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
- CellOrigin secured a new round of investment for developing its globally proprietary iPSC-CAR-Macrophage technology platform - WWNY - October 16th, 2021
- ASU professor researches origins of Alzheimers to find a cure - Eight, Arizona PBS - October 16th, 2021
- Personalized Cell Therapy Market Size Worth US$ 53.8 Billion With a CAGR of 23.5% By 2028 Otterbein 360 - Otterbein 360 - October 16th, 2021
- Citius Pharmaceuticals to Host Investor Webcast to Discuss the Acquisition of Late Phase 3 Cancer Immunotherapy I/ONTAK (E7777) - Yahoo Finance - October 16th, 2021
- Wolter Earns Young Investigator Award | Newsroom - UNC Health and UNC School of Medicine - October 16th, 2021
- NIH awards nearly $2M to Huebsch for study | The Source - Washington University in St. Louis Newsroom - August 31st, 2021
- Shoreline Launches with $4 Billion in Partnerships PharmaLive - PharmaLive - August 31st, 2021
- Bone Therapeutics announces topline results from Phase III knee osteoarthritis study with its enhanced viscosupplement JTA-004 - GlobeNewswire - August 31st, 2021
- Roslin Tech in multi-million bid to fund sustainable food - HeraldScotland - August 31st, 2021
- Global Induced Pluripotent Stem Cells Market Industry Analysis, Post COVID-19 Impact, Emerging Trends, Business Growth by 2027||Fate Therapeutics,... - July 22nd, 2021
- Bayer's BlueRock Therapeutics gains FDA fast track for Parkinson's disease cell therapy - PMLiVE - July 22nd, 2021
- Global Induced Pluripotent Stem Cell (iPSC) Market to Reach $2.3 Billion by 2026 - PRNewswire - July 7th, 2021
- Impact of NK cell-based therapeutics for Lung Cancer Therapy | BTT - Dove Medical Press - July 7th, 2021
- Caribou eyes $100M IPO as it aims off-the-shelf CAR-Ts at the clinic - FierceBiotech - July 7th, 2021
- Genexine, Toolgen to co-develop CAR-NK cell gene therapy - Korea Biomedical Review - July 7th, 2021
- Reversal of biological age detected in mouse and human embryos - BioNews - July 7th, 2021
- Century Therapeutics Announces Closing of Initial Public Offering and Full Exercise of Underwriters' Option to Purchase - GlobeNewswire - June 25th, 2021
- Univ. of Washington and Sana researchers use gene editing to prep stem cells for heart repair - GeekWire - June 25th, 2021
- Ubiquitination is essential for recovery of cellular activities after heat shock - Science Magazine - June 25th, 2021
- The CCL2-CCR2 astrocyte-cancer cell axis in tumor extravasation at the brain - Science Advances - June 25th, 2021
- Pharmacogenomic approach to cure the pain of the 'burning man' - Lab + Life Scientist - June 25th, 2021
- The Stem Cell Assay Market To Grow Through Mergers Amongst Market Participants The Courier - The Courier - June 25th, 2021
- Accelerated Biosciences' Immune-Privileged Human Trophoblast Stem Cells (hTSCs) Offer Breakthrough Opportunities in Cancer-Targeting Therapeutics and... - May 15th, 2021
- Study Models the Effect of Herpes Infection on Fetal Brain Development - Pharmacy Times - May 15th, 2021
- Induced Pluripotent Stem Cells Market is set for Lucrative Growth | Top Companies Thermo Fisher Scientific Inc., FUJIFILM Corporation, KSU | The... - May 15th, 2021
- Stem Cell Therapy Market by Type, Therapeutic Application and Cell Source - Global Forecasts to 2026 - ResearchAndMarkets.com - Business Wire - May 15th, 2021
- Global Induced Pluripotent Market Showing Impressive Growth by 2027||Thermo Fisher Scientific; Cell Applications, Inc.; Axol Bioscience Ltd.;... - May 15th, 2021
- Global Induced Pluripotent Stem Cells Market 2020 Top Manufactures, Growth Opportunities and Investment Feasibility 2025 The Courier - The Courier - May 3rd, 2021
- Stem Cell Therapy Market worth $401 million by 2026 - Exclusive Report by MarketsandMarkets - PRNewswire - May 3rd, 2021
- Fate Therapeutics Announces Four Presentations at the 2021 ASGCT Annual Meeting - GlobeNewswire - May 3rd, 2021
- Induced Pluripotent Stem Cells (iPSCs) Market 2021 Is Booming Across the Globe by Share, Size, Growth, Segments and Forecast to 2027 | Top Players... - May 3rd, 2021
- Induced Pluripotent Stem Cells (iPSCs) Market 2021 Industry Size, Share, Growth and Top Companies Analysis- Fujifilm Holding Corporation (CDI),... - May 3rd, 2021
- A New CRISPR Tool Flips Genes On and Off Like a Light Switch - Singularity Hub - May 3rd, 2021
- Stem Cell Characterization Kits Market Report 2021 by Global Key Players, Types, Applications, Countries, Size, Forecast to 2027 Good News Gum - Good... - May 3rd, 2021
- Induced Pluripotent Stem Cells Market Growth Opportunities, Analysis and Forecasts Report 2020-2026 with key players position (Fujifilm Holding... - February 17th, 2021
- Cytovia Therapeutics and Cellectis Partner to Develop TALEN Gene-Edited iPSC-Derived Natural Killer Cells - BioSpace - February 17th, 2021
- Can we freeze them? Yes we can, says Nkarta - Vantage - February 17th, 2021
- Global Induced Pluripotent Market Positive Outlook, Revenue Generation & Leading Manufacturers, Forecast 2026||CELGENE CORPORATION; Astellas... - February 17th, 2021
- Induced Pluripotent Stem Cells Market Growth, Competitive Landscape, Research Methodology, Business Opportunities, Statistics and Industry Analysis... - February 17th, 2021
- Evotec and Medical Center Hamburg-Eppendorf Enter Partnership to Develop iPSC-Based Tissue Therapy f - PharmiWeb.com - February 4th, 2021
- Breakthrough stem cell therapy may reverse life-threatening conditions in dogs - Study Finds - February 4th, 2021
- SLAS Technology Special Collection on Artificial Intelligence in Process Automation Available Now - Newswise - February 4th, 2021
- Exacis Biotherapeutics Announces Key Addition To Its Executive Leadership Team With Dirk Huebner MD Joining As Chief Medical Officer - PRNewswire - February 4th, 2021
- Induced Pluripotent Stem Cells Market 2020 Global Share, Growth, Size, Opportunities, Trends, Regional Overview, Leading Company Analysis And Forecast... - February 4th, 2021
- Money on the Move: January 27 February 2 - BioSpace - February 4th, 2021
- Poultry Healthcare Products Market Share, Growth, Statistics, by Application, Production, Revenue & Forecast to 2025 - AlgosOnline - January 27th, 2021
- Impact Of Covid 19 On Stem Cells Industry 2020 Market Challenges, Business Overview And Forecast Research Study 2026 Murphy's Hockey Law - Murphy's... - January 27th, 2021
- SREBP1 suppresses the differentiation and epithelial function of hiPSC-derived endothelial cells by inhibiting the microRNA199b-5p pathway - DocWire... - January 25th, 2021
- Induced Pluripotent Stem Cells Market Segmentation and Analysis by Latest Trends, Development and Growth by Trending Regions 2020 with key players... - January 25th, 2021
- Induced Pluripotent Stem Cells (iPSCs) Market to Witness Massive Growth During 2021-2027 | Fujifilm Holding Corporation (CDI), Ncardia, Sumitomo... - January 25th, 2021
- Hands across the water as bio bonds stand test of time | Business Weekly - Business Weekly - January 25th, 2021
- Exercise mimetics and JAK inhibition attenuate IFN-induced wasting in engineered human skeletal muscle - Science Advances - January 25th, 2021
- Can Science Save the Northern White Rhino? - Freethink - January 25th, 2021
- Induced Pluripotent Stem Cells Market to Grow with an Impressive CAGR - Farming Sector - January 9th, 2021
- Accelerating cell-based therapies by providing safe therapeutic MSC products - BioSpace - January 9th, 2021
- Exacis Biotherapeutics Announces Its Launch and mRNA Technology In-Licensing For Targeted CAR-NK And CAR-T Cell Cancer Therapies | DNA RNA and Cells |... - January 9th, 2021
- Environmental Factor - January 2021: Intramural Papers of the Month - Environmental Factor Newsletter - January 9th, 2021
- Global Induced Pluripotent Stem Cells Market To Witness Huge Gains Over 2020-2026 - Factory Gate - December 28th, 2020
- Exosomes act as messengers and decoys to save healthy cells from viral infection - Massive Science - December 28th, 2020
- Scientists have restored youth to aging eyes in mice - Massive Science - December 21st, 2020
- Top Technical Advances of 2020 - The Scientist - December 21st, 2020
- A new psychedelic drug gives psychiatric benefits without causing hallucinations - Massive Science - December 21st, 2020
- I Peace, Inc. and Avery Therapeutics announce collaboration to bring iPSC derived cell therapy for heart failure to the clinic - PRNewswire - December 17th, 2020
- Network of Genes Involved in Congenital Heart Disease Identified - Technology Networks - December 17th, 2020
- Global Induced Pluripotent Market 2020-26 Steering Forces Heading Towards Impressive CAGR With CELGENE CORPORATION; Astellas Pharma Inc.; Thermo... - December 17th, 2020
- Global Induced Pluripotent Stem Cells (iPSCs) Market Expectable to Exceed Global Market Revenue, Size, Segments and Market Competition Trend to... - December 14th, 2020
- Evotec and Sartorius Partner with Start-Up Curexsys on IPSC-Based Therapeutic Exosome Approach - BioSpace - December 9th, 2020
- Multiple gene edits and computer simulations could help treat rare genetic diseases - University of Wisconsin-Madison - December 9th, 2020
- Cytovia Therapeutics announces plans to initiate in 2021 Clinical Development of Universal iPSC NK Cell Therapy for Hematological and Solid Tumors -... - December 9th, 2020
- Fate Therapeutics Reports Positive Interim Data from its Phase 1 Study of FT516 in Combination with Rituximab for B-cell Lymphoma - GlobeNewswire - December 9th, 2020
- Constantly Growing Applications and Innovations to Push Sales of Amniotic Membrane Market Up To ~US$2.4 Bn by 2027, Observes TMR - PRNewswire - December 9th, 2020
- Tulane researcher shows enhanced therapeutic stem cell migration improves neurodegenerative disease - News from Tulane - December 8th, 2020
- Researchers restore lost sight in mice, offering clues to reversing aging - Science Magazine - December 8th, 2020
- Fate Therapeutics Reports Positive Interim Data from its Phase 1 Study of FT516 in Combination with Rituximab for B-cell Lymphoma | DNA RNA and Cells... - December 8th, 2020
- Bayer and Atara Biotherapeutics in CAR T-cell therapy deal - BioPharma-Reporter.com - December 8th, 2020
- Repairing the Brain With Stem Cells? A Conversation With Prof. Jack Price - Being Patient - December 3rd, 2020
- Global Induced Pluripotent Stem Cells Market Size, Comprehensive Analysis, Development Strategy, Future Plans and Industry Growth with High CAGR by... - December 3rd, 2020
- Industry Verticals: Induced Pluripotent Stem Cells (iPSC) Market Latest and updated Scope and Application 2020-2027 - The Blend - December 3rd, 2020
- Bayer Launches Cell and Gene Therapy Platform to Maximize Recent Acquisitions - PharmaLive - December 3rd, 2020
- Stem Cells Market Size on Target to Reach US$ 17.79 Billion 2027 - Cheshire Media - December 3rd, 2020