Sven Kili is a busy and influential man. In addition to being CEO of Geneva startup Antion Biosciences, hes also an Honorary Professor at University College London, a board member of the Centre for Commercialization of Regenerative Medicine, Chair of the BioIndustry Associations Cell and Gene Therapy Advisory Committee, Co-chair of the Finance and Business Development Committee at the International Society for Cell & Gene Therapy (ISCT), and the list goes on
We sat down with Kili to discuss his perspective on the future of cell and gene therapy and the success of ISCT 2022.
My time serving as a meeting co-chair at ISCT 2022 was absolutely amazing a reawakening! It was the second in person, three dimensional meeting that I had attended since the onset of the pandemic, and it was a real treat to return to catching up with real people old friends, in fact over real cups of coffee. Something as simple as being present in a room while a speaker was presenting, and then being able to interact with them in person was really great.
There were several noticeable changes. The first was the way one asked questions. Very often in remote meetings, your questions are asked in a text box. Then, they could end up being interpreted literally or figuratively by a moderator, before they reach the speaker.
In addition, asking follow-up questions or requesting clarification becomes very onerous. Thus, all questions in virtual events tend to be short and open ended.
By contrast, offline events are far better suited for dialogue for a back and forth that allows for additional clarifying questions. These can then be taken even further after the event, by approaching the given speaker for a longer discussion, perhaps over one of those real cups of coffee.
There is the magic of chance encounters. For example, you might run into an old friend in a corridor, and find yourself lured into tagging along at a session that turns out to be much better than you would have expected, covering topics and taking a format you might never have anticipated. Such novelty only happens offline. A little bit of chaos goes a long way.
More powerful forms of learning happen offline, too. This year I attended a session outside my comfort zone, on manufacturing. At the session, the speaker presented a comparison of a number of cell therapy manufacturing machines and compared their output. It opened up lines of thought in me that would have remained closed had I attended via laptop, perched on a distant dining room chair.
First, there was a roundtable on off-target effects of gene editing. Its a hot topic in the field, so I and many others were very keen to join in and learn. Next, there was another roundtable on the developing needs of the cell and gene therapy workforce. That was also extremely interesting and gave me an opportunity to learn about what other people in other countries and institutions are doing to address the growing shortage. Some of the ideas were truly inspirational. Third, there was a talk on making cell and gene therapies available to patients in less-wealthy countries. This was interesting for me because in my previous life at GSK my colleagues and I did a lot of work trying to make sure that patients in low- and medium-income countries got access to therapies. Sadly it is still a challenge, albeit better understood, and thus the talk allowed us to have a really constructive, engaging discussion and share ideas and successes.
We are now considering not only how to mint more MScs and postdocs, but also how to engage schoolchildren in science and the cell and gene therapy field.
Beyond those three, I attended a roundtable and a talk on CAR T infrastructure. The roundtable focused on the potential for disseminating CAR T administration from a very limited number of key centers to many more smaller hospitals to treat a greater number of patients. The talk was on CAR T optimization, specifically improving the efficacy of allogeneic therapies.
Without a doubt, yes. Though I cant claim to own a crystal ball, there are some areas in which the field is already starting to see effects linking back to the conversations on headcount and workforce planning that took place at our conference. Before the conference, we had already seen a number of articles in magazines and journals hitting on this topic, and even some podcast episodes being put together. Awareness of the challenges is now higher, and efforts to tackle them are growing more coordinated. We are now considering not only how to mint more MScs and postdocs, but also how to engage schoolchildren in science and the cell and gene therapy field. In fact in the UK, this very topic has reached the level of government engagement.
Another really important area where our conversations should have a massive effect is gene editing. Many people are realizing that editing as powerful as the technology may be has its limits. Theyre realizing, too, that within developing technologies lie alternatives to gene editing, especially for more complex engineering tasks.
Were also seeing ongoing dialogue on making advanced therapies available in less wealthy countries. The solution here is not something as simple as shipping cells directly off to the worlds poorest regions. This is about starting at home, and thinking about how we manufacture these technologies at a lower cost, but with the same safety and the same efficacy. By improving the cost:benefit ratio, we can open the door to patients in lower- and medium-income countries. To pull that off, however, we need to keep these critical conversations going.
Without a doubt, we're seeing a shift in priorities towards employees quality of life.
The last hot topic Ill mention is engagement with regulators. In the last year or so it has been very difficult to engage face-to-face with regulators, such as the EMA and the FDA all thanks to a pandemic that not only closed off travel options but left many institutions, services, and corporations short-staffed. And so it was all the more special to have the opportunity to bring representatives from those regulatory bodies into ISCT 2022 for in-person discussions. Very recently, the FDA announced that from 2023 they will be moving back to business as usual, and hiring more staff for assessments and reviews. I like to think that our discussions (and the concerns we relayed) at ISCT 2022 helped sow seeds that will have influenced that decision.
Multiplex cell engineering presents serious challenges. No matter how efficient the modality, once you move into multiplexing, things rapidly grow more complex. Lets say you are editing out four different receptors to silence them. Each edit requires two double-stranded breaks. Thats four DNA breaks multiplied by four targets, giving a total of 16 breaks in the genome. Expecting them all to heal up spontaneously after disruption with congruent end-to-end joining and no translocations or other malformations would be unwise, because this is very seldom possible.
Furthermore, we should acknowledge that editing is not 100 percent effective. If you put 100 cells into the petri dish, youre not going to successfully edit all of them. Youll generally manage a rate of 9096 percent, but in some cases youll score as low as 3040 percent. As you introduce more edits, fewer cells survive the process. To mitigate that, you need to edit in steps. So you do one edit, then another, then another and at every step you have to select and expand the cells that have actually been edited. If youre doing that to T cells repeatedly pushing them into battle-readiness theyll be absolutely exhausted after just three or four edits. From there, theyll be of little to no use in the body.
Without a doubt, were seeing a shift in priorities towards employees quality of life. Instead of paying prospective staff over the odds, a number of companies are now advertising a market rate salary but then offering additional incentives tied to quality of life, such as uncapped holiday time, gym memberships, regular health checkups, coaching and development, and so on.
Much of the point here is to generate mutual feelings and engagement and purpose between the company and the employee. In some ways, it does feel like a return to older models that prioritized bonds and loyalty over employees ship-jumping self-interest and managements hire-and-fire flexibility. Were even seeing this play out in smaller companies that dont necessarily have the financial power to offer super high salaries.
These smaller companies can find themselves at a disadvantage in terms of staff loyalty, since they may lack the resources to train and develop their staff for a range of wider and more advanced roles. If you come in as a technician, youll likely stay a technician. In a larger company, that wont necessarily be the case. And thats why state-sponsored organizations, such as the UKs Cell and Gene Catapult and Canadas CCRM, are absolutely critical; they can provide essential on-the-job training to people working outside big pharma.
Apprenticeship schemes are a great option, too. Either way, on-the-job training is absolutely critical in our environment. We need it to let people grow, develop, and expand their knowledge and horizons. As companies grow in size, theyre able to offer more and more training, which then has a trickle down effect benefiting smaller companies that can continue to germinate and generate knowledge and skills. This tendency should encourage people to stay longer at their companies and develop more value. Ultimately, its about making the working environment as appealing to the worker as possible.
It depends on the company developing the therapy, and it depends on the indication and where the patients are located. For beta thalassemia or sickle cell disease, Sub-Saharan Africa is the most interesting spot. But then for certain other indications, Southeast Asia or South America will be far more interesting.
At ISCT 2022, we were fortunate enough to have speakers from across the global south. We had someone from India, someone from South Africa, and someone from South America, all there to talk about their experiences. Sitting in our comparatively very rich countries, we have a tendency to group all the low- and middle-income countries together. But, of course, what works in Chile will not necessarily work in Nigeria or Thailand. Different countries require different solutions. Understanding those specific contexts and challenges in detail is key to helping cell and gene therapy go global. The rest of the world just needs some love and cell attention.
Follow this link:
Sven Kili on reconvening with the ISCT - The Medicine Maker
- BENITEC BIOPHARMA INC. Management's Discussion and Analysis of Financial Condition and Results of Operations (form 10-Q) - Marketscreener.com - February 13th, 2023 [February 13th, 2023]
- CENTOGENE to Participate in Upcoming Conferences in February in the Lead Up to Rare Disease Day - Marketscreener.com - February 7th, 2023 [February 7th, 2023]
- Gene | Definition, Structure, Expression, & Facts | Britannica - January 27th, 2023 [January 27th, 2023]
- New gene therapy delivers treatment directly to brain - January 27th, 2023 [January 27th, 2023]
- Indian Pharma Congress: Gene-cell therapy, preventive medicine future of health care, says expert - Economic Times - January 25th, 2023 [January 25th, 2023]
- A blood test that identifies people at higher risk of miscarriage? Thats the goal of this award-winning Rutgers med student. - The Philadelphia... - January 19th, 2023 [January 19th, 2023]
- Gene Therapy: Genes As Medicine | Pfizer - January 6th, 2023 [January 6th, 2023]
- How Genomics will ensure a risk-free and beneficial treatment for good health and well-being - The Financial Express - December 28th, 2022 [December 28th, 2022]
- Regenerative Medicine Advanced Therapy Designation | FDA - December 18th, 2022 [December 18th, 2022]
- 3576 - Gene ResultCXCL8 C-X-C motif chemokine ligand 8 [ (human)] - November 23rd, 2022 [November 23rd, 2022]
- Study identifies new gene that drives colon cancer - EurekAlert - October 17th, 2022 [October 17th, 2022]
- Updated Stroke Gene Panels: Rapid evolution of knowledge on monogenic causes of stroke | European Journal of Human Genetics - Nature.com - October 17th, 2022 [October 17th, 2022]
- The challenges of translating CRISPR to the clinic - Labiotech.eu - October 17th, 2022 [October 17th, 2022]
- Editas Medicine Presents Preclinical Data on EDIT-103 for Rhodopsin-associated Autosomal Dominant Retinitis Pigmentosa at the European Society of Gene... - October 17th, 2022 [October 17th, 2022]
- 'We have to find a way': FDA seeks solutions to aid bespoke gene therapy - BioPharma Dive - October 17th, 2022 [October 17th, 2022]
- Mathematical model could bring us closer to effective stem cell therapies - Michigan Medicine - October 17th, 2022 [October 17th, 2022]
- Approval, Commercialization Highlighted at Cell & Gene Meeting on the Mesa - Genetic Engineering & Biotechnology News - October 17th, 2022 [October 17th, 2022]
- CANbridge-UMass Chan Medical School Gene Therapy Research in Oral Presentation at the European Society of Gene and Cell Therapy (ESGCT) 29th Annual... - October 17th, 2022 [October 17th, 2022]
- Depression Treatment: How Genetic Testing Can Help Find the Right Medication - Dunya News - October 17th, 2022 [October 17th, 2022]
- The Risk-Reward Proposition for CGT Clinical Trials - Applied Clinical Trials Online - October 17th, 2022 [October 17th, 2022]
- Precision Medicine Could Get Even More Precise With Allarity Therapeutics Next-Generation Diagnostics - Benzinga - October 17th, 2022 [October 17th, 2022]
- Decibel Therapeutics Receives FDA Clearance of IND Application for DB-OTO, a Gene Therapy Product Candidate Designed to Provide Hearing to Individuals... - October 17th, 2022 [October 17th, 2022]
- Time for your medicine: unlocking the power of our body clocks - The Guardian - October 17th, 2022 [October 17th, 2022]
- Replay establishes distinguished Scientific Advisory Board of genomic medicine and cell therapy experts - Yahoo Finance - October 17th, 2022 [October 17th, 2022]
- Scientists Reappraise the Role of Zombie Cells That Anti-aging Medicine Has Sought to Eliminate - Neuroscience News - October 17th, 2022 [October 17th, 2022]
- Forge Biologics Announces Updated Positive Clinical Data in RESKUE, a Novel Phase 1/2 Gene Therapy Trial for Patients with Krabbe Disease - Business... - October 17th, 2022 [October 17th, 2022]
- Gene Expression Signatures Are Analyzed for Biomarkers of Response in HCC - Targeted Oncology - October 17th, 2022 [October 17th, 2022]
- NHS England World-first national genetic testing service to deliver rapid life-saving checks for babies and kids - NHS England - October 17th, 2022 [October 17th, 2022]
- The proteinprotein relationship that could mend a broken heart - RegMedNet - October 17th, 2022 [October 17th, 2022]
- Study finds microprotein correlated to Alzheimers risk - Daily Trojan Online - October 11th, 2022 [October 11th, 2022]
- Passage Bio Announces Appointment of William Chou, M.D. as Chief Executive Officer - Yahoo Finance - October 11th, 2022 [October 11th, 2022]
- Gene Therapy Rapidly Improves Night Vision in Adults with Congenital Blindness - Newswise - October 11th, 2022 [October 11th, 2022]
- Scientists Discover Protein Partners that Could Heal Heart Muscle | Newsroom - UNC Health and UNC School of Medicine - October 11th, 2022 [October 11th, 2022]
- The Pros and Cons of Lentiviral and Adeno-Associated Viral Vectors - The Medicine Maker - October 11th, 2022 [October 11th, 2022]
- Insights & Outcomes: Foreign DNA, quantum potholes and relapsing fever - Yale News - October 11th, 2022 [October 11th, 2022]
- Expediting IND applications with drug master files - BioPharma Dive - October 11th, 2022 [October 11th, 2022]
- UNC School of Medicine Awarded $3 Million to Lead Study to Reduce PTSD Frequency, Severity | Newsroom - UNC Health and UNC School of Medicine - October 11th, 2022 [October 11th, 2022]
- Lineage to Present at Alliance for Regenerative Medicine 2022 Cell & Gene Meeting on the Mesa - businesswire.com - October 8th, 2022 [October 8th, 2022]
- The Next Crispr Gene Editing IPO Could Be Near - Henry Herald - October 8th, 2022 [October 8th, 2022]
- 10-year CRISPR anniversary: How gene editing revolutionized medicine, and what lies ahead - Genetic Literacy Project - October 8th, 2022 [October 8th, 2022]
- Blood from a baby at birth can be gene sequenced to prevent diseases - USA TODAY - October 8th, 2022 [October 8th, 2022]
- What doctors wish patients knew about breast-cancer prevention - American Medical Association - October 8th, 2022 [October 8th, 2022]
- Growth in Cell and Gene Therapy Market - Pharmaceutical Technology Magazine - October 8th, 2022 [October 8th, 2022]
- Gene Editing Service Market 2022 : Top Players to Reflect Impressive Growth Rate till 2029: Caribou Biosciences, CRISPR Therapeutics, Merck KGaA,... - October 8th, 2022 [October 8th, 2022]
- Tip Sheet: $78 million to support new precision oncology institute, update on experimental gene therapy for herpes and the launch of Fred Hutch's new... - October 8th, 2022 [October 8th, 2022]
- Cell and Gene Therapy: Rewriting the Future of Medicine - Technology Networks - October 2nd, 2022 [October 2nd, 2022]
- Growth in Cell and Gene Therapy Market - BioPharm International - October 2nd, 2022 [October 2nd, 2022]
- CRISPR Therapeutics Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX130 for the Treatment of Cutaneous T-Cell... - October 2nd, 2022 [October 2nd, 2022]
- BioMarin Resubmits Biologics License Application (BLA) for Valoctocogene Roxaparvovec AAV Gene Therapy for Severe Hemophilia A to the FDA - PR... - October 2nd, 2022 [October 2nd, 2022]
- Fighting Breast and Ovarian Cancer With a Lupus Antibody - Yale School of Medicine - October 2nd, 2022 [October 2nd, 2022]
- This gene therapy company is testing new tech to 'switch off' diabetes and obesity with a pill - Euronews - October 2nd, 2022 [October 2nd, 2022]
- Tenaya Therapeutics to Participate in Inaugural Hypertrophic Cardiomyopathy Medical Societys 2022 Scientific Sessions - Yahoo Finance - October 2nd, 2022 [October 2nd, 2022]
- Risk of Alzheimer's dementia may be predicted with help of new tool Washington University School of Medicine in St. Louis - Washington University... - October 2nd, 2022 [October 2nd, 2022]
- Tiny Sea Creature's Genes Shed Light on Evolution of Immunity - UPMC - October 2nd, 2022 [October 2nd, 2022]
- Who will get the call from Stockholm? It's time for STAT's 2022 Nobel Prize predictions - STAT - October 2nd, 2022 [October 2nd, 2022]
- Excision BioTherapeutics Awarded California Institute for Regenerative Medicine (CIRM) Grant to Support Ongoing Phase 1/2 Trial Evaluating EBT-101 as... - October 2nd, 2022 [October 2nd, 2022]
- NeuroVoices: Emma Ciafaloni, MD, on the Vast Expansion of Innovative Approaches to Duchenne Muscular Dystrophy - Neurology Live - October 2nd, 2022 [October 2nd, 2022]
- COVID mRNA Jabs and Testing Kicked Off This Industry of Drug Development: Here's What You Need to Know - The Epoch Times - October 2nd, 2022 [October 2nd, 2022]
- Kidney resident macrophages have distinct subpopulations and occupy distinct microenvironments - University of Alabama at Birmingham - October 2nd, 2022 [October 2nd, 2022]
- Nobel Prize for medicine: the full list of winners - The National - October 2nd, 2022 [October 2nd, 2022]
- The surprising link between circadian disruption and cancer may have to do with temperature - EurekAlert - October 2nd, 2022 [October 2nd, 2022]
- The global live cell imaging market is expected to grow at a CAGR of 8.44% during 2022-2027 - Yahoo Finance - October 2nd, 2022 [October 2nd, 2022]
- Chroma Medicine Announces Formation of Scientific Advisory Board of Global Experts in Gene Editing and Cell and Gene Therapy - PR Newswire - September 20th, 2022 [September 20th, 2022]
- Ring Therapeutics Announces Issuance of U.S. Patent for its Anellovector Compositions - Yahoo Finance - September 20th, 2022 [September 20th, 2022]
- Cholesterol gene mutation: Why would a healthy 27-year-old have severe heart problems? - 69News WFMZ-TV - September 20th, 2022 [September 20th, 2022]
- Gene Therapy for Severe Hemophilia B Could Be More Cost Effective Than Current Treatments - Managed Healthcare Executive - September 20th, 2022 [September 20th, 2022]
- AVROBIO Receives Rare Pediatric Disease Designation from U.S. Food and Drug Administration (FDA) for First Gene Therapy in Development for Cystinosis... - September 20th, 2022 [September 20th, 2022]
- The Biggest CGT Breakthroughs Through the Eyes of Our 2022 Power List - The Medicine Maker - September 20th, 2022 [September 20th, 2022]
- Leading Virus Researcher to Chair UVA's Department of Microbiology, Immunology and Cancer Biology - UVA Health Newsroom - September 20th, 2022 [September 20th, 2022]
- Work remains on Tay-Sachs and other Ashkenazi genetic disorders J. - The Jewish News of Northern California - September 20th, 2022 [September 20th, 2022]
- Study Shows Genetic Link to Moving to the Beat of Music - Newswise - September 20th, 2022 [September 20th, 2022]
- Viewpoint: In the post Roe v Wade world, what changes should a biology textbook writer make to address the medical repercussions of Dobbs? - Genetic... - September 20th, 2022 [September 20th, 2022]
- Alnylam Receives Approval in Europe for AMVUTTRA (vutrisiran) for the Treatment of Hereditary Transthyretin-mediated (hATTR) Amyloidosis in Adult... - September 20th, 2022 [September 20th, 2022]
- CSL flexes gene therapy muscle with latest drug - Sydney Morning Herald - September 20th, 2022 [September 20th, 2022]
- The MIT Press releases new book on the science of the heart from cardiac expert Dr. Sian Harding - EurekAlert - September 20th, 2022 [September 20th, 2022]
- Global Pharmaceutical Contract Manufacturing Market is projected to reach a market value of US$329.7 Billion in 2032: Visiongain Reports Ltd - Yahoo... - September 20th, 2022 [September 20th, 2022]
- Alzheon Reports Industry-Leading Biomarker, Brain Preservation and Clinical Effects Following 12 Months of Treatment in Phase 2 Trial of Oral ALZ-801... - September 20th, 2022 [September 20th, 2022]
- Do You Have Lung Cancer With An EGFR Mutation? If So, The Drug Tagrisso Might Be Right For You Based On New Results From A 'Practice Changing' Trial -... - September 20th, 2022 [September 20th, 2022]
- Getting rid of unwanted transformed cells: Possible new directions in cancer therapy - EurekAlert - September 20th, 2022 [September 20th, 2022]
- Study refutes long-held belief that the Corin gene causes hypertension in African Americans - University of Alabama at Birmingham - September 14th, 2022 [September 14th, 2022]