Tag: cas9

Visiongain Reports Ltd Visiongain has published a new report entitled: Translational Regenerative Medicine Market Report 2024-2034: Forecasts by Product (Stem Cell Therapy (Autologous, Allogenic), Tissue Engineering (Scaffold, Hydrogels), Gene Therapy, Others)), by Application (Oncology, Dermatology, Musculoskeletal, Neurology, Cardiovascular, Wound Healing, Ophthalmology, Others) AND Regional and Leading National Market Analysis PLUS…

Visiongain has published a new report entitled: Translational Regenerative Medicine Market Report 2024-2034: Forecasts by Product (Stem Cell Therapy (Autologous, Allogenic), Tissue Engineering (Scaffold, Hydrogels), Gene Therapy, Others)), by Application (Oncology, Dermatology, Musculoskeletal, Neurology, Cardiovascular, Wound Healing, Ophthalmology, Others) AND Regional and Leading National Market Analysis PLUS Analysis of Leading…

For this year’s women and girls in science day, UAR chose to highlight the career of a world-renowned scientist and how animal research shaped her path to becoming one of the most respected and revered researchers. Working in fundamental science and microbiology, Emmanuelle Charpentier gradually obtained prizes, eventually receiving one…

Abstract CRISPR-based high-throughput genome-wide loss-of function screens are a valuable approach to functional genetics and strain engineering. The yeast Komagataella phaffii is a host of particular interest in the biopharmaceutical industry and as a metabolic engineering host for proteins and metabolites. Here, we design and validate a highly active 6-fold…

Company Logo Global Genome Editing Market Global Genome Editing Market Dublin, Feb. 09, 2024 (GLOBE NEWSWIRE) — The “Global Genome Editing Market, 2023-2035: Focus on Technology, Industry Trends and Forecasts” report has been added to ResearchAndMarkets.com‘s offering. The global genome editing market size for technologies is estimated to be worth…

Shondra Pruett-Miller, a biochemist at St. Jude Children’s Research Hospital, found her calling during her first year of graduate school at the University of Texas, Southwestern. In 2003, during the departmental faculty research talks, she watched with excitement as Matthew Porteus, now a biochemist at Stanford University, presented his research…

The Science Newswise — CRISPR-Cas is a tool that allows scientists to make targeted changes to an organism’s DNA. This tool consists of two parts. The first is a microscopic substance called a nuclease Cas protein (e.g., Cas9) that can cleave DNA. The second is an RNA molecule (also called…

Product information “ZsGreen/Luciferase Safe-Harbor HEK293 Cell Line” Recombinant stable HEK293 cell line constitutively expressing ZsGreen (bright green fluorescent protein derived from Zoanthus sp. reef coral) and firefly luciferase, which have been stably integrated into the AAVS1 safe harbor locus on chromosome 19 using CRISPR/Cas9. Expression of ZsGreen and the luciferase…

Precision Therapies For Targeted Results Precision therapies have always been the ultimate goal of medicine. Instead of drugs acting on multiple parts of the body, these therapies would only target one organ, one cell, or even one gene. In theory, not only would this be a lot more efficient, but…

Drugs like PARP inhibitors and chimeric antigen receptor (CAR) therapies, and advancements in artificial intelligence in drug discovery, have spurred hope for improved cancer care in the past year. Rigorous research has paved the way for a declining cancer death rate – by 33% to be precise – since the…

At the end of 2023, the FDA approved Casgevy for sickle cell disease, its first approval for a therapeutic that used the genome editing tool clustered regulatory interspaced palindromic repeats (CRISPR) to specifically inactivate a human gene as a treatment for a genetic disease. While CRISPR-based gene therapies could potentially…

Gene editing technologies, particularly those based on Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR), have revolutionized our ability to accurately and swiftly detect pathogens. However, even these advanced methods have their limitations. This article explores these limitations and highlights recent advancements that are paving the way for faster, more accurate…

CRISPR-Cas (clustered regularly interspaced short palindromic repeats-CRISPR-associated protein) genome editing enables precise, targeted genetic modification of plants. To date, the genomes of several crops, including rice, tomato, corn, wheat, soybean, barley, potato, sorghum, apple, grapefruit, and orange, have been edited using CRISPR-Cas-based genome editing technology. The CRISPR system cuts not…

While cancer mortality continues to decline, the number of new cases is projected to top 2 million in 2024, according to the American Cancer Society’s annual report on cancer statistics. The FDA approved exagamglogene autotemcel (Casgevy) for transfusion-dependent beta thalassemia in patients 12 years and older, maker Vertex announced. This…

Using CRISPR, an immune system bacteria use to protect themselves from viruses, scientists have harnessed the power to edit genetic information within cells. In fact, the first CRISPR-based therapeutic was recently approved by the FDA to treat sickle cell disease in December 2023. That therapy is based on a highly…

 Cornell researchers have taken an important step toward harnessing CRISPR gene editing in “targeted, safe and potent” cancer treatment, according to Ailong Ke, professor of chemistry and chemical biology in the College of Arts and Sciences. The enzyme CRISPR-Cas3, one of many different CRISPR systems found naturally in the immune systems…

Capturing proteins associated with SARS-CoV-2 virions To identify host cell proteins potentially present in extracellular SARS-CoV-2 virions, viral particles were produced from two lung epithelial cell models, A549 cells overexpressing ACE2 receptor (A549-ACE2) and Calu-3 cells that naturally express ACE2 receptor and TMPRSS2. Following infection with the D614G SARS-CoV-2 strain32,…

With the advent and advancements in nanotechnology, the field of cancer therapy is witnessing a paradigm shift. Nanoparticles, owing to their unique properties, are proving to be promising carriers for drug delivery in cancer therapy. The study scrutinizes the transport of nanoparticles in two abnormal microenvironments through a 2-D simulation…

Option 2: Genetics is a rapidly evolving area of science. Each year, advances in genetics bring exciting new technologies to the market. Areas such as forensics, genealogy, and healthcare have all been affected by new genetic technologies. Choose a genetic technology and report on how this technology is affecting or…

Using CRISPR, an immune system bacteria use to protect themselves from viruses, scientists have harnessed the power to edit genetic information within cells. In fact, the first CRISPR-based therapeutic was recently approved by the FDA to treat sickle cell disease in December 2023. That therapy is based on a highly…

When CASGEVY, the first CRISPR-based therapy, was approved by the FDA in December, it gave CRISPR technology the validation it needed. Any promise it had held before that moment was simply investigational. In this case, the approval of CASGEVY was granted for the treatment of two blood disorders – sickle…

Photo by Sangharsh Lohakare on Unsplash In the landscape of scientific advancement, the emergence of CRISPR technology has propelled humanity into a new era of genetic possibilities. While the revolutionary gene-editing tool offers unprecedented opportunities to reshape the blueprint of life, it also raises profound ethical dilemmas that echo through…

Expression pattern of miR-195 during embryonic development We analyzed the expression pattern of miR-195 by in situ hybridization at two distinct developmental stages in mouse embryos. Whole-mount in situ hybridization at E10.5, demonstrated a wide expression of miR-195 along the entire embryo, including the central nervous system, the cardiopharyngeal area,…

Key Points bluebird bio has three FDA-approved gene therapy treatments that are planning to commercialize after its $150 million secondary offering of common shares priced at $1.50 per share. bluebird bio’s latest FDA approval, LYFGENIA, is a one-time treatment for sickle cell disease, which is priced around $3.1 million per…

Unveiling Cancer’s Genetic Mechanisms: Role of Sox2 Gene Enhancers In a groundbreaking study led by Professor Jennifer Mitchell at the University of Toronto’s Department of Cell and Systems Biology, vital insights were garnered on the role of ‘enhancer’ regions in gene expression and their implications for cancer growth. Enhancers, non-coding…

(MENAFN– PR Newswire) DUBLIN, Jan. 8, 2024 /PRNewswire/ — The “Genome Editing Market – Global Industry Size, Share, Trends, Opportunity, and Forecast, 2018-2028F” report has been added to ResearchAndMarkets’s offering. The transformative power of genome editing continues to spur significant growth in the biotechnology sector, with the Global Genome Editing…

ZUG, Switzerland and BOSTON, Jan. 08, 2024 (GLOBE NEWSWIRE) — CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today highlighted its strategic priorities and 2024 outlook as the Company enters its next phase of growth. “We had a landmark year in 2023, marked…

The OECD recently published an update on key biotechnology indicators, an opportunity for us to delve into the top-performing countries in the field. With the highest number of active biotech companies (2,840) and an R&D intensity added value of 17%, the U.S. remains a strong leader in the industry. But…

CRISPR guide RNA libraries have been iteratively improved to provide increasingly efficient reagents, although their large size is a barrier for many applications. We design an optimised minimal genome-wide human CRISPR-Cas9 library (MinLibCas9) by mining existing large-scale gene loss-of-function datasets, resulting in a greater than 42% reduction in size compared…

Market Overview The Middle East and Africa (MEA) region is witnessing significant advancements in the field of regenerative medicine, particularly in the area of induced pluripotent stem cells (iPSCs). iPSCs are derived from adult cells and possess the remarkable ability to differentiate into various cell types, making them a valuable…

Rommie Amaro Ivet Bahar Jennifer Doudna The Biophysical Society has named seven 2024 society fellows, and three of them are members of the American Society for Biochemistry and Molecular Biology: Rommie Amaro, Ivet Bahar and Jennifer Doudna. Amaro is a professor and endowed chair in the molecular biology department and…

The year 2023 has been marked by significant advancements and breakthroughs in science and technology. From AI and quantum computing to genomic medicine and renewable energy, emerging technologies have reshaped various industries. With trends like the metaverse and advancements in space exploration, the future looks promising for continued progress and…

Abstract In mammals, lipoprotein lipase (LPL) has been found to play an important role in lipid mentalism and deposition. LPL deficiency in humans (Homo sapiens) and mice (Mus musculus) tends to cause hypertriglyceridemia. The lpl gene is not expressed in adult mammalian liver, but is in adult fish liver. The…

Arabidopsis contains hundreds of ribosomal DNA copies organized within the nucleolar organizing regions (NORs) in chromosomes 2 and 4. There are four major types of variants of rDNA, VAR1–4, based on the polymorphisms of 3’ external transcribed sequences. The variants are known to be differentially expressed during plant development. We…

Cancer is one of the leading causes of disease-associated mortality, with a rising incidence worldwide [1]. Although many therapeutic methods have been used in cancer treatment, such as chemotherapy, radiotherapy, surgery, and targeted therapy, the overall therapeutic outcome remains unsatisfactory. Therefore, developing new therapeutic means is urgently needed. With advances…

Prof. Eugene Koonin, one of the world’s leading evolutionary biologists, recently visited Israel as a gesture of solidarity and even took the time to volunteer in the Gaza border communities that came under attack on October 7.   Koonin is among the scientists who discovered the CRISPR mechanism, the hallmark…

Two new gene therapies to treat sickle cell disease were approved by the U.S. Food and Drug Administration Dec. 8. The treatments, both produced by Boston-area companies, join 32 other approved gene therapies and are approved to treat the disease in the United States. Experts say the developments are a…

MAVS maintains mitochondrial homeostasis and regulates cellular senescence through stabilizing OPA1. A. Depletion of MAVS accelerates cellular senescence of human mesenchymal stem cells. B. MAVS deficiency compromises mitochondrial dynamics. C. A model illustrating the MAVS-OPA1 axis regulates mitochondrial homeostasis and human stem cell senescence. Credit: Research A recent study led…

Human tissue samples The preoperative diagnosis of CS mainly relies on stool microscopy, enzyme-linked immunosorbent assay, or endoscopy. Fresh liver and intrahepatic bile duct specimens were collected from ICC patients with (CS-ICC group) and without (NC-ICC group) CS infection during partial hepatectomy at the First Hospital of Jilin University. Tumor tissues…

DNA sequencing I. Goal II. Methodology A. Sanger DNA sequencing ● Up to 900 base pairs ● Primer extension with labeled ddNTPs (radioactive or fluorescent) ● Prior knowledge on target needed ● Difficult to detect variation in mixtures ● High accuracy + still in use for genotyping When it is…

Charles River Laboratories International, Inc. CRL recently achieved an important milestone in its strategic collaboration with Vertex Pharmaceuticals to manufacture CASGEVY (exagamglogene autotemcel [exa-cel]). The company’s Memphis facility has been approved to manufacture Vertex’s CASGEVY — the first-ever gene-edited therapy in the world that targets severe sickle cell disease (SCD)….

MANHATTAN, Kan. (WIBW) – Kansas State University researchers have recently discovered ways to keep those with gluten allergies safer. Researchers have reported a breakthrough in developing wheat-based foods that contain lower amounts of gluten, a discovery that may lessen the adverse effects for those with celiac or other autoimmune diseases….

Share Tweet Share Share Email Among the various branches of biotechnology, genetic engineering stands out as a revolutionary field that holds immense potential for transforming healthcare as we know it. This article explores the latest biotechnological breakthroughs, focusing on how genetic engineering is…

Rahul Kakkar, MD, Tome Biosciences president and CEO While the CRISPR community celebrates the recent first FDA approval of a CRISPR-Cas9 therapy (Casgevy), new entities are making strides in their efforts to write perhaps the definitive new chapter in programmable genome editing. Last week, Tome Biosciences—a developer of genome editing…

CRISPR/Cas9-induced homozygous knockout of nexn causes progressive cardiac dysfunction without affecting skeletal muscle function in zebrafish Several studies in different animal models and cardiomyopathy patients have shown that loss of NEXN is leading to DCM which is characterized by an impaired contractility of the heart6,10,12. Homozygous loss of NEXN mostly…

Small-cap stocks are often attractive investments in bullish markets. While a bull market in 2024 is not guaranteed, some small caps are already warming up in anticipation of better economic conditions next year. As a result, it may be a smart move to start adding some select small caps to…

Visiongain has published a new report entitled Viral Vectors & Plasmid DNA Manufacturing Market Report 2024-2034: Forecasts by Vector Type (Adenovirus, Retrovirus, Plasmid DNA, AAV, Lentivirus, Others), by Application (Antisense and RNAi, Gene Therapy, Cell Therapy, Vaccinology), by End-use (Pharma and Biopharma Companies, Research Institutes), by Disease (Oncology, Genetic Disorders,…

A European Medicines Agency committee has issued a positive opinion on the gene-editing therapy Casgevy (exagamglogene autotemcel) in treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The Committee for Medicinal Products for Human Use (CHMP) recommendation will be considered by the European Commission in making a final approval decision…

Tome Biosciences made a splash last week when it emerged from stealth mode with $213 million in funding. The programmable genomic integration (PGI) company based in Watertown, Massachusetts, raised significant financing from investors like Andreessen Horowitz (a16z) Bio + Health, Arch Venture Partners, Fujifilm Corporation and others.  Tome CEO Rahul Kakkar, MD…

HG302 effectively generates stable genome editing with minimal off-target effects in-vitro and in-vivo A single intravenous injection of HG302 restores functional dystrophin protein expression HG302 demonstrates therapeutic effects by improving muscle functions in humanized DMD mice SHANGHAI and CLINTON, N.J., Dec. 19, 2023 /PRNewswire/ — HuidaGene Therapeutics (辉大基因”HuidaGene”) today announced…

Transcribed image text: 33. The coding DNA sequence of a human gene contains 2001 base pairs. A CRISPR-Cas9 experiment is setting up to knock out this gene in the fibroblast cell lines. It is known that the GC content of the coding sequence of this gene is 60%. In CRISPR-Cas…

By Olivia Trani  The U.S. Food and Drug Administration recently approved two gene therapies, Casgevy and Lyfgenia, to treat sickle cell disease in people ages 12 and older. One of the therapies, Casgevy, use a novel gene-editing technique called CRISPR/Cas9, which can modify a cell’s DNA at a targeted location…

Vertex Pharmaceuticals Incorporated VRTX announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (“EMA”) had adopted a positive opinion, recommending the conditional approval of Casgevy to treat severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The CHMP’s recommendation, although not legally binding, will…

The scenario for the biotech industry in 2024 looks upbeat after a challenging ride in 2023. New drug approvals, pipeline development, and an increase in mergers & acquisitions (M&A) activity boosted investor sentiment in the last couple of months, even though an uncertain macroeconomic environment was a headwind. Pharma and…

(MENAFN– The Express Wire) Global |117 Pages| Report on “Oligonucleotide Library Market” offers a detailed analysis (2024-2031)which is expected to witness remarkable growth in the coming years. The implementation of new technologies and innovative solutions will drive the market’s revenue generation and increase its market share by 2031 with Revenue…

Nkarta is using CRISPR technology for cell therapies for leukemia. The FDA just approved a similar cell therapy treatment for Vertex using the same CRISPR technology. Nkarta is trading well below cash value and has a significant short position sinking underwater. We did very well with Talaris as a takeover…

Tome Biosciences – a programmable genomic integration company – recently announced it has launched to usher in a new era of genomic medicines based on programmable genomic integration (PGI). PGI enables the insertion of any DNA sequence of any size into any programmed genomic location. And the company has raised…

Charles River Laboratories reached a milestone in its strategic collaboration to manufacture CASGEVY (exagamglogene autotemcel [exa-cel]). CASGEVY has been approved in some countries for certain eligible patients. The news follows Charles River’s Memphis center of excellence passing back-to-back audits from both the U.S. FDA and the Health Products Regulatory Authority, on…

In recent years, the field of genetic engineering in agriculture has been rapidly advancing, offering innovative solutions to some of the most pressing challenges in food production. As we look ahead to the period from 2023 to 2030, the global genetic engineering in agriculture market is poised to undergo significant…

The yeast genome contains redesigned chromosome sequences that can shed light on the impact of genetic variations on individual traits and potentially be used to reveal the causes of genetic diseases. Chromosomes are long DNA molecules that collectively form a genome, containing all the genetic material of an organism. Advances…

Global CRISPR-Cas9 Gene Editing Service Market” research and analysis play a crucial role in CRISPR-Cas9 Gene Editing Service Market strategies, as businesses seek to discover which CRISPR-Cas9 Gene Editing Services are most relevant and have the highest search volume within their niche. By understanding the CRISPR-Cas9 Gene Editing Service Market,…

Aldevron complies with the requirements of the General Data Protection Regulation (GDPR). What We CollectInformation collected on this form will be used to contact you (by email or telephone) about your chosen areas of interest, or previous or proposed business transactions. How to withdraw your consentYou can choose whether or…

Sometimes trends are short-term, and as a long-term investor, you probably won’t want to pay too much attention to them. But some trends, because of their potential to change the way things are done for the better, are worth a second look – and even investment. Today, both artificial intelligence…

The European Medicines Agency (EMA) has recommended granting conditional marketing authorization for Casgevy (exagamglogene autotemcel) for the treatment of transfusion-dependent beta-thalassemia and severe sickle cell disease in patients 12 years or older who are appropriate candidates for hematopoietic stem cell transplantation but a suitable donor is not available. The treatment…

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended approval of Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel) to treat transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD). SCD and beta thalassemia are genetic conditions caused by errors in the gene…

In the realm of genetic manipulation, Crispr technology emerges as a double-edged sword. Enabling precise gene editing, it holds promise for eradicating diseases like sickle cell through treatments like Casgevy. Yet, with the power to shape the very fabric of life, ethical concerns loom large. While some foresee a future…

Casgevy became first FDA-approved medicine to use CRISPR/Cas9 gene-editing technology. Image credit: Shutterstock/Nemes Laszlo. Vertex Pharmaceuticals has gained a positive opinion from the EMA’s CHMP for Casgevy (exa-cel), paving the way for European approval of the sickle cell gene therapy early next year. The CHMP will now send their recommendation…

18 Dec 2023 — Researchers from Huazhong Agricultural University in China set out to promote the advancement of probiotic research by reviewing the latest advances and hurdles in CRISPR-Cas systems discovered in microorganisms. They propose a design route for CRISPR systems related to genetically engineered probiotics. While…

By 2049, our global population will reach ~9 billion people. Pests, diseases and adverse environmental conditions are impacting crops across the globe, compounding the issue of feeding a growing population. Traditional breeding techniques have enabled scientists and farmers to develop many varieties of plants and livestock tailored for specific agricultural…

Scientists Use AI to Discover New CRISPR Systems CRISPR is a gene editing tool that has revolutionized the way scientists study and treat diseases. But there are still many unknowns about CRISPR, and scientists are constantly searching for new and improved ways to use it. A team of scientists at…

[High-concept in Medicine] The Latest Research in Medicine Photo by National Cancer Institute on Unsplash The future is rushing toward us with dizzying speed, bearing dazzling innovations that promise to reshape the landscape of medicine. Genetic diseases erased with the flick of a molecular scalpel; custom-grown organs fresh off the…

Everything we covered on IAM over the last seven days – and all you need to know from the global IP market to set yourself up for the start of another busy week. MONDAY 11 December First US CRISPR Cas9 approval marks start of new era of patent wars Data…

[Dec. 17, 2023: JJ Shavit, The Brighter Side of News] Dual action cell therapy is engineered to eliminate established tumors and train the immune system to eliminate the primary tumor and prevent cancer recurrence. (Credit: Creative Commons) In a bold effort to rewrite the story of cancer treatment, scientists are…

All articles published by MDPI are made immediately available worldwide under an open access license. No special permission is required to reuse all or part of the article published by MDPI, including figures and tables. For articles published under an open access Creative Common CC BY license, any part of…

On Dec. 8, CRISPR Therapeutics (CRSP -1.37%) and its collaboration partner Vertex Pharmaceuticals (VRTX 1.00%) made history when the Food and Drug Administration (FDA) gave them the green light to commercialize their gene therapy for sickle cell disease (SCD), which is the first medicine to use the much vaunted CRISPR-Cas9…

Related investors with two people in the background On 8th December, CRISPR Therapeutics (NASDAQ: CRSP) and its collaboration partners Vertex Pharmaceuticals (NASDAQ:VRTX) made history when the Food and Drug Administration (FDA) gave them the green light to commercialize their gene therapy for sickle cell disease (SCD) using the much-hyped CRISPR-Cas9…

(RTTNews) – Vertex Pharmaceuticals Inc. (VRTX) announced Friday that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the conditional approval of CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of severe sickle cell disease (SCD) and…

Kalkan, Ali Kerem; Palaz, Fahreddin; Sofija, Semeniuk; Elmousa, Nada; Ledezma, Yuri; Cachat, Elise; Rios-Solis, Leonardo; (2023) Improving recombinant protein production in CHO cells using the CRISPR-Cas system. Biotechnology Advances , 64 , Article 108115. 10.1016/j.biotechadv.2023.108115. Text Improving recombinant protein production in CHO cells using the CRISPR-Cas system.pdf – Accepted Version…

On December 8, 2023, the FDA approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD) in patients 12 years and older—Vertex’s CASGEVY and Bluebird’s LYFGENIA.  SCD is a group of inherited blood disorders affecting approximately 100,000 people in the U.S. and inducing a median life…

A new gene-editing strategy that does not require the use of a virus can effectively correct a COL7A1 gene mutation in skin cells derived from people with recessive dystrophic epidermolysis bullosa (RDEB), according to a recent report. Most gene therapies in development require the use of viral carriers to help…

Drug the undrugable, treat the untreatable, the revolution in drug development is happening all around us The pharma stock leaders can at times seem overly risk averse with their approach to developing new medicines and cures. With the difficulty of clinical trials and the uncertainty of Federal Drug Administration (FDA) approval,…

Credit: Pixabay/CC0 Public Domain In 2007, Luciano Marraffini struck out on what was then a scientifically lonely path: to understand CRISPR, which had been discovered in bacteria only about a decade before. Seventeen years later, we all know what CRISPR is: a revolution in medicine; a once-in-a-lifetime scientific breakthrough; the…

In a landmark decision last week, the US Food and Drug Administration (FDA) approved two treatments involving cell-based gene therapies.  An agency within the US Department of Health and Human Services, the FDA assures the safety, effectiveness, and security of human and veterinary drugs, vaccines, and other biological products for…

Transcribed image text: 6. A few years ago I set up a cross between BDSC \#79543 females and \#51324 males. Here are the relevant parts of their genotypes: #79543 = P{TKO.GS02468}attP40/CyO  [P\{TKO.GS02468\}attP40 is an inserted element on chromosome 2 that expresses an sgRNA for Cas9-mediated mutagenesis of the white gene] #51324=w; PBac {y[+ mDint2] GFP[E.3xP3]=vas-Cas9}VK00027  […

cygb2 mutant phenotype presents organ laterality defects. a CRISPR/Cas9 mediated genome editing of cygb2. Two different gRNA were targeted to exon 1 (denoted by red text) and resulted in 4 bp and 1 bp frame shift mutations (beginning in the blue shaded region of the predicted protein structure) named cygb2801a and cygb2801b,…

Pictured: European Medicines Agency sign on building/iStock, Lubo Ivanko Following in the footsteps of U.K. and U.S. regulators, a European Medicines Agency committee on Thursday endorsed Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy, which is intended for the treatment of sickle cell disease and transfusion‑dependent beta thalassemia in Europe.  EMA’s Committee…

EMA has recommended approval of the first medicine using CRISPR/Cas9, a novel gene-editing technology. Casgevy (exagamglogene autotemcel) is indicated for the treatment of transfusion‑dependent beta thalassemia and severe sickle cell disease in patients 12 years of age and older for whom haematopoietic stem cell transplantation is appropriate and a suitable donor…

Dive Brief: Days after winning U.S. approval of the first CRISPR gene-editing medicine, Vertex Pharmaceuticals is clearing up potential intellectual property questions about the sickle cell disease therapy by signing a deal with Editas Medicine. In return for a non-exclusive license to use the gene editing technology in the treatment,…

Methods of editing the genome are powering the development of new cell and gene therapies for diseases that have been challenging to treat. Companies like Tome Biosciences are part of a wave of startups building commercial platforms that leverage CRISPR/Cas9 and other genome editing technologies.  Earlier this week, Tome raised…

PRESS RELEASE Published December 14, 2023 The global gene therapy market is set for robust growth, with a strong compound annual growth rate (CAGR) of 19.6% projected from 2023 to 2030. This remarkable expansion is expected to drive market revenue from US$7.8 billion in 2022 to a staggering US$27.3 billion…

HOUSTON ― The University of Texas MD Anderson Cancer Center’s Research Highlights showcases the latest breakthroughs in cancer care, research and prevention. These advances are made possible through seamless collaboration between MD Anderson’s world-leading clinicians and scientists, bringing discoveries from the lab to the clinic and back. Recent developments at…

Vertex Pharmaceuticals to obtain a non-exclusive license for Cas9 for CASGEVY™ (exagamglogene autotemcel) Agreement extends Editas Medicine’s cash runway into 2026 CAMBRIDGE, Mass., Dec. 13, 2023 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced that the Company and Vertex Pharmaceuticals entered into a license…

New regulatory decisions in the U.S. and the U.K. have made Casgevy (exagamglogene autotemcel) the first gene-editing therapy to be approved for adults and adolescents with sickle cell disease (SCD) who experience painful vaso-occlusive crises (VOCs). With these recent approvals, the therapy from Vertex Pharmaceuticals and CRISPR Therapeutics also became…

Editas Medicine, Inc. EDIT announced entering into a licensing agreement with Vertex Pharmaceuticals VRTX for the development of the latter’s new sickle cell disease (SCD) gene therapy, Casgevy. Per the terms of the agreement, Vertex will receive a non-exclusive license to utilize Editas’ Cas9 gene editing technology for ex vivo gene editing medicines…

RoslinCT will manufacture Vertex Pharmaceuticals’ CRISPR-based gene therapy, Casgevy, recently approved for treating sickle cell disease and β thalassemia. On Dec. 11, 2023, RoslinCT, a UK-based cell and gene therapy contract development and manufacturing organization (CDMO), announced that it will manufacture Vertex Pharmaceuticals’ Casgevy (exagamglogene autotemcel [exa-cel]), a recently approved…

Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) is a leader in the global cystic fibrosis therapeutics market. In addition, the company has an extensive portfolio of experimental medicines aimed at treating common diseases such as type 1 diabetes, sickle cell anemia and APOL1-mediated kidney disease (AMKD). Investment thesis On Dec. 8, the Food…

Tada Images / Shutterstock.com More on this story Other related Biotech firm agrees licence deal days after its gene editing treatment gets landmark FDA approval | Patents owned by the Broad Institute protect technology sold via Editas Medicines. Life Sciences Intellectual Property Review (LSIPR) tracks the increasing challenges for intellectual property specialists…

Understand how science, health policy, and medicine shape the world everyday. Sign up for our Morning Rounds newsletter here. Good morning. Reproductive health is dominating the news and the newsletter this morning. We also have a look at the decade-long battle over a patent for CRISPR. It’s been a rollicking…

Want to stay on top of the science and politics driving biotech today? Sign up to get our biotech newsletter in your inbox. Hello, everyone. Damian here with the fallout from Pfizer’s downbeat update, a new entrant in obesity research, and an orthogonal CRISPR business. The need-to-know this morning Apellis Pharmaceuticals said a…

Vertex will pay $50m to Editas upfront, with an additional contingent $50m payment. Image credit: Shutterstock/Catalin Rusnac. Vertex Pharmaceuticals has entered a license agreement with Editas Medicine to use the latter company’s Cas9 gene editing technology. Vertex will pay $50m to Editas upfront, with an additional contingent $50m payment, according…