Ginkgo grows its gene therapy offerings with StrideBio deal
Bio Pharma Dive
APRIL 5, 2023
The deal hands Ginkgo technology for discovering and engineering capsids — the outer shell that protects the helpful genetic material in gene therapies.
Engineer Gene Genetics 
Experiment Reveals Human Genes Can Be Controlled With Electricity
AuroBlog - Aurous Healthcare Clinical Trials blog
AUGUST 7, 2023
New research from ETH Zürich in Switzerland could see future wearable devices (with perhaps a few implants and a touch of genetic engineering) boost our health directly. Fitness trackers help you stay healthy by keeping count of your steps and monitoring your heart rate, driving you on to hit those cardio goals.
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Capsida Biotherapeutics and Prevail to develop CNS gene therapies
Pharmaceutical Technology
JANUARY 5, 2023
Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.
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ElevateBio raises funds to advance cell and gene therapies
Pharmaceutical Technology
MAY 25, 2023
ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.
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Biologists create new genetic systems to neutralize gene drives
Scienmag
SEPTEMBER 18, 2020
Two active genetics strategies help address concerns about gene-drive releases into the wild Credit: Ana Silva In the past decade, researchers have engineered an array of new tools that control the balance of genetic inheritance.
Genetics 86
86 
Ginkgo buys StrideBio’s AAV discovery and engineering platform assets
Pharmaceutical Technology
APRIL 6, 2023
Ginkgo Bioworks has strengthened its end-to-end R&D capabilities in gene therapy with the purchase of adeno-associated virus (AAV) capsid discovery and engineering assets from StrideBio, for an undisclosed sum. The new capabilities and IP will be incorporated under Ginkgo’s end-to-end AAV gene therapy development platform.
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Gene editing: beyond the hype
pharmaphorum
JANUARY 29, 2021
Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers.
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Asklepios partners with ReCode on gene-editing platform
Pharmaceutical Technology
JANUARY 10, 2023
Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP. By Cytiva Thematic.
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Opinion: Secrecy: A demon of gene therapy’s past bedevils its future
STAT News
JULY 11, 2022
Twenty-three years ago, the field of gene therapy was bursting with the promise of breakthrough treatments. Then it was almost instantly derailed by the death of an 18-year-old clinical trial volunteer named Jesse Gelsinger after he received a genetically engineered virus that had been developed to treat his rare liver condition.
Gene 111
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Scribe and Prevail partner for CRISPR-based genetic medicines
Pharmaceutical Technology
MAY 17, 2023
The deal will see the integration of Scribe’s new CRISPR by Design approach and Prevail’s expertise in developing genetic medicines for neurological disorders for specific genetic targets. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
Genetics 130
130 
How epilepsy researchers are moving the needle past anti-seizure treatments
Pharmaceutical Technology
FEBRUARY 13, 2023
Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. Meanwhile, others are researching the link between gene variation and different responses to treatments.
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CRISPR Pioneer Goes Small to Transform Genetic Medicine with Epigenetic Engineering
BioSpace
JULY 11, 2022
Epic Bio aims to transform genetic medicine by developing a new class of drugs that target the epigenome to alter gene expression.
Genetics 81
81 
Moderna partners with Life Edit for mRNA gene editing therapies
Pharmaceutical Technology
FEBRUARY 23, 2023
Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”
Gene Editing 147
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Leading innovators in IgG gene expressing animal models for the pharmaceutical industry
Pharmaceutical Technology
FEBRUARY 15, 2023
In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: IgG gene expressing animal models. To obtain human antibody responses within a mouse, mice are genetically engineered to be humanised for their Ig genes.
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Leading innovators in gene splicing using nucleases for the pharmaceutical industry
Pharmaceutical Technology
FEBRUARY 15, 2023
In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Gene splicing using nucleases. They are engineered to cut specific genomic targets in order to modify the expression of single genes and proteins.
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AbbVie and Capsida partner for gene therapies to treat eye diseases
Pharmaceutical Technology
FEBRUARY 24, 2023
AbbVie is expanding its strategic collaboration with Capsida Biotherapeutics for the development of targeted genetic medicines to treat eye diseases with high unmet needs. In pursuing the promise of genetic medicine-based therapeutics, AbbVie continues to expand our capabilities, and we are pleased to have Capsida as a partner.”
Gene Therapy 130
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Targovax announces plans to rebrand as Circio
Pharmaceutical Technology
APRIL 20, 2023
Circio aims to develop new circRNA medicines initially for cancer, then plans to expand rapidly into vaccines and gene therapy. The circVec platform is a modular genetic cassette that provides instructions for the generation of multifunctional circRNAs.
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Leading innovators in transcription factors for AAV for the pharmaceutical industry
Pharmaceutical Technology
JUNE 4, 2023
Innovation S-curve for the pharmaceutical industry Transcription factors for AAV is a key innovation area in pharmaceutical Adeno-associated virus (AAV) vectors are widely used for gene therapy. There are two main genes in the AAV genome, rep and cap, which encode nine different proteins.
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Synthetic SPECIES developed for use as a confinable gene drive
Scienmag
JUNE 2, 2021
Researchers create novel CRISPR-based fly species as a new method of controlling gene drive spread Credit: Akbari lab, UC San Diego CRISPR-based technologies offer enormous potential to benefit human health and safety, from disease eradication to fortified food supplies.
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STAT+: CRISPR patent fight redux? A new battle is brewing among biotechs over next-gen gene-editing tools
STAT News
MARCH 1, 2023
Genome editing summits are generally friendly, nerdy affairs, but for a moment at a Lisbon hotel last June, the conversation at the FASEB genome engineering conference grew tense. ” It was a rare moment: Tessera had raised around $600 million and passed a $1 billion valuation, but published little in academic medical journals.
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Biogen’s ambitions in gene therapy hit by another failed pivotal trial
pharmaphorum
JUNE 15, 2021
When Biogen acquired Nightstar Therapeutics in 2019, it added a pair inherited retinal disorder (IRD) gene therapies that it hoped would accelerate a push into the fast-evolving category. . The post Biogen’s ambitions in gene therapy hit by another failed pivotal trial appeared first on.
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Risk Assessment for use of Engineered Genetic Materials in Clinical Research
Advarra
MARCH 31, 2023
The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.
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Mapping Networks of Immune Genes Behind Autoimmune Diseases
XTalks
JULY 14, 2022
Every gene in a cell is expressed at exact levels due to complex gene regulatory networks. The connections between regulatory genes and their downstream targets have been mapped to some extent — they can be thought of as a subway or metro map, with connections existing between major “hubs.”
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Opinion: Needed: stricter screening of gene synthesis orders, customers
STAT News
OCTOBER 5, 2022
Scientists rely on gene synthesis technologies as a research tool for everything from basic research to vaccine development and drug target identification. Ever since the inception of gene synthesis, there have been concerns about possible misuse of synthetic genes. Read the rest…
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Enabling the next wave of innovative drug therapies with speciality enzymes
Pharmaceutical Technology
JUNE 29, 2022
The next wave of medicine is well on course to be cell and gene-based. For cell and gene therapy applications, you need a variety of speciality enzymes of the highest purity, specificity, and consistency. Novozymes has a long legacy of enzyme discovery and the ability to genetically engineer these speciality enzymes to be superior.
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Pfizer’s haemophilia B gene therapy meets phase 3 primary endpoint
pharmaphorum
JANUARY 3, 2023
As December 2022 closed out, Pfizer announced positive top-line results from its phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec (SPK-9001), its investigational gene therapy for treatment of adult males with moderately severe to severe haemophilia B.
Gene Therapy 98
98 
Combining genetic information with EMRs to pinpoint childhood epilepsies
Scienmag
AUGUST 11, 2020
Assessing clinical data across the lifespan identified the impact of genetic epilepsies on affected children’s lives Philadelphia, August 11, 2020 – A team of researchers at Children’s Hospital of Philadelphia (CHOP) affiliated with the CHOP Epilepsy Neurogenetics Initiative (ENGIN) further bridged the gap between genomic information (..)
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IBC vs. IRB: What’s the Difference?
Advarra
JUNE 12, 2023
Gene therapy research is exciting and full of promise, but because of the risks involved, it’s also highly regulated, requiring an institutional biosafety committee (IBC) to provide additional oversight and risk assessment. What Does an IBC Review? How is IBC Membership Composed?
STAT+: Pharmalittle: FDA approves a gene therapy for a rare blood disorder; pharmacy chains ordered to pay $650 million over opioid crisis
STAT News
AUGUST 18, 2022
Food and Drug Administration approved a new treatment that delivers a potentially permanent, genetic fix for patients with the inherited blood disorder beta thalassemia — and quite possibly a financial lifeline for its manufacturer, Bluebird Bio , STAT tells us. Zynteglo will cost $2.8 Continue to STAT+ to read the full story…
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Cancer immunotherapy approach targets common genetic alteration
Scienmag
MARCH 15, 2021
Engineered T cells can recognize and destroy cells with loss of one gene copy Credit: Elizabeth Cook Researchers developed a prototype for a new cancer immunotherapy that uses engineered T cells to target a genetic alteration common among all cancers.
Genetics 54
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Genetic eraser: Newly developed technology precisely and rapidly degrades targeted proteins
Scienmag
NOVEMBER 11, 2020
“Conditional gene knockout and small interfering RNA […]. Credit: Masato Kanemaki Researchers can now more accurately and precisely target specific proteins in yeast, mammalian cells and mice to study how knocking down specific protein traits can influence physical manifestation in a cell or organism.
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The Importance of Hazard Communications in Clinical Trials Involving Genetic Engineering
Advarra
JUNE 13, 2024
Recombinant DNA technologies and genetically modified biological agents are being adapted for a wide scope of therapeutic applications, and their use is becoming increasingly common in clinical trials. How can I protect myself from exposure? What should I do if I’m exposed?
Casting aside CRISPR scissors and making a point with base editors
pharmaphorum
FEBRUARY 3, 2021
Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.
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#news #biotech Genetic engineering without unwanted side effects helps fight parasites
BioTech 365
DECEMBER 23, 2020
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Genetic engineering without unwanted side effects helps fight parasites.Modified CRISPR-Cas9 gene editing scissors are enabling researchers at the University of Zurich to make alterations to the genetic … Continue reading (..)
Opinion: Where are the guidelines for the production of animals with intentional genomic alterations?
STAT News
APRIL 17, 2023
Genetic engineering has the potential to transform how we raise animals for meat and other products, making food safer, improving animal health and welfare, and shrinking animal agriculture’s environmental footprint. Pigs that are less likely to induce allergic reactions in humans.
Genomics 98
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New ‘split-drive’ system puts scientists in the (gene) driver seat
Scienmag
MARCH 5, 2021
Researchers develop tunable system that harnesses the spread of cargo carried by gene drives Credit: Gerard Terradas, UC San Diego Powerful new genetic engineering methods have given scientists the potential to revolutionize several sectors of global urgency.
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Singapore scientists develop novel gene editor to correct disease-causing mutations
Scienmag
MARCH 11, 2021
The CRISPR-based gene editor, C-to-G Base Editor (CGBE), opens up treatment avenues for up to 40 per cent of genetic disorders caused by single-nucleotide mutations Credit: Agency for Science, Technology and Research (A*STAR), Genome Institute of Singapore (GIS) A team of researchers from the Agency for Science, Technology and Research’s (A*STAR) (..)
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Sonoma signs deal to set up manufacturing centre for cell therapies
Pharmaceutical Technology
AUGUST 25, 2022
The centre, which will expand the company’s operations in Seattle, US, will aid in the R&D and manufacturing at scale its gene-modified regulatory T cell (T reg ) therapy pipeline for autoimmune and inflammatory ailments. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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Generating Over a Billion Cells with CRISPR for Next Generation Cell Therapies
XTalks
AUGUST 29, 2022
Now a common gene editing tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. Harnessing the Cellular Engineering Potential of CRISPR.
Gene Editing 98
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#news #biotech Biologists create new genetic systems to neutralize gene drives
BioTech 365
SEPTEMBER 18, 2020
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Biologists create new genetic systems to neutralize gene drives.In the past decade, researchers have engineered an array of new tools that control the balance of genetic inheritance.
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Ctexli (Chenodiol) Gains FDA Approval as First CTX Treatment
XTalks
FEBRUARY 25, 2025
CTX is a rare, progressive genetic disorder caused by mutations in the CYP27A1 gene, which disrupts the livers ability to produce chenodeoxycholic acid, a bile acid. Preclinical studies in a CTX mouse model demonstrated that VTX806 normalizes toxic bile acid metabolite levels in the blood, liver, tendons and brain.
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Scribe and Sanofi partner to develop cell therapies for cancer
Pharmaceutical Technology
SEPTEMBER 29, 2022
The alliance will leverage the CRISPR genome editing technologies of Scribe to facilitate in genetic modification of new natural killer (NK) cell therapeutics for cancer. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. According to the deal, Sanofi will make an upfront payment of $25m to Scribe.
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AstraZeneca pays record 660% premium for gene editing company LogicBio
pharmaphorum
OCTOBER 4, 2022
AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. The post AstraZeneca pays record 660% premium for gene editing company LogicBio appeared first on.
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Molecule holds promise to reprogram white blood cells for better cancer treatment
Scienmag
DECEMBER 13, 2020
In this type of therapy, T cells, a type of white blood cell, are collected from a patient’s blood and subjected to genetic engineering to produce T cells carrying a synthetic molecule termed chimeric antigen receptor (CAR) […].
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