The relatively short history of cell and gene therapy is not lacking in dramatic moments. A previous outlier, this vibrant field now represents the next great hope and so, when roadblocks to progress are removed or even lowered, theres reason to celebrate. Here, seven members of The Medicine Maker Power List 2022, reflect on the most impactful cell and gene milestones.
There have been many significant breakthroughs in cell and gene therapy over the past few years. Specifically in gene-modified cell therapy, the CAR T story is remarkable. Over the past several years, multiple autologous CAR T therapies have been successfully translated from bench to bedside and received marketing authorization as potentially curative therapies for patients with recalcitrant cancer indications: Kymriah and Yescarta for treating r/r/ ALL, MCL, and LBCL, and Abecma for treating r/r multiple myeloma.
Equally impressive in gene therapy, Zolgensma, an AAVSMN1 gene replacement product, has been developed for use as a one-time gene replacement treatment for infants with spinal muscular atrophy (SMA). The 15 year follow-up study these tiny patients are enrolled in after treatment will inform us on the long-term safety and efficacy of gene replacement therapy.
These products have been translated by academia and SMEs and partnered for advanced development with pharma to achieve both medical and commercial success.
The biggest breakthrough is our increasing ability to edit genes with a growing number of new classes of gene editing tools. This advance has led to the boom of CAR T products and is opening the path to cell engineering and in vivo gene therapy.
In parallel, we are seeing an evolution from viral delivery to alternatives with growing payload capacity. This will, as we are already seeing, lead to cures in diseases where that was unthinkable before!
Cell and gene therapies are at the forefront of innovation and transforming how we treat and potentially cure certain diseases. Cell and gene therapies(CGTs) have the potential to treat severe diseases, such as cancer, as well as rare diseases. Several such therapies are now on the market, including a treatment for an inherited retinal disease that causes blindness. That particular CGT represents an important medical milestone because it was the first curative gene therapy approved for use. Personally, I was excited and humbled at the same time to have been the Global Head bringing this transformational therapy to patients around the world. Many other CGTs are now in development and hopefully will lead to an expansion of the still-limited treatment options available to many patients and transform the clinical paradigm.
An important breakthrough? The demonstration that gain-of-function genetically weaponized somatic cells are potent pharmaceuticals in their own right: living synthetic therapeutics (LSTs).Case in point, after a quarter century of work with TILs and LAKs struggling to meet utilitarian endpoints, enter gain-of-function CAR engineering, and thus history is made.The same paradigm of cell gain-of-function genetic enhancement can readily be applied to alternate somatic cell platforms think MSCs and iPSCs with a limitless potential to improve clinical outcomes for acute and chronic ailments.
Id like to emphasize three milestones. First, the commercialization of gene therapies in general. The efficacy and safety have improved a lot since the 1990s.
Secondly, the explosion of immunotherapies. Onco-hematology has become a major opportunity for patients with otherwise lethal blood cancers.
Finally, the advances in gene editing technologies. These have opened the door to new therapies which we would have considered utterly incredible a few years ago.
The recent approval for Yescarta in second-line (2L) relapsed/refractory large B-cell lymphoma (LBCL) means that an order-of-magnitude more patients just became eligible for potentially curative therapies. One recent industry insight from Celltelligence suggested that moving from 3L to 2L will potentially double the targetable population in diffuse LBCL alone for CAR T cell therapy. As cell therapies move up the treatment paradigm and cell-based therapeutics are eventually approved to treat a range of cancers, the spotlight will turn (again) to manufacturing capacity. At Cellares, our belief is that high-throughput, end-to-end automation is set to revolutionize cell therapy manufacturing, allowing us to deliver more doses at lower cost to meet the demand. Its a truly exciting time for our industry!
The success of the CAR T cell therapy approach and how it has led to cures for childhood leukemias and lymphomas is an amazing story. Thanks to these incredible advances, kids who would no longer be here today are now effectively cured, and are going to live long, relatively healthy lives without suffering the long-term side effects of traditional chemotherapy and radiation. By allowing investigators to be highly creative in developing this approach, a fascinating new treatment process was developed, for both autologous and allogeneic CAR T cell therapies. Now, an entire industry has been born from utilizing patients and donors stem cells and a modified version of the AIDS virus to cure leukemia. This is truly a mind-blowing advancement that combines so many complex processes and biologics and really showcases the power of creative investigators to come up with amazing new treatment solutions.
Read the original post:
The Biggest CGT Breakthroughs Through the Eyes of Our 2022 Power List - The Medicine Maker
- CRISPR: A game-changing genetic engineering technique - January 4th, 2023
- Genetic Counseling Online Course - School of Medicine Columbia ... - January 4th, 2023
- Genetics & Medicine - Site Guide - NCBI - National Center for ... - December 27th, 2022
- Lilly, ProQR to expand genetic medicine development agreement - December 27th, 2022
- Central Dogma and Genetic Medicine - HHMI BioInteractive - November 24th, 2022
- Social, Environmental, Cognitive, and Genetic Influences on the Use of ... - November 24th, 2022
- Genetically modified food controversies - Wikipedia - October 29th, 2022
- BSGM - The British Society for Genetic Medicine - October 13th, 2022
- Genetic and Genomic Medicine - Nationwide Children's Hospital - October 13th, 2022
- Carrier Screening for Genetic Conditions | ACOG - October 13th, 2022
- New NHS genetic testing service could save thousands of children in England - The Guardian - October 13th, 2022
- Vertex, after setbacks, moves forward with second-generation rare disease drug - BioPharma Dive - October 13th, 2022
- Passage Bio Announces Appointment of William Chou, M.D. as Chief Executive Officer - Yahoo Finance - October 13th, 2022
- Metagenomi Announces Participation in October Investor and Industry Conferences - Business Wire - October 4th, 2022
- NovaSeq X machines will make genetic analysis even faster and cheaper - Mezha.Media - October 4th, 2022
- ORYZON to Give Updates on Corporate Progress in October - BioSpace - October 4th, 2022
- e-therapeutics: Fundraise of 13.5 million - BioSpace - October 4th, 2022
- The inheritance of hope curing genetic heart disease now within grasp - British Heart Foundation - October 4th, 2022
- MS Genetic Counseling | Ohio State College of Medicine - September 16th, 2022
- Genetic Counselor - Mayo Clinic College of Medicine & Science - September 16th, 2022
- UCLA researcher awarded National Academy of Medicine prize for work on genetics of autism - Newswise - September 16th, 2022
- Medical research doesn't serve everyone. This effort hopes to build one of the most diverse health databases ever - Colorado Public Radio - September 16th, 2022
- Precision Medicine Offers Hope for More Targeted Rash Diagnosis and Treatment - Dermatology Times - September 16th, 2022
- Redmond firefighters to 'Fill the Boot' for Muscular Dystrophy Association - KTVZ - September 16th, 2022
- Why Monkeypox Wasnt Another COVID-19 - FiveThirtyEight - September 16th, 2022
- Endometriosis Researchers One Step Closer to Diagnosing Condition With Menstrual Blood - Business Wire - September 16th, 2022
- Humans evolved with their microbiomes like genes, your gut microbes pass from one generation to the next - The Conversation - September 16th, 2022
- When to Use Targeted Therapy for Iodine-Refractory Thyroid Cancer - Targeted Oncology - September 16th, 2022
- Gracell Biotechnologies to Participate in Three Upcoming Investor Conferences - PR Newswire - September 16th, 2022
- CRISPR is 10: A decade of gene editing refinements presents new ways to address agricultural diseases thought to be incurable - Genetic Literacy... - September 16th, 2022
- Sarepta Therapeutics Announces Recipients of Route 79, The Duchenne Scholarship Program, for ... - The Bakersfield Californian - September 8th, 2022
- 'Record' Gains in Bringing Research Closer to the Patient - Applied Clinical Trials Online - September 8th, 2022
- Metagenomi Announces Participation in September Investor and Industry Conferences - Business Wire - September 8th, 2022
- Sarepta Therapeutics Announces That FDA has Lifted its Clinical Hold on SRP-5051 for the Treatment of Duchenne Muscular Dystrophy - Yahoo Finance - September 8th, 2022
- Studies Reveal Aggressive Prostate Cancer Linked to Ancestral Heritage - DocWire News - September 8th, 2022
- Lonza and Touchlight collaboration will expands customer's access to DNA - Labiotech.eu - September 8th, 2022
- Genetic variants in miR-145 gene are associated with the risk of asthma in Taiwan | Scientific Reports - Nature.com - September 8th, 2022
- Elderly are more affected by COVID brain fog, but 'cognitive rehab' could be an effective treatment - Genetic Literacy Project - September 8th, 2022
- A therapy found to improve cognitive function in patients with Down syndrome - EurekAlert - September 8th, 2022
- Ovid turns to gene therapy startup to restock drug pipeline - BioPharma Dive - August 30th, 2022
- Morris Animal Foundation Golden Retriever Lifetime Study Celebrates 10 Years - PR Newswire - August 30th, 2022
- Immortal jellyfish genes identified that may explain their long lives - New Scientist - August 30th, 2022
- Walk Again Or Stop Blindness. How Gene Therapy Is Revolutionizing Medicine - Nation World News - August 30th, 2022
- ElevateBio and the University of Pittsburgh Announce Creation of Pitt BioForge BioManufacturing Center at Hazelwood Green to Accelerate Cell and Gene... - August 30th, 2022
- Therapeutic Effects of Tretinoin | JIR - Dove Medical Press - August 30th, 2022
- Physical Activity May Have a Stronger Role than Genes in Longevity - Newswise - August 30th, 2022
- BridgeBio, Baylor College of Medicine to Collaborate on Genetic Disease ... - August 22nd, 2022
- New $2.8-million gene therapy becomes most expensive medicine in history - New Atlas - August 22nd, 2022
- Molecular Map Reveals Insights Into the Genetic Drivers of CLL - The ASCO Post - August 22nd, 2022
- Biopharmaceutical Company Expands Clinical Trials On Texas A&M-Developed Angelman Syndrome Treatment - Texas A&M University Today - August 22nd, 2022
- Restrictive abortion laws are limiting the options parents have after receiving genetic test results, experts say - Yahoo News - August 22nd, 2022
- Marketing and Industry Trends Influencing Precision Medicine in 2022 - Healthcare Tech Outlook - August 22nd, 2022
- expert reaction to study investigating use of genetic risk score for prostate cancer in men with lower urinary tract symptoms to predict diagnosis -... - August 22nd, 2022
- As the Smithsonian wraps a genome exhibit, leaders in the field reflect - STAT - August 22nd, 2022
- Why some people suffer more from COVID-19 than others - EurekAlert - August 22nd, 2022
- Q3 2022 Earnings Forecast for LogicBio Therapeutics, Inc. Issued By William Blair (NASDAQ:LOGC) - Defense World - August 22nd, 2022
- Next generation patient avatars: Expanding the possibilities with MicroOrganospheres - EurekAlert - August 22nd, 2022
- Masters Program in Genetic Counseling - Perelman School of Medicine at ... - August 14th, 2022
- Admissions | Master's Program in Human Genetics & Genetic Counseling ... - August 14th, 2022
- The Silver Lining Of Innovation in Genetic Medicine - Pharmaceutical Executive - August 14th, 2022
- Genetics Team Serves as Go-to for Mom of Son With Extremely Rare Disorder - Stanford Medicine Children's Health Blog - Stanford Children's Health - August 14th, 2022
- UI professor receives grant from National Institutes of Health - Iowa Now - August 14th, 2022
- Lilly's Taltz (ixekizumab) Now Available in New, Citrate-Free Formulation to Reduce Injection Site Pain for Improved Patient Experience - PR Newswire - August 14th, 2022
- Research Roundup: 21 Genes Linked to Increased Risk of Alzheimer's and More - BioSpace - August 14th, 2022
- Personalised medicine made in hospitals can revolutionise the way diseases are treated the challenge now will be implementing it - The Conversation... - August 14th, 2022
- Beyond Paxlovid for covid-19: The hunt for better covid medications - Grid - August 14th, 2022
- PVM to Play Role in Research on New Patent-pending Method to Mass-produce Antitumor Cells to Treat Blood Diseases and Cancer - Purdue University - August 14th, 2022
- Analyzing Kiromic BioPharma (NASDAQ:KRBP) and LogicBio Therapeutics (NASDAQ:LOGC) - Defense World - August 14th, 2022
- Corporate America's Abortion Radicalism - The American Conservative - August 14th, 2022
- A team of Oxford and Mexican researchers want to diversify genomic databases. Can they end 'parachute science,' too? - STAT - August 14th, 2022
- The aging heart accumulates mutations while losing the ability to repair them - EurekAlert - August 14th, 2022
- Generation Bio Reports Business Highlights and Second Quarter 2022 Financial Results - GuruFocus.com - August 5th, 2022
- Population Genetic Testing: Save Lives And Money, While Avoiding Financial Toxicity - Forbes - August 5th, 2022
- Genetic Counselors Scramble Post- Roe to Provide Routine Pregnancy Services without Being Accused of a Crime - Scientific American - August 5th, 2022
- GENERATION BIO CO. Management's Discussion and Analysis of Financial Condition and Results of Operations. (form 10-Q) - Marketscreener.com - August 5th, 2022
- Sangamo Therapeutics Reports Recent Business Highlights and Second Quarter 2022 Financial Results - Yahoo Finance - August 5th, 2022
- Researchers discover one of the largest known bacteria-to-animal gene transfer inside a fruit fly - EurekAlert - August 5th, 2022
- Intellia Therapeutics Announces Second Quarter 2022 Financial Results and Highlights Recent Company Progress - Yahoo Finance - August 5th, 2022
- Medical Student's Crime In Cipolletti: They Found A Man's DNA Under The Victim's Thumb - Nation World News - August 5th, 2022
- A new era: After winding path and safety scares, gene therapy enters the clinic - Sydney Morning Herald - August 5th, 2022