BioTech Review

Nature is the foremost international weekly scientific journal in the world and is the flagship journal for Nature Portfolio. It publishes the finest peer-reviewed research in all fields of science and technology on the basis of its originality, importance, interdisciplinary interest, timeliness, accessibility, elegance and surprising conclusions. Nature publishes landmark papers, award winning news, leading comment and expert opinion on important, topical scientific news and events that enable readers to share the latest discoveries in science and evolve the discussion amongst the global scientific community.

When a neuron in our body gets damaged, segments of RNA produce proteins that can help repair the injury.

In a groundbreaking development that could revolutionize the therapeutic landscape for glioblastoma, researchers have unveiled a novel mechanism by which androgen receptor inhibition markedly sensitizes glioblastoma stem cells (GSCs) to temozolomide (TMZ), the current frontline chemotherapeutic agent for this aggressive brain tumor. This breakthrough not only sheds light on the intricate molecular interplay within glioblastoma […]

Researchers at Stanford University have unveiled a groundbreaking technology that might redefine the treatment of neurological disorders by enhancing the delivery of RNA molecules to specific locations within neurons. This novel approach, termed “spatial RNA medicine,” could promise revolutionary therapeutic advancements for conditions like amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy, as well as […]

A diverse group of experts is gathering in Cambridge, Massachusetts, to debate human heritable genome editing. To mark the event, is publishing 18 invited Perspectives from participating authors. The post Global Observatory Gathers to Expand Debate on Human Genome Editing appeared first on GEN - Genetic Engineering and Biotechnology News.

For some biotechs, the office is a place where scientists gather and pipelines advance. For others, it’s a boat anchor dragging them to the bottom of the sea. Four years into the biotech downturn, some ...

Nebraska has become the fourth state to ban lab-grown protein. On Tuesday, Governor Jim Pillen signed a bill into law that bans the production, sale, promotion and distribution in the state. While it doesn’t affect plant-based alternatives, the measure specifically targets proteins derived from animal cells. He says the long-term health impacts are unknown, and […] The post Pillen signs bill banning sale of lab grown protein appeared first on Brownfield Ag News.

India’s Genome-Edited Rice Set to Change Farming Forever! We live in a country where rice/paddy fields stretch endlessly across the landscape, providing food to millions of people who depend on this staple for their survival. Rice isn’t just a crop; it is the lifeblood of India. Yet, it deals with daunting crises such as water […] The post From Water Crisis to Crop Miracle: India’s Genome-Edited Rice Set to Change Farming Forever! appeared first on BioTecNika.

Using CRISPR and stem cell–derived neurons, researchers identified early mitochondrial dysfunction shared across ALS-linked mutations—pointing to common disease pathways and potential therapeutic targets, long before traditional signs of neurodegeneration appear. The post Potential ALS Mitochondrial Target Identified with CRISPR, scRNA-seq appeared first on GEN - Genetic Engineering and Biotechnology News.

CRISPR Therapeutics AG (NASDAQ:CRSP) has entered a $95 million upfront partnership with China-based Sirius Therapeutics to co-develop an siRNA drug targeting blood clotting disorders. This marks a strategic shift for CRISPR, traditionally focused on gene editing, recognizing that siRNA’s reversible gene silencing offers advantages for some conditions where permanent DNA edits are risky, such as […]

Researchers have used a personalised base-editing gene therapy to successfully treat an infant with carbamoyl-phosphate synthetase 1 (CPS1) deficiency, a rare and life-threatening inborn error of metabolism. This pioneering approach chemically corrects the underlying mutation without cutting DNA, showing promise for precision treatment of ultrarare genetic disorders.

Using the gene scissors CRISPR and stem cells, researchers at Stockholm University and the UK Dementia Research Institute (UK DRI) at King's College London have managed to identify a common denominator for different gene mutations that all cause the neurological disease amyotrophic lateral sclerosis (ALS). The research shows that ALS-linked dysfunction occurs in the energy factories of nerve cells, the mitochondria, before the cells show other signs of disease, which was not previously known. The study was recently published in the journal Nature Communications.

The gene editing company CRISPR Therapeutics struck a deal worth $95 million upfront with China-based biotech Sirius Therapeutics to jointly develop its experimental gene-silencing siRNA drug for blood clotting disorders. The

Discover how scientists essentially added a guide molecule to the gene editor to help it insert an entire gene into the most effective place.

Prime Medicine, known for its goal of treating hundreds of diseases with CRISPR, says it's narrowing its focus to two. It also announced big layoffs and a new CEO.

What has happened?US doctors have developed and safely delivered a customised gene editing therapy to treat a baby boy with carbamoyl phosphate synthetase 1 (CPS1) deficiency, diagnosed soon after birth. The condition, which affects only one in 1.3 million people, is characterised by an inability to fully break down byproducts from protein metabolism in the liver. This causes a build up of ammonia to toxic levels that can cause severe damage to the liver and brain. Treatment includes a low protein diet until the child is old enough to receive a liver transplant. However, during the waiting period there is a risk of rapid organ failure. The disorder is estimated to kill about half of those children affected in early infancy.What is CRISPR?CRISPR (clustered regularly interspaced short palindromic repeats) is a technology that allows precise changes to DNA inside living cells. A team at the Children’s Hospital of Philadelphia and...

Intellia’s CRISPR-based therapy maintained reductions in the levels of a misfolded protein in the nerves of patients with a rare disease called transthyretin amyloidosis. Functional improvement was also seen in the patients. The data come ...

Researchers have devised another way to insert large genes into precise spots in human cells, adding a serious contender to the increasingly heated race of technologies broadly known as targeted gene insertion ...

The new CRISPR system uses laboratory engineered versions of transposases from Pseudoalteromonas bacteria that were evolved over several generations to efficiently insert copies of healthy genes into human cells at therapeutically useful levels. The post EvoCAST Harnesses CRISPR-Linked Bacterial Transposes to Insert Genes Into Human Cells appeared first on GEN - Genetic Engineering and Biotechnology News.

WEST LAFAYETTE, Ind. — Farmers have been improving the genetics of their animals for thousands of years through selective breeding. Wild boars were bred until they lost their tusks and gained fattier meat. Breeding cattle...

A new gene editor may soon open the door to gene therapies for a wider array of diseases.

Researchers have developed a method to confidently produce blood cell precursors from stem cells in mice, by activating a set of seven key genes in the laboratory. The team takes a step forward towards the production of precursor cells able to restore the bone marrow of blood cancer patients, in a successful example of regenerative medicine.

Newly discovered weapons of bacterial self-defense take different approaches to achieving the same goal: preventing a virus from spreading through the bacterial population.

Baby KJ's bleak future is brighter as CRISPR breakthrough paves the way to treat rare genetic disorders.

A CRISPR treatment seems to have been effective for a baby’s devastating disease, but it is not clear whether such bespoke therapies can be widely applied

The N-of-1 accomplishment provides a template for swift, personalized genetic therapies.

By the second day of his life, Kyle and Nicole Muldoon knew their newborn son was very sick.During a routine exam in the NICU, his doctor noticed his arm was shaking down slowly after being lifted, rather than falling like a typical baby. Then, his labs showed extremely high levels of ammonia in his blood, indicating that he had a urea cycle disorder.Kyle and Nicole said the following weeks were a “crash course” in urea cycle deficiencies and their son’s specific condition: carbamoyl phosphate synthetase 1 (CPS1) deficiency.The infant’s doctor explained that CPS1

KJ was born with a metabolic disorder that kills up to 50% of infants.

FRIDAY, May 16, 2025 — A baby born with a rare and deadly genetic disease is the world's first known patient to receive an experimental gene editing treatment designed just for him.KJ Muldoon from Clifton Heights, Pa., is thriving after the therapy...

Every living creature on Earth needs to protect itself from things that would do it harm. Bacteria are no different. And despite their relative simplicity, they deploy remarkably savvy defensive strategies against viral invaders. The most well-known is CRISPR-Cas9, adapted for human use as the first FDA-approved genetic editing technique.

Stem cells can produce any other cell type, it is just a matter of telling them in the right way. From a biological perspective, this means activating the proper genetic program by pressing the right keys, that is, the right genes, at the right moment. Quite often, blood cancer patients require the replacement of their blood stem cells in the bone marrow, the tissue producing blood cells where their cancer grows.

This is today’s edition of The Download, our weekday newsletter that provides a daily dose of what’s going on in the world of technology. This baby boy was treated with the first personalized gene-editing drug Doctors say they constructed a bespoke gene-editing treatment in less than seven months and used it to treat a baby with a…

Ask scientists what gene editing tool is most needed to advance gene therapy, and they'd probably describe a system that's now close to realization in the labs of Samuel Sternberg at Columbia University Vagelos College of Physicians and Surgeons and David Liu at the Broad Institute of MIT and Harvard.

An infant with a severe genetic condition has shown signs of improvement after receiving a gene-editing treatment tailored to his specific mutation

In a groundbreaking development in the field of gene therapy, researchers have unveiled the evoCAST gene editor, an innovative tool designed to enhance the precision and effectiveness of gene editing. This revolutionary system, being worked on in the laboratories of Samuel Sternberg at Columbia University and David Liu at the Broad Institute of MIT and […]

At a gene therapy conference, there was excitement about the first correction of a genetic misspelling, along with questions about Sarepta and Editas data.

Ask scientists which gene-editing tool is most needed to advance gene therapy, and they'd probably describe a system that's now close to realization in the labs of Samuel Sternberg at Columbia University Vagelos College of Physicians and Surgeons and David Liu at the Broad Institute of MIT and Harvard.

A new gene editor takes advantage of CRISPR-associated proteins to insert whole genes into the genome, scientists report.

A baby known as KJ is the first person in the world to receive a customized CRISPR therapy designed to fix a specific mutation.

A team of doctors and scientists have successfully treated a rare genetic condition with the first-ever personalized gene-editing therapy. Results of the groundbreaking treatment have been published in The New England Journal of Medicine, with an accompanying editorial by a doctor who had previously overseen the FDA's gene-therapy regulation efforts. The patient in this historic case was KJ, an infant born with CPS1 deficiency, which has about a 50 percent mortality rate within the first week. Patients that do survive can experience severe brain disease, mental and developmental delays, and potential liver transplants. His care team developed a personalized gene-editing treatment based on CRISPR, a technology for modifying human DNA. The successful gene repair for KJ combined years' worth of previous federally-funded medical research, including the discovery of CRISPR and human genome sequencing that allowed the mutation to be identified. This approach to gene editing could

The gene-editing treatment was designed specifically for the infant to correct his liver defect.

The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other uncommon genetic diseases. The post Baby is healed with world’s first personalized gene-editing treatment appeared first on Boston.com.

In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of Philadelphia (CHOP) and Penn Medicine.

Custom CRISPR paves the way for treating genetic disorders in tailormade ways

An infant patient with a severe urea cycle disorder shows strong signs of improvement after receiving an individualized gene editing therapy that corrects mutations directly on the genome. The post ASGCT 2025: World’s First Patient Treated with Personalized CRISPR Therapy appeared first on GEN - Genetic Engineering and Biotechnology News.

What circumstances might justify deliberate, full extinction of a species?

Programmable gene integration in human cells has the potential to enable mutation-agnostic treatments for loss-of-function genetic diseases and facilitate many applications in the life sciences. CRISPR-associated transposases (CASTs) catalyze RNA-guided ...

In a landmark advancement poised to redefine the landscape of precision medicine, researchers at the Children’s Hospital of Philadelphia (CHOP) in collaboration with Penn Medicine have achieved a groundbreaking milestone in treating a rare metabolic disorder using bespoke CRISPR-based gene editing therapy. This pioneering intervention involved an infant named KJ, diagnosed with severe carbamoyl phosphate […]

Scientists at the McGovern Institute and the Broad Institute of MIT and Harvard have reengineered a compact RNA-guided enzyme they found in bacteria into an efficient, programmable editor of human DNA. The engineered protein, called NovaIscB, can be adapted to make precise changes to the genetic code, modulate the activity of specific genes, or carry out other editing tasks. Because its small size simplifies delivery to cells, NovaIscB's developers say it is a promising candidate for developing gene therapies to treat or prevent disease.

Doctors say they constructed a bespoke gene-editing treatment in less than seven months and used it to treat a baby with a deadly metabolic condition. The rapid-fire attempt to rewrite the child’s DNA marks the first time gene editing has been tailored to treat a single individual, according to a report published in the New…

A gene editing drug custom-made for a critically ill baby showed that, for some ultra-rare diseases, it’s possible to design and test a new CRISPR medicine in just a few months. 

The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other uncommon genetic diseases.

The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other uncommon genetic diseases.

PHILADELPHIA — Soon after Kyle Patrick Muldoon, Jr. was born in August 2024, his doctors knew something was wrong. KJ, as his parents call him, was too tired to feed, his ...

In a historic medical breakthrough, a child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy by a team at Children's Hospital of Philadelphia (CHOP) and Penn Medicine.

The technique used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other uncommon genetic diseases.

In a world first, scientists used CRISPR to fix a baby's unique genetic mutation. The report offers hope for personalized treatments of rare diseases.

Spearhead Bio’s TAHITI platform merges CRISPR/Cas gene editing technology with engineered transposable elements to achieve precise gene insertion in high-value agricultural crops. The post SpearHead Bio Offers Precise Plant Engineering Using CRISPR-Directed Jumping Genes appeared first on GEN - Genetic Engineering and Biotechnology News.

An MP is urging the UK government to protect 'hard-won' gene editing rules from being watered down in post-Brexit talks with the EU. Form...

Governor Greg Gianforte recently signed into law a bill to ban the manufacturing and sale of lab-grown meat in Montana. “If you’ve ever had the pleasure of enjoying a cut…

Current setbacks cannot prevent gene-editing companies from advancing closer to “one-and-done” cures and new products. The post Gene Editing Marches On Despite Missed Beats appeared first on GEN - Genetic Engineering and Biotechnology News.

The mood at the annual meeting of the American Society of Cell and Gene Therapy isn’t amazing. The biotech market has been bad for years. But many researchers tried to…

In this Revvity GENCast episode 2, experts will discuss applications of gene editing to improve scientists’ understanding of the relationship between variant and disease in functional genomics studies. They will also talk about the Dharmacon product suite which is designed for CRISPR-based targeted editing in a wide range of cells. The post From Functional Genomics to Cell Therapy: The Role of CRISPR-based Gene Editing appeared first on GEN - Genetic Engineering and Biotechnology News.

Leading trade organizations representing the makers of cell and gene therapies are calling for a 10-year moratorium on germline gene editing.

An order from a federal appeals court has kicked the battle wide open again over who holds the key patents for CRISPR-Cas9, a revolutionary gene editing technology. The US Court of Appeals for the Federal ...

LOUIS, MO. – The Danforth Technology Company (DTC) today announced the launch of Spearhead Bio, a startup pioneering a breakthrough platform to enhance the efficiency of crop genome engineering by harnessing the plant’s own natural DNA. Spearhead’s Transposase Assisted Homology Independent Targeted Insertion (TAHITI) technology allows the seamless integration of genes into both transgenic and […] The post Danforth Technology Company Launches Genome Editing Startup appeared first on Morning Ag Clips.

A joint research team affiliated with UNIST has developed a 3D-printed artificial tumor tissue capable of replicating the in vivo conditions of patient-derived cancer cells. This innovative model not only simulates the tumor microenvironment but also integrates artificial intelligence (AI) technology that can predict patient prognosis solely from images of tumor growth.

This is today’s edition of The Download, our weekday newsletter that provides a daily dose of what’s going on in the world of technology. A US court just put ownership of CRISPR back in play The CRISPR patents are back in play. Yesterday, the US Court of Appeals for the Federal Circuit said scientists Jennifer Doudna and…

The CRISPR patents are back in play. On Monday, the US Court of Appeals for the Federal Circuit said scientists Jennifer Doudna and Emmanuelle Charpentier will get another chance to show they ought to own the key patents on what many consider the defining biotechnology invention of the 21st century. The pair shared a 2020…

A federal appeals court said the patent office made legal errors in awarding foundational patents for CRISPR-Cas9 to the Broad Institute

The chief operating officer at Genus PIC says pigs produced to be resistant to Porcine Reproductive and Respiratory Syndrome (PRRS) have been approved for sale to U.S. consumers by the Food and Drug Administration. Matt Culbertson tells Brownfield it’s the latest step in bringing gene-edited pigs to market. “It doesn’t actually trigger the first sales […] The post Gene-edited pigs approved for U.S. market appeared first on Brownfield Ag News.

Researchers at Novartis BioMedical Research in the US and Switzerland have identified a molecular target that may provide a new therapeutic pathway for cancers driven by NRAS mutations. Findings suggest that interfering with specific protein interactions could disrupt signaling pathways associated with uncontrolled cell proliferation in malignancies lacking targeted treatments.

Stylus Medicine, an ambitious startup founded by Patrick Hsu, has raised $85 million to develop a suite of technologies that it believes will help solve some of the biggest and most competitive ...

WEST LAFAYETTE, Ind. — Farmers have been improving the genetics of their animals for thousands of years through selective breeding. Wild boars were bred until they lost their tusks and gained fattier meat. Breeding cattle were chosen for favorable traits like muscularity or high milk production. At the same time, advances in genomics and biotechnology […] The post Food Products from Animals with Heritable Genetic Modifications appeared first on Morning Ag Clips.

In a groundbreaking advancement set to reshape regenerative medicine, a team of researchers led by Li, W., Shi, Z., Jing, H., and colleagues have developed a novel metal-based hydrogel that dramatically enhances stem cell differentiation and supports extracellular matrix homeostasis, ultimately facilitating effective cartilage repair. This pioneering study, recently published in Nature Communications, elucidates the […]

University of Manchester scientists have successfully pioneered a way to create functioning human spinal disks, aiming to revolutionize our understanding of back pain and disk degeneration in a leap for medical science.

An ultrasound-guided 3D printing technique could make it possible to fabricate medical implants in vivo and deliver tailored therapies to tissues deep inside the body—all without invasive surgery. The post In Vivo Bioprinting Shows Promise for 3D Printed Implants Without Surgery appeared first on GEN - Genetic Engineering and Biotechnology News.

Dental caries (tooth decay) is a common oral health condition that often causes significant pain and discomfort and may even lead to tooth loss. In severe and untreated cases, bacterial infection combined with the host's immune response can cause bone resorption, or the breakdown of bone tissue in the tooth root. Moreover, traditional treatments for advanced dental caries, such as surgery, can result in bone defects that require complex bone grafting procedures.

A groundbreaking advancement in biomedical engineering has emerged with the development of an innovative ultrasound-guided 3D printing technique that enables the fabrication of medical implants directly within living tissues. This cutting-edge method promises a transformation in personalized medicine by allowing for the creation of custom-tailored therapies delivered precisely to targeted tissue sites deep inside the […]

Across hundreds of published papers, CBER’s new chief has argued for more stringent measures of assessing drugs for cancer and rare diseases, suggesting he might push for higher standards in the review of medicines under his division's purview.

Fyodor Urnov had given a version of his speech dozens of times before. Yet as he leaned into the microphone to address the FDA’s top regulators late last year, he felt his heart racing. The ...

The University of Bayreuth's Biomaterials research group has, for the first time, successfully applied the CRISPR-Cas9 gene-editing tool to spiders. Following the genetic modification, the spiders produced red fluorescent silk.

Early phase data from human trials of a single, low-dose gene editing therapy using CRISPR-Cas12 proves promising for improving DMD symptoms with no adverse effects, scientists say. The post ASGCT 2025: CRISPR-Cas12 Editing Shows Early Clinical Benefit in Duchenne Muscular Dystrophy appeared first on GEN - Genetic Engineering and Biotechnology News.

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A UK parliamentary committee has greenlit gene-edited plants. This is great news, as it will boost food production and reduce waste, says Michael Le Page

CRISPR Therapeutics has revealed promising but early data from its first clinical study of a gene editing therapy to lower the risk of heart disease, setting the stage for competition with other drugmakers working on ...

A breakthrough in cultivated meat research could accelerate the production of realistic, animal-free meat alternatives, British scientists s...

Base-editing technologies along with AAV engineering not only help reduce costs but can also accelerate preclinical therapeutic development. The post Precise and Flexible Gene Editing and Delivery Tools appeared first on GEN - Genetic Engineering and Biotechnology News.

Yale researchers have built a 3D-bioprinted synthetic aorta that they have successfully implanted into rats. This technology could advance the treatment of cardiovascular diseases such as coronary artery disease or peripheral arterial disease by allowing scientists to engineer and replace blood vessels in humans.

Pigs produced to be resistant to one of the world’s most costly livestock diseases, using technology developed by the Roslin Institute, have been approved for sale to US consumers. The US Food and Drug Administration has approved the use of a gene-editing technology that makes pigs resistant to Porcine Reproductive and Respiratory Syndrome (PRRS) for … The post Gene-Edited Pigs Approved for US Market appeared first on California Ag Network.

The authors of this review paper have a positive view of the future of regenerative medicine built on the ability to generate induced pluripotent stem cells from any patient cell sample. That should be tempered by a realistic expectation on timelines. At this point almost two decades have passed since the discovery of the first approach to reprogramming adult cells into induced pluripotent stem cells, but relatively little progress has been made on bringing therapies into even initial clinical trials. Perhaps the biggest challenge is that working with cells is very expensive and very challenging, far more so than development of small molecule drugs. Higher costs means fewer programs, slower progress. Aging-related diseases often involve the dysfunction or loss of specific cell types, leading to […]

Although immunotherapeutic strategies have made huge strides in treatment for solid tumors, the promising results have not moved over into the treatment of gastrointestinal cancer. Now, a first-in-human clinical trial shows encouraging signs of safety and potential effectiveness of a CRISPR/Cas9 gene-editing technique to help TILs fight advanced colorectal cancer. The post CRISPR-Edited TILs Fight Advanced Colorectal Cancer in Patients appeared first on GEN - Genetic Engineering and Biotechnology News.