach USD 119.3 Billion by 2032 at a 24.9% of CAGR - PharmiWeb.com - October 22nd, 2024
- Buy, Sell, Hold: Cell and Gene Therapy - Part 3 - Pharmaceutical Technology Magazine - October 22nd, 2024
- The role of quality assurance in accelerating drug development for emerging therapies - pharmaphorum - October 22nd, 2024
- Cellectis to Present Data on TALE-Base Editors and Non-Viral Gene Therapy at the ESGCT 31st Annual Congress - The Manila Times - October 22nd, 2024
- Nucleic Acid and Gene Therapies in Neuromuscular Disorders Market is projected to grow at a CAGR of - PharmiWeb.com - October 22nd, 2024