Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD).

Gene Editing 130

Gene Editing Gene Gene Therapy In-Vivo 130

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.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

pharmaphorum

FEBRUARY 15, 2024

Gene-editing biotech Prime Medicine has priced its IPO, hoping to raise $140m to advance its pipeline of one-shot therapies for severe genetic disorders.

Gene Editing 64

Gene Editing Gene Medicine Genetics 64

STAT News

FEBRUARY 22, 2023

Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions.

Gene Editing 126

Gene Editing Gene Genetic Disease Research 126

BioSpace

MARCH 24, 2024

Ubiquitous potential, possible safety advantages and the recent growth of cell and gene therapy are driving investment in a different type of genetic editing.

Gene Editing 140

Gene Editing Gene Gene Therapy Genetics 140

Bio Pharma Dive

NOVEMBER 20, 2020

Through a deal with Precision Biosciences, the pharma aims to develop therapies for genetic disorders, including Duchenne muscular dystrophy.

Gene Editing 264

Gene Editing Gene Genetics Development 264

Bio Pharma Dive

FEBRUARY 28, 2024

The acquisition of Proof Diagnostics, which was formed to develop a COVID-19 test, gives Ginkgo a library of novel enzymes that can be used to make genetic medicines.

Gene Editing 182

Gene Editing Gene Genetics Medicine 182

XTalks

DECEMBER 14, 2022

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.

Gene Editing 92

Gene Editing DNA Gene Gene Sequencing 92

Bio Pharma Dive

JUNE 15, 2021

A deal with biotech startup Capsida, which recently raised $140 million, gives CRISPR access to a Caltech technology that aims to improve the delivery of genetic medicines.

Gene Editing 290

Gene Editing Gene Genetics Development 290

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

BioPharma Reporter

FEBRUARY 23, 2023

The messenger RNA (mRNA) specialist Moderna has teamed up with ElevateBio-owned Life Edit Therapeutics to develop gene editing therapies that are delivered into patients in vivo.

Gene Editing 52

Gene Editing Genetic Disease Gene In-Vivo 52

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.

Gene Therapy 243

Gene Therapy Gene Gene Editing Development 243

pharmaphorum

SEPTEMBER 28, 2022

Vertex Pharma and partner CRISPR Therapeutics will start a rolling marketing application in the US for their gene-editing drug for sickle cell disease (SCD) and beta thalassaemia later this year. The time places exa-cel in pole position to become the first drug developed based on CRISPR/Cas9 gene-editing technology to reach the market.

Gene Editing 59

Gene Editing Gene In-Vivo Clinical Trials 59

Scienmag

MAY 2, 2022

WINSTON-SALEM, NC – May 2, 2022 — Wake Forest Institute for Regenerative Medicine (WFIRM) scientists working on CRISPR/Cas9-mediated gene editing technology have developed a method to increase efficiency of editing while minimizing DNA deletion sizes, a key step toward developing gene editing therapies to treat genetic diseases.

Gene Editing 58

Gene Editing DNA Gene Development 58

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.

Gene Editing 64

Gene Editing Gene Genome Genomics 64

BioSpace

MARCH 21, 2024

Tegoprubart, Eledon Pharmaceuticals’ experimental anti-CD40L antibody, was part of the immunosuppressive treatment regimen used following the first-ever transplant of a kidney from a genetically modified pig to a human.

Gene Editing 99

Gene Editing Antibody Gene Genetics 99

pharmaphorum

OCTOBER 13, 2022

Gene therapies and research into them have grown immensely in recent years, offering more novel tools in regenerative medicine to fight disease, including rare diseases and genetic disorders. Beta-thalassemia is a rare blood disorder caused by a genetic defect in hemoglobin. Novartis also recently inked an up-to $1.5

Gene Editing 52

Gene Editing Gene Gene Therapy Genetics 52

Worldwide Clinical Trials

OCTOBER 24, 2023

Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. Roughly 80% of rare diseases are due to a known genetic driver. Over the years, I have attended several Global Genes Summits.

Gene 130

Gene Gene Editing Gene Therapy Clinical Trials 130

pharmaphorum

AUGUST 24, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2 If approved, it could mount a challenge to bluebird bio’s gene therapy Zynteglo, which is already approved in Europe.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Medical Xpress

MARCH 17, 2023

The study, to be published March 17 in the Journal of Experimental Medicine, uses a new, highly versatile form of CRISPR-based genome editing with the potential to correct a wide variety of disease-causing genetic mutations.

Gene Editing 98

Gene Editing Gene Genome Genomics 98

XTalks

MARCH 7, 2024

In a pivotal move for the food industry, the US Food and Drug Administration (FDA) has unveiled industry guidance for genome edited foods derived from plants. Genome editing stands as a cutting-edge technique that ingeniously introduces new traits into plants.

Gene Editing 52

Gene Editing Gene Genome Genomics 52

BioTech 365

MARCH 30, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Prime editing enables precise gene editing without collateral damage.The latest gene editing technology, prime editing, expands the ‘genetic toolbox’ for more precisely creating disease models and correcting … (..)

Gene Editing 40

Gene Editing Gene Genetics Research 40

Scienmag

JANUARY 12, 2021

Martins Scientists have used gene-editing advances to achieve a tenfold increase in the production of super-bug targeting formicamycin antibiotics. Credit: Dr Dino J. The John Innes Centre researchers used the technology to create a new strain of Streptomyces formicae bacteria which over-produces the medically promising molecules.

Gene Editing 53

Gene Editing Gene Bacteria Scientist 53

STAT News

MARCH 1, 2023

Tessera Therapeutics chief scientist Michael Holmes had just finished presenting a much-anticipated peek at a technology the company had previously said could “ revolutionize genetic medicine ” and “cure nearly any genetic disease.”

Gene Editing 119

Gene Editing Genome Genetic Disease Genomics 119

Scienmag

AUGUST 18, 2020

Credit: Rasgon Lab, Penn State Researchers who developed an improved method of gene editing for the study of arthropods will expand the technology for use in vertebrate species such as mice, fish and birds after receiving new funding from the National Science Foundation.

Gene Editing 61

Gene Editing Gene Development Research 61

BioTech 365

DECEMBER 4, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Advancing gene editing with new CRISPR/Cas9 variant.Using a new variant to repair DNA will improve both safety and effectiveness of the much-touted CRISPR-Cas9 tool in genetic research, … Continue reading →

Gene Editing 40

Gene Editing Gene DNA Genetics 40

pharmaphorum

OCTOBER 8, 2020

Scribe Therapeutics, a start-up focusing on gene-editing using CRISPR/Cas9, has burst onto the biotech scene with a $415 million deal with Biogen. Further back in development are drugs that will be administered to edit genes within the body, but the first of these candidates are now in clinical trials. Jennifer Doudna.

Gene Editing 69

Gene Editing Gene In-Vivo Clinical Trials 69

Scienmag

NOVEMBER 19, 2020

Media contacts: Jennifer Kuzma, jkuzma@ncsu.edu Khara Grieger, kdgriege@ncsu.edu Mick Kulikowski, News Services, 919.218.5937 or mick_kulikowski@ncsu.edu Researchers at North Carolina State University call for a coalition of biotech industry, government and non-government organizations, trade organizations, and academic experts to work together to (..)

Gene Editing 44

Gene Editing Gene Research Production 44

pharmaphorum

FEBRUARY 15, 2024

Gene-editing biotech Prime Medicine has priced an initial public offering, hoping to raise $140 million to advance its pipeline of one-shot therapies for severe genetic disorders. The Cambridge, Massachusetts-based company is offering 19.2 million shares at $6.25

Medicine 105

Medicine Gene Editing Genetics Gene 105

Pharmaceutical Technology

MARCH 28, 2023

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

Gene Editing 235

Gene Editing DNA Gene Gene Therapy 235

Pharmaceutical Technology

AUGUST 24, 2022

A lead product candidate of the company, GTP-506 is a single-dose gene editing therapy. The ARCUS nuclease vector (GTP-506A) targets gene editing in the PCSK9 gene locus that is well characterised, while the therapeutic donor vector (GTP-506D) introduces the OTC gene to provide the required genetic correction.

Gene Editing 130

Gene Editing Gene Therapy Gene Genetics 130

BioTech 365

MAY 1, 2021

Editas Medicine to Present Preclinical Data Demonstrating Advancements in In Vivo Gene Editing Approach for the Treatment of Genetic Ocular Diseases at the Association for Research in Vision and Ophthalmology Annual Meeting Editas Medicine to Present Preclinical Data Demonstrating Advancements … Continue reading →

Gene Editing 40

Gene Editing In-Vivo Genetics Gene 40

STAT News

MARCH 9, 2023

LONDON — Scientists at this year’s genome editing summit spent Tuesday showing the world just how far CRISPR -based medicines for treating human diseases have come in a decade.

Gene Editing 98

Gene Editing Genome Genomics DNA 98

Medical Xpress

DECEMBER 21, 2022

Using the CRISPR-Cas9 gene editing system, UT Southwestern researchers corrected mutations responsible for a common inherited heart condition called dilated cardiomyopathy (DCM) in human cells and a mouse model of the disease.

Gene Therapy 93

Gene Therapy Gene Editing Gene Research 93

pharmaphorum

APRIL 23, 2021

Vertex Pharma has ramped up its involvement in gene-editing medicines for the second time in a week, paying Obsidian Therapeutics $75 million upfront to access its technology platform. . Alternatively, a drug can be administered that switches off the genetic medicine and brings protein expression to a halt.

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

Scienmag

MAY 26, 2022

A line of powerful tools has been developed from the popular CRISPR-Cas9 to cure genetic diseases. ITHACA, N.Y. — CRISPR has ushered in the era of genomic medicine. However, there is a last-mile problem – these tools need to be effectively delivered into every cell of the patient, and most Cas9s are too big to be fitted […].

Gene Editing 63

Gene Editing Genetic Disease Gene Genome 63

XTalks

JANUARY 4, 2024

Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. There is a significant focus on developing gene therapies as longer-term solutions for the disease.

Gene Therapy 110

Gene Therapy Gene Gene Editing Sales 110