Last year left us with this piece of bombshell news: He Jiankui, the mastermind behind the CRISPR babies scandal, has been sentenced to three years in prison for violating Chinese laws on scientific research and medical management. Two of his colleagues also face prison for genetically engineering human embryos that eventually became the worlds first CRISPRd babies.
The story isnt over: at least one other scientist is eagerly following Hes footsteps in creating gene-edited humans, although he stresses that he wont implant any engineered embryos until receiving regulatory approval.
Biotech stories are rarely this dramatic. But as gene editing tools and assisted reproductive technologies increase in safety and precision, were bound to see ever more mind-bending headlines. Add in a dose of deep learning for drug discovery and synthetic biology, and its fair to say were getting closer to reshaping biology from the ground upboth ourselves and other living creatures around us.
Here are two stories in biotech were keeping our eyes on. Although successes likely wont come to fruition this year (sorry), these futuristic projects may be closer to reality than you think.
The idea of human-animal chimeras immediately triggers ethical aversion, but the dream of engineering replacement human organs in other animals is gaining momentum.
There are two main ways to do this. The slightly less ethically-fraught idea is to grow a fleet of pigs with heavily CRISPRd organs to make them more human-like. It sounds crazy, but scientists have already successfully transplanted pig hearts into baboonsa stand-in for people with heart failurewith some recipients living up to 180 days before they were euthanized. Despite having foreign hearts, the baboons were healthy and acted like their normal buoyant selves post-op.
But for cross-species transplantation, or xenotransplants to work in humans, we need to deal with PERVsa group of nasty pig genes scattered across the porcine genome, remnants of ancient viral infections that can tag along and potentially infect unsuspecting human recipients.
Theres plenty of progress here too: back in 2017 scientists at eGenesis, a startup spun off from Dr. George Churchs lab, used CRISPR to make PERV-free pig cells that eventually became PERV-free piglets after cloning. Then last month, eGenesis reported the birth of Pig3.0, the worlds most CRISPRd animal to further increase organ compatibility. These PERV-free genetic wonders had three pig genes that stimulate immunorejection removed, and nine brand new human genes to make themin theorymore compatible with human physiology. When raised to adulthood, pig3.0 could reproduce and pass on their genetic edits.
Although only a first clinical propotype that needs further validation and refinement, eGenesis is hopeful. According to one (perhaps overzealous) estimate, the first pig-to-human xenotranplant clinical trial could come in just two years.
The more ethically-challenged idea is to grow human organs directly inside other animalsin other words, engineer human-animal hybrid embryos and bring them to term. This approach marries two ethically uncomfortable technologies, germline editing and hybrids, into one solution that has many wondering if these engineered animals may somehow receive a dose of humanness by accident during development. What if, for example, human donor cells end up migrating to the hybrid animals brain?
Nevertheless, this year scientists at the University of Tokyo are planning to grow human tissue in rodent and pig embryos and transplant those hybrids into surrogates for further development. For now, bringing the embryos to term is completely out of the question. But the line between humans and other animals will only be further blurred in 2020, and scientists have begun debating a new label, substantially human, for living organisms that are mainly human in characteristicsbut not completely so.
With over 800 gene therapy trials in the running and several in mature stages, well likely see a leap in new gene medicine approvals and growth in CAR-T spheres. For now, although transformative, the three approved gene therapies have had lackluster market results, spurring some to ponder whether companies may cut down on investment.
The research community, however, is going strong, with a curious bifurcating trend emerging. Let me explain.
Genetic medicine, a grab-bag term for treatments that directly change genes or their expression, is usually an off-the-shelf solution. Cell therapies, such as the blood cancer breakthrough CAR-T, are extremely personalized in that a patients own immune cells are genetically enhanced. But the true power of genetic medicine lies in its potential for hyper-personalization, especially when it comes to rare genetic disorders. In contrast, CAR-Ts broader success may eventually rely on its ability to become one-size-fits-all.
One example of hyper-tailored gene medicine success is the harrowing story of Mila, a six-year-old with Batten disease, a neurodegenerative genetic disorder that is always fatal and was previously untreatable. Thanks to remarkable efforts from multiple teams, however, in just over a year scientists developed a new experimental therapy tailored to her unique genetic mutation. Since receiving the drug, Milas condition improved significantly.
Milas case is a proof-of-concept of the power of N=1 genetic medicine. Its unclear whether other children also carry her particular mutationBatten has more than a dozen different variants, each stemming from different genetic miscodingor if anyone else would ever benefit from the treatment.
For now, monumental costs and other necessary resources make it impossible to pull off similar feats for a broader population. This is a shame, because inherited diseases rarely have a single genetic cause. But costs for genome mapping and DNA synthesis are rapidly declining. Were starting to better understand how mutations lead to varied disorders. And with multiple gene medicines, such as antisense oligonucleotides (ASOs) finally making a comeback after 40 years, its not hard to envision a new era of hyper-personalized genetic treatments, especially for rare diseases.
In contrast, the path forward for CAR-T is to strip its personalization. Both FDA-approved CAR-T therapies require doctors to collect a patients own immune T cells, preserved and shipped to a manufacturer, genetically engineered to boost their cancer-hunting abilities, and infused back into patients. Each cycle is a race against the cancer clock, requiring about three to four weeks to manufacture. Shipping and labor costs further drive up the treatments price tag to hundreds of thousands of dollars per treatment.
These considerable problems have pushed scientists to actively research off-the-shelf CAR-T therapies, which can be made from healthy donor cells in giant batches and cryopreserved. The main stumbling block is immunorejection: engineered cells from donors can cause life-threatening immune problems, or be completely eliminated by the cancer patients immune system and lose efficacy.
The good news? Promising results are coming soon. One idea is to use T cells from umbilical cord blood, which are less likely to generate an immune response. Another is to engineer T cells from induced pluripotent stem cells (iPSC)mature cells returned back to a young, stem-like state. A patients skin cells, for example, could be made into iPSCs that constantly renew themselves, and only pushed to develop into cancer-fighting T cells when needed.
Yet another idea is to use gene editing to delete proteins on T cells that can trigger an immune responsethe first clinical trials with this approach are already underway. With at least nine different off-the-shelf CAR-T in early human trials, well likely see movement in industrialized CAR-T this year.
Theres lots of other stories in biotech we here at Singularity Hub are watching. For example, the use of AI in drug discovery, after years of hype, may finally meet its reckoning. That is, can the technology actually speed up the arduous process of finding new drug targets or the design of new drugs?
Another potentially game-changing story is that of Biogens Alzheimers drug candidate, which reported contradicting results last year but was still submitted to the FDA. If approved, itll be the first drug to slow cognitive decline in a decade. And of course, theres always the potential for another mind-breaking technological leap (or stumble?) thats hard to predict.
In other words: we cant wait to bring you new stories from biotechs cutting edge in 2020.
Image Credit: Image by Konstantin Kolosov from Pixabay
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The Top Biotech Trends We'll Be Watching in 2020 - Singularity Hub
- Technion team discovers important adaptive strategy of the microbiome, impacting immune system - The Jerusalem Post - April 16th, 2024
- Targeting aging and age-related diseases with vaccines - Nature.com - April 16th, 2024
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- After the Smoke Clears: Scars on the Immune System - The Scientist - April 16th, 2024
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- Harnessing the power of the body's own cells to defeat cancer - Press Publications Inc. - April 16th, 2024
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- New insights on B cells: Researchers explore building better antibodies and curbing autoimmune diseases - Medical Xpress - April 16th, 2024
- Immune cells' intense reaction to the coronavirus may lead to pneumonia - Science News Magazine - April 16th, 2024
- The telltale traces long Covid leaves in the blood - healthcare-in-europe.com - April 16th, 2024
- Overview of the Immune System - The Merck Manuals - March 18th, 2024
- SUNDAY Unraveling The Gut-Brain Connection: How Infant Gut Bacteria Shape Immune Resilience | TheHealthSit - TheHealthSite - March 18th, 2024
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- A new strategy to attack aggressive brain cancer shrank tumors in two early tests - ABC News - March 18th, 2024
- Turning on the Bat Signal - The Scientist - March 18th, 2024
- Power Foods That Can Support Your Immune System - Videos from The Weather Channel - The Weather Channel - March 18th, 2024
- Report: Aggressive brain tumors respond to new, immune-focused therapy - UPI News - March 18th, 2024
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- 20.2: Introduction to the Immune System - Biology LibreTexts - February 27th, 2024
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- YOUR HEALTH: The HAMR fights cancer - WAFB - February 27th, 2024
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- How the powerhouse of the cell could be cancers Achilles heel - Freethink - February 27th, 2024
- Food is medicine: The science behind zinc and other supplements for immune health - Healio - February 27th, 2024
- Unleashing Our Immune Response to Quash Cancer - Medscape - February 27th, 2024
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- Is the 100-year old TB vaccine a new weapon against Alzheimers? - The Guardian - February 27th, 2024
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- Had COVID recently? Here's what to know about how long immunity lasts, long COVID, and more - AAMC - February 10th, 2024
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- Research reveals a process tumors use to induce immune suppressor cells and evade immunotherapy - Medical Xpress - February 10th, 2024
- Immune targeting of HIV-1 reservoir cells: a path to elimination strategies and cure - Nature.com - February 10th, 2024
- Preventing severe allergic reactions with nanoparticles - National Institutes of Health (NIH) (.gov) - February 10th, 2024
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- Understanding Immune Checkpoint Inhibition Therapy: Challenges and Strategies - Medriva - February 10th, 2024
- One Simple Change May Dramatically Boost The Effect of COVID-19 Vaccines - ScienceAlert - February 10th, 2024
- The gut virome is associated with stress-induced changes in behaviour and immune responses in mice - Nature.com - February 10th, 2024
- Cancer vaccines are in the works to fight BRCA-linked gene mutations - The Philadelphia Inquirer - February 10th, 2024
- What are the organs of the immune system? - InformedHealth.org - NCBI ... - January 17th, 2024
- Novel insights into the immune response to bacterial T cell superantigens - Nature.com - January 17th, 2024
- FDA signs off on Takeda's HyQvia as maintenance therapy for CIDP - FiercePharma - January 17th, 2024
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- Boosting. What To Do. - Science Based Medicine - January 17th, 2024
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- Long COVID manifests with T cell dysregulation, inflammation and an uncoordinated adaptive immune response to ... - Nature.com - January 17th, 2024
- Opinion | Thanks to mRNA, Future Drugs Will Be Easier and Faster to Make - Mississippi Free Press - January 17th, 2024
- Elon research team models the COVID immune response, one equation at a time - Today at Elon - January 17th, 2024
- Are plant-based meals good for your immune system? 4 things to know about improving your health this week. - Yahoo Life - January 17th, 2024
- Scientists Find Key To Potential Breast Cancer Prevention, Treatment | Newsroom - UNC Health and UNC School of Medicine - January 17th, 2024
- What if every germ hit you at the exact same time? An immunologist explains - The Conversation - January 17th, 2024
- Why Don't We Have a Staph Vaccine? - Healthnews.com - January 17th, 2024
- Best ways to improve your immune system - The Business Standard - January 17th, 2024
- Stanford University researchers think future pandemics could be prevented with universal vaccines - KGO-TV - January 17th, 2024
- Why you may feel depressed and anxious when you're ill and how to cope with it - The Conversation - January 17th, 2024
- New mechanism with potential to boost checkpoint-blocking cancer immunotherapies identified - Medical Xpress - January 17th, 2024
- Battling Bugs and Blues: The Interplay of Infection and Emotion - News-Medical.Net - January 17th, 2024