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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Gene Editing Gene Gene Therapy In-Vivo 145

Drug Discovery World

DECEMBER 6, 2023

Gene editing company Eligo Bioscience has announced a successful $30 million Series B funding round, led by Sanofi Ventures. “We We feel this reflects the strong support for our vision and confirms the potential of Eligo to create a novel class of transformative genetic medicines.”

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

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.

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Gene Editing Gene Genetics Development 272

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

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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Gene Therapy Gene Gene Editing Manufacturing 243

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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Gene Editing Gene Genome Genomics 64

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.

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Gene Editing DNA Gene Development 58

Pharmaceutical Technology

JUNE 8, 2023

Biotechnology company Hopewell Therapeutics has raised $25m in seed financing to accelerate the development of next-generation lipid nanoparticles for targeted delivery of genomic medicines. Hopewell Therapeutics is engaged in discovering, synthesising and developing advanced ttLNPs to provide next-generation genomic medicines.

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Genome Genomics Medicine Development 130

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.

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Gene Editing Gene Genome Genomics 98

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

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Gene Editing Gene Gene Therapy Genetics 52

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 The post Vertex builds in gene editing yet again, with $1.2bn Arbor deal appeared first on.

Gene Editing 52

Gene Editing Gene In-Vivo Genetics 52

Drug Discovery World

FEBRUARY 16, 2023

Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. Aera’s technology also includes a licensed therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene editing enzymes.

Genetics 52

Genetics Medicine Gene Editing In-Vivo 52

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.”

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Gene Editing Genetic Disease Genome Genomics 119

Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin.

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Gene Therapy Gene Gene Editing Clinical Trials 109

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 →

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Gene Editing In-Vivo Genetics Gene 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. At the moment, most CRISPR-based medicines rely on the modification of cells outside the body, that are then used as therapies. Jennifer Doudna. Benjamin Oakes.

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Gene Editing Gene In-Vivo Clinical Trials 69

Medical Xpress

DECEMBER 12, 2022

An international research team led by Dr. Ana Guadaño at the Alberto Sols Biomedical Research Institute (IIBM, a combined CSIC-UAM center) and involving the Complutense University of Madrid (UCM), used CRISPR gene editing techniques to incorporate into mice a mutation of the MCT8 protein responsible for transporting thyroid hormones to the interior (..)

Gene Editing 75

Gene Editing Genetics Hormones Medicine 75

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 98

Gene Therapy Gene Editing Gene Research 98

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.

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Gene Editing Genome Genomics DNA 98

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.

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Gene Therapy Gene Gene Editing Sales 111

Scienmag

MAY 26, 2022

— CRISPR has ushered in the era of genomic medicine. A line of powerful tools has been developed from the popular CRISPR-Cas9 to cure genetic diseases. ITHACA, N.Y.

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Gene Editing Genetic Disease Gene Genome 63

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. . For example, adding a small-molecule might stabilise the medicine, allowing levels of the target protein to increase.

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

Scienmag

DECEMBER 3, 2020

Using a new variant to repair DNA will improve both safety and effectiveness of the much-touted CRISPR-Cas9 tool in genetic research, Michigan Medicine researchers say. Eugene Chen, M.D., […].

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Gene Editing Gene DNA Genetics 41

STAT News

SEPTEMBER 20, 2022

A new CRISPR startup — backed by some big names in venture capital — is planning to develop gene-editing treatments that can insert a genetic sequence of any length, at any location in the DNA strand, according to industry insiders and documents.

DNA 98

DNA Gene Editing Genome Genomics 98

Drug Discovery World

OCTOBER 10, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

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Gene Editing Genome Genomics Genetics 57

XTalks

OCTOBER 20, 2023

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

In-Vivo 52

In-Vivo Gene Editing Trials Gene 52

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Scienmag

APRIL 21, 2021

New approach to gene therapy can correct any disease-causing mutation within a gene UNIVERSITY PARK, Pa.

Gene 52

Gene Gene Editing Gene Therapy Genetic Disease 52

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.”

Gene 98

Gene Regulation Protein Gene Editing 98

XTalks

JANUARY 26, 2024

Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. By editing these genes, researchers can effectively neutralize their cancer-promoting effects.

Research 118

Research Gene Editing Gene Genetics 118

Drug Discovery World

MAY 24, 2023

This exceeds the current, but growing, global supply of plasmid DNA for genetic medicine, and dramatically increases Touchlight’s capacity to meet growing demand for DNA from biopharmaceutical companies. Touchlight has contracts to supply many top biopharmaceutical companies, including Pfizer and Lonza.

DNA 52

DNA Gene Editing Gene Therapy Genetics 52

Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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Gene Therapy FDA Approval Gene Gene Editing 52

Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Drugs 52

Drugs Gene Editing Genome Genomics 52

Drug Discovery World

NOVEMBER 14, 2023

FH is one of the most common genetic conditions, affecting around one in 300 people globally. It is caused by a single gene mutation that impairs the body’s ability to mediate LDL cholesterol. Since the mutation only affects a single gene, it makes it a prime candidate for genome editing treatments.

In-Vivo 52

In-Vivo Gene Editing Gene Genome 52

Drug Discovery World

OCTOBER 10, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Gene Editing 52

Gene Editing Genome Genomics Genetics 52

Drug Discovery World

JUNE 22, 2023

“This is part of our long-term business strategy of leveraging exosome-mediated delivery to create and enable genetic medicines such as gene therapy and genome editors”.

Gene Therapy 52

Gene Therapy Gene Editing Drug Delivery Gene 52

XTalks

FEBRUARY 11, 2021

This has ushered in a new era of genomics that is fostering rapid, detailed and personalized insights into human genetics. Xtalks is celebrating the International Day of Women and Girls in Science with a special overview of notable female scientists who have made revolutionary contributions to the field of genetics. Rosalind Franklin.

Genetics 119

Genetics DNA Genome Genomics 119