One day, cell and gene therapies will be as common as small molecules and antibody-based therapies are today, according to panelists at BIOs June 8 virtual session, The Next Generation of Medicine: Cell Therapies, Gene Therapies and Beyond.
Because cell and gene therapies have the potential to address complex biological issues such as dysregulation, translocation and mutations, they can use that power to change what the body is doing.
So, while small molecules and antibody therapies will still be around 30 years from now, they will be less important. Cell and gene therapies will dominate, James Sabry, global head of pharma partnering at Roche, said.
In focusing on the future of healthcare, Roche is investing in small company innovation. Its deals with Spark Therapeutics and Adaptive Biotechnologies are prime examples of what it seeks to achieve 10, 20 or even 30 years from now.
Were interested not just in the incremental improvements of antibody therapies or small molecules, but in identifying what could be the therapeutic modalities that will dominate the landscape, Sabry said.
That means cell and gene therapies. Already several gene therapies are approved and others are various companies pipelines.
Spark Therapeutics is working extensively in gene therapies for monogenetic disorders, including hemophilia, said Federico Mingozzi, CSO of Spark.
Spark is validating its gene therapy platform approach with the goal of using it against many more complex diseases.
Assuming a long time frame, one could even imagine using the body as a factory for making antibodies, Sabry said. Essentially, it could be possible to one day turn tissues into biofactories.
Adaptive Biotechnologies has taken its approach to immune-drive medicine from diagnostics into therapeutics.
The immune system doesnt make the distinction between therapeutics and diagnostics the way we do, said Harlan Robins, CSO and co-founder, Adaptive Biotechnologies.
His company focuses on T and B cell receptors, which bind to either pathogens or parts of pathogens, or, in the case of cancer, to mutated pieces of genes.
That binding is how the immune system discovers theres a problem, and its also is how it initiates an immune response, Robins said. Therefore, the same molecule is the diagnostic and targeting molecule. Whats distinct, he said, is the horsepower needed to move a therapeutic along in terms of development. Development for therapeutics is much more intense than for diagnostics. In this case, its partner Roche is providing the horsepower.
Next generation vectors are another hurdle to surmount in advancing cell and gene therapies to the next level, where they may tackle more complex diseases.
There are still enormous opportunities to use (our viral capsid) delivery vehicle for nucleic acids to make them more specific, Mingozzi said. There are a lot of ways to make AAVs more potent and drive expression of the therapeutic gene and then make it more controllable.
Improving the delivery platform will open it to new indications.
Eventually, adenoviral vectors (AVVs) likely will be replaced with more efficient (but not yet determined) delivery systems.
If you think about vectors as a delivery system and a genetic payload, you begin to think that are other ways to getting genetic material into a body, Sabry added.
Exosomes are one possibility, but the future may offer a library of different delivery mechanisms.
Sabry envisions a future in which genetic surgery is performed as routinely as anatomic surgery is performed today.
Imagine going in and removing a disease gene and replacing it with a normal gene, he said. Tight targeting could integrate it at the specific location of the disorder gene. Its not out of the realm of the possible.
The limitations of how to soup-up the genetic manipulations, have not yet been reached, panelists agreed. But there are a lot of barriers. Targeting, especially in cancer, remains a hurdle. To overcome that, Robins suggested making the cells themselves more powerful, but acknowledged safety concerns.
Another approach is to tailor cell therapies specifically to each patient, essentially redefining the meaning of personalized medicine by making therapies for individuals in real time. This necessitates considering not just the cells and the targeting mechanisms, but the integrated and interrelated networks the cells form.
Sabry suggested that Once we start to reestablish normal immune regulation, some of the concerns we have now about having cell therapy either too powerful or not powerful enough will go away. The reason, he said, is that you will be using the regular, nuanced regulatory systems that exist within the immune system to regulate its power and amplitude.
Achieving the future these panelists envision for cell and gene therapies requires companies to take risks. Currently, big pharma does that largely by partnering with or acquiring smaller, innovative companies. In acquisition, though, there is another risk: that the smaller company will be subsumed and lose its innovative drive and, thus, the very reason it was acquired. Therefore its important, Sabry insisted, that innovative companies operate independently, like Genentech and Spark, which both were acquired by Roche.
Its a matter of balance, Mingozzi said. You cant do everything yourself.
Read this article:
BIO 2020: Cell and Gene Therapies Will Dominate Medicine in 30 Years - BioSpace
- Yes, But. The Annotated Atlantic. - November 7th, 2009 [November 7th, 2009]
- Health Insurance Benefit Costs by Region - November 8th, 2009 [November 8th, 2009]
- For an Operator, Please Press... - November 8th, 2009 [November 8th, 2009]
- Pollyanna With a Pen: Maine Governor Signs 18 New Health Care Bills into Law - November 8th, 2009 [November 8th, 2009]
- AMA Sounds the Alarm, Medicare Making Yet Another Attempt to Cut Reimbursement - November 8th, 2009 [November 8th, 2009]
- Mass Governor Asks Blue Cross to Keep Higher Employer Contribution - November 8th, 2009 [November 8th, 2009]
- Lifespan and Care New England Plan Monopoly (Again) - November 8th, 2009 [November 8th, 2009]
- Dirigo Health: Con Artists, Liars, and Thieves? - November 8th, 2009 [November 8th, 2009]
- New Orleans: Health Challenges - November 8th, 2009 [November 8th, 2009]
- August a Flurry of Activity - November 8th, 2009 [November 8th, 2009]
- Maine's Dirigo Health Savings One-Third of Original Estimate - November 8th, 2009 [November 8th, 2009]
- “Methodolatry”: My new favorite term for one of the shortcomings of evidence-based medicine - November 8th, 2009 [November 8th, 2009]
- Suzanne Somers’ Knockout: Dangerous misinformation about cancer (part 1) - November 8th, 2009 [November 8th, 2009]
- A science-based blog about GMO - November 8th, 2009 [November 8th, 2009]
- A Not-So-Split Decision - November 8th, 2009 [November 8th, 2009]
- Military Medicine in Iraq - November 8th, 2009 [November 8th, 2009]
- The effective wordsmithing of Amy Wallace - November 8th, 2009 [November 8th, 2009]
- A Science Lesson from a Homeopath and Behavioral Optometrist - November 8th, 2009 [November 8th, 2009]
- Join CFI in opposing funding mandates for quackery in health care reform - November 8th, 2009 [November 8th, 2009]
- Mainstreaming Science-Based Medicine: A Novel Approach - November 8th, 2009 [November 8th, 2009]
- Those who live in glass houses… - November 8th, 2009 [November 8th, 2009]
- J.B. Handley of the anti-vaccine group Generation Rescue: Misogynistic attacks on journalists who champion science - November 8th, 2009 [November 8th, 2009]
- When homeopaths attack medicine and physics - November 8th, 2009 [November 8th, 2009]
- The cancer screening kerfuffle erupts again: “Rethinking” screening for breast and prostate cancer - November 8th, 2009 [November 8th, 2009]
- All Medicines Are Poison! - November 8th, 2009 [November 8th, 2009]
- When Loud Wins: Will Your Tax Dollars Pay For Prayer? - November 8th, 2009 [November 8th, 2009]
- It’s All in Your Head - November 8th, 2009 [November 8th, 2009]
- The Skeptical O.B. joins the Science-Based Medicine crew - November 8th, 2009 [November 8th, 2009]
- The Tragic Death Toll of Homebirth - November 8th, 2009 [November 8th, 2009]
- What’s the right C-section rate? Higher than you think. - November 8th, 2009 [November 8th, 2009]
- Recombinant Human Antithrombin – Milking Nanny Goats for Big Bucks - December 13th, 2009 [December 13th, 2009]
- Does C-section increase the rate of neonatal death? - December 13th, 2009 [December 13th, 2009]
- Man in Coma 23 Years – Is He Really Conscious? - December 13th, 2009 [December 13th, 2009]
- Why Universal Hepatitis B Vaccination Isn’t Quite Universal - December 13th, 2009 [December 13th, 2009]
- Ontario naturopathic prescribing proposal is bad medicine - December 13th, 2009 [December 13th, 2009]
- Naturopaths and the anti-vaccine movement: Hijacking the law in service of pseudoscience - December 13th, 2009 [December 13th, 2009]
- The Institute for Science in Medicine enters the health care reform fray - December 13th, 2009 [December 13th, 2009]
- Neti pots – Ancient Ayurvedic Treatment Validated by Scientific Evidence - December 13th, 2009 [December 13th, 2009]
- Early Intervention for Autism - December 13th, 2009 [December 13th, 2009]
- A temporary reprieve from legislative madness - December 13th, 2009 [December 13th, 2009]
- A critique of the leading study of American homebirth - December 13th, 2009 [December 13th, 2009]
- Lose those holiday pounds - December 13th, 2009 [December 13th, 2009]
- Endocrine disruptors—the one true cause? - December 13th, 2009 [December 13th, 2009]
- Acupuncture for Chronic Prostatitis/Chronic Pelvic Pain Syndrome - December 13th, 2009 [December 13th, 2009]
- Evidence in Medicine: Experimental Studies - December 13th, 2009 [December 13th, 2009]
- Midwives and the assault on scientific evidence - December 13th, 2009 [December 13th, 2009]
- The Mammogram Post-Mortem - December 13th, 2009 [December 13th, 2009]
- An Influenza Recap: The End of the Second Wave - December 13th, 2009 [December 13th, 2009]
- The End of Chiropractic - December 13th, 2009 [December 13th, 2009]
- Cell phones and cancer again, or: Oh, no! My cell phone’s going to give me cancer! (revisited) - December 20th, 2009 [December 20th, 2009]
- Another wrinkle to the USPSTF mammogram guidelines kerfuffle: What about African-American women? - December 20th, 2009 [December 20th, 2009]
- Acupuncture, the P-Value Fallacy, and Honesty - December 20th, 2009 [December 20th, 2009]
- The One True Cause of All Disease - December 20th, 2009 [December 20th, 2009]
- Communicating with the Locked-In - December 20th, 2009 [December 20th, 2009]
- Are the benefits of breastfeeding oversold? - December 20th, 2009 [December 20th, 2009]
- Measles - December 20th, 2009 [December 20th, 2009]
- Radiation from medical imaging and cancer risk - December 21st, 2009 [December 21st, 2009]
- Multiple Sclerosis and Irrational Exuberance - December 21st, 2009 [December 21st, 2009]
- Medical Fun with Christmas Carols - December 22nd, 2009 [December 22nd, 2009]
- Lithium for ALS – Angioplasty for MS - December 23rd, 2009 [December 23rd, 2009]
- “Toxins”: the new evil humours - December 24th, 2009 [December 24th, 2009]
- 2009’s Top 5 Threats To Science In Medicine - December 24th, 2009 [December 24th, 2009]
- Buteyko Breathing Technique – Nothing to Hyperventilate About - December 26th, 2009 [December 26th, 2009]
- The Graston Technique – Inducing Microtrauma with Instruments - December 29th, 2009 [December 29th, 2009]
- The “pharma shill” gambit - December 29th, 2009 [December 29th, 2009]
- Ginkgo biloba – No Effect - December 30th, 2009 [December 30th, 2009]
- Oppose “Big Floss”; practice alternative dentistry - January 1st, 2010 [January 1st, 2010]
- Causation and Hill’s Criteria - January 3rd, 2010 [January 3rd, 2010]
- The life cycle of translational research - January 10th, 2010 [January 10th, 2010]
- The anti-vaccine movement strikes back against Dr. Paul Offit - January 10th, 2010 [January 10th, 2010]
- Osteoporosis Drugs: Good Medicine or Big Pharma Scam? - January 10th, 2010 [January 10th, 2010]
- Acupuncture for Hot Flashes - January 10th, 2010 [January 10th, 2010]
- The case for neonatal circumcision - January 10th, 2010 [January 10th, 2010]
- A victory for science-based medicine - January 10th, 2010 [January 10th, 2010]
- James Ray and testosterone replacement therapy (TRT) - January 10th, 2010 [January 10th, 2010]
- The Water Cure: Another Example of Self Deception and the “Lone Genius” - January 12th, 2010 [January 12th, 2010]
- Be careful what you wish for, Dr. Dossey, you just might get it - January 13th, 2010 [January 13th, 2010]
- You. You. Who are you calling a You You? - January 15th, 2010 [January 15th, 2010]
- The War on Salt - January 16th, 2010 [January 16th, 2010]
- Is breech vaginal delivery safe? - January 16th, 2010 [January 16th, 2010]