Itâ€™s that time of year again! With 2021 behind us, weâ€™re going down memory lane to highlight biotech innovations that shaped the yearâ€”with impact that will likely reverberate for many years to come. Covid-19 dominated the news, but science didnâ€™t stand still.
Take gene editing. CRISPR spun off variations with breathtaking speed, expanding into a hefty toolbox packed with powerhouse gene editors far more efficient, reliable, and safer than their predecessors.Â CRISPRoff,Â for example, hijacks epigenetic processes to reversibly turn genes on and offâ€”all without actually snipping or damaging the gene itself. Prime editing, the nip-tuck of DNA editing that only snipsâ€”rather than fully cuttingâ€”DNAÂ received an upgradeÂ to precisely edit up to 10,000 DNA letters in a variety of cells.Â Twin prime editingÂ can rework entire genes. These powered-up CRISPR tools now make it possible to tackle previously untouchable genetic disorders.
Yet weâ€™re still only scratching the surface of gene editing. Peeking intoÂ the CRISPR family tree, scientists found a vast universe of alternative CRISPR-like systems to further explore. AI is now helping identify new CRISPR proteinsâ€”and theirÂ kill switch. Other ideas jumped ship from CRISPR altogether, tapping intoÂ another powerful bacterial systemÂ to edit millions of DNA sequences without breaking a single DNA strand. Without doubt, the gene editing toolbox will keep expanding.
In other news,Â quantum mechanics hooked up with neuroscienceÂ toÂ speed up AI. AI is nowÂ designing its ownÂ hardware chips at Google in an efficient full circle. Hopping into our own brains, in a stunning proof-of-concept, AI-powered brain implants were able to fightÂ depression, with ongoing work to treatÂ chronic painÂ and translate the brainâ€™s electrical signals fromÂ thought to text. In the medical world,Â a fierce debateÂ onÂ an Alzheimerâ€™s treatmentÂ sparked aÂ new roundÂ of alluring ideasÂ to tackle and tame our long-time mind-eating foe.
Thereâ€™s a ton more. But here are the top three advances thatâ€™ll keep reshaping biotech far past 2021, with some runners-up.
I know, I know. Weâ€™re all tired of hearing about Covid-19 and vaccines. Yet their remarkable ability to fight a completely novel infectious virus is â€œnothing short of miraculous.â€ It also showcased the power of the decades-old technology that previously languished in labs, with a platform thatâ€™s far faster, simpler, and more adaptable than any previous vaccine technology. Because they no longer rely on physical target proteins from a virusâ€”rather, just the genetic code for those proteinsâ€”designing a vaccine just requires a laptop and some ingenuity. â€œThe era of the digital vaccine is here,â€Â wroteÂ a team from GlaxoSmithKline.
To enthusiasts, mRNA vaccines could transform current treatments for a wealth of diseases, and the field is exploding. Moderna, for example, launchedÂ an HIV vaccineÂ humanÂ trial in August to begin assessing its safety, tackling a virus thatâ€™s escaped classic vaccine tactics for four decades. Along with the National Institutes of Health (NIH), the company also published data onÂ an HIV vaccineÂ candidate that lowered the chance of infection by nearly 80 percent in monkeys, with all subjects developing antibodies against 12 tested strains of HIV.Â Itâ€™s no small featâ€”the HIV target,Â Env, is a formidable target due to its complexity and is coated with a sugar armor to mask vaccine target points. The mRNA vaccine offers new hope.
Viruses aside, mRNA vaccines also represent a new solution to autoimmune or neurodegenerative diseases. BioNTech, the partner of Pfizer for developing Covid-19 vaccines, is applying the technology to tackle multiple sclerosis (MS). In MS, the immune system gradually strips away the insulation on nerve fibers, causing gradual and irreversible damage.Â Initial resultsÂ in mice are positive, with the approach â€œhighly flexible, fast, and cost efficient,â€ while potentially being personalized to each patient.
Further down the pipeline are mRNA vaccines that tackleÂ cancerÂ or those that deal withÂ antibiotic resistance. Whether the tech can solve some of our toughest diseases remains to be seen, but the field is on a roll.
In Vivo Gene Therapy
CRISPRâ€™s long been touted as a tool that can radically transform gene therapy.Â Earlier studiesÂ used the gene editing tool to bolster immune T-cells, transforming them into super soldiers that enhance their fight againstÂ blood cancersÂ (CAR-T therapy). The tool alsoÂ scored successesÂ in battling anemia and other symptoms in patients with blood disorders. The down side was that cells needed to be gene-edited outside the body and infused back into the bloodstream. This year elevated CRISPR to the ultimate goal: directly editing genes inside the body, opening the door to curing hundreds of disorders resulting from faulty genetic code.
In a breakthrough,Â one trialÂ from University College London edited a mutated gene in the liver that eventually leads to heart and nerve damage. Unlike previous attempts, here the CRISPR machinery was delivered into the bloodstream with a single infusion to switch the gene off, sharply decreasing the production of the mutant protein in six patients.Â Another trialÂ snipped a dysfunctional gene that causes blindness. By directly injecting the treatment into the retina, volunteers were able to better sense light.
Both are edge cases. For the liver trial, CRISPR was delivered using lipid nanoparticlesâ€”little fatty space shipsâ€”that have an affinity for the liver, with more transient gene-editing effects. And unlike the retina, most of our bodyâ€™s tissues arenâ€™t immediately accessible to a simple injection. But as proofs of concept, the trials finally bring CRISPR into a vast world of gene-editing possibilities inside the body. Along withÂ advances in delivery, CRISPRâ€”and its many upgradesâ€”is set to treat the untreatable.
An Unprecedented Look Into Human Development
The first few hours and days of a human embryoâ€™s development are a black boxâ€”one we need to crack. Understanding early pregnancy is key to limiting birth defects and pregnancy loss, and improving assistive reproduction technologies.
The problem? Early embryos are hard to come by, and carry significant ethical and legal challenges. This year,Â several studiesÂ circumvented these problems, instead transforming skin cells into blastocysts, a cellular structure that resembles the very first stage of a human embryo.
Torpedoing the usual â€œsperm meets eggâ€ narrative, the studies engineered the â€œfirst complete model of the human embryoâ€ using embryonic stem cells and skin cellsâ€”no reproductive cells needed. Bathed in a nutritious liquid, the cells developed into blastocysts, containing cell types that eventually lead to all lineages to build our bodies. The artificial embryos are genetically similar to natural ones, stirring up debate on how long they should be allowed to develop. The nightmare scenario? Imagine a mini-brain growing inside an embryo made out of skin cells!
For now thatâ€™s technically impossible, but the ethical quandary has stirred up concern at the International Society for Stem Cell Research (ISSCR), which governs research related to human stem cells and embryos. Yet surprisingly, this year,Â they relaxed the 14-day ruleÂ for culturing embryos, giving permission to push embryo research past two weeks. With relaxed guidelines, upcoming studies could reveal what happens to a human embryo after implanting into the uterus, andÂ gastrulationâ€”when genetic cues lay out the bodyâ€™s overall patterning and set the stage for organ development.
Itâ€™s a decisionÂ mired in controversy, but provides an unprecedented opportunity to revise IVF and, for the first time, examine the first stages of human development. Itâ€™s also bound to raise ethical quandaries: what if the embryosâ€”natural or artificialâ€”begin developing neurons that fire, or heart cells that pulse? As artificial blastocysts increasingly embody their biological counterparts, one thing is clear: with great power comes great responsibility.
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