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

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.

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Gene Editing Gene In-Vivo Genetics 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.” Continue to STAT+ to read the full story…

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Gene Editing Genetic Disease Genomics Genome 119

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.

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Gene Editing Genetic Disease Gene Genomics 63

Pharmaceutical Technology

JUNE 8, 2023

The ttLNP platform can deliver a range of high-impact genomic payloads, including gene editing, siRNA, mRNA, DNA and gene-writing apparatus, to tissues, cells and organs. The development will be advanced through both internal pipeline programmes and external collaborations.

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Genomics Genome Medicine Development 130

STAT News

MARCH 3, 2023

Continue to STAT+ to read the full story…

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Genomics Genome Gene Editing Genetic Disease 111

Scienmag

APRIL 21, 2021

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

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Gene Gene Editing Gene Therapy Genetic Disease 52

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.

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Gene Editing DNA Gene Genome 88

STAT News

SEPTEMBER 16, 2022

Preliminary results from the study — just the second to show that CRISPR-based gene editing can be delivered systemically and performed inside the body — found the treatment, NTLA-2002, reduced levels of the disease-causing protein, kallikrein, by 65% and 92% in the low- and high-dose cohort, respectively.

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Gene Editing Genetic Disease Genetics Protein 94

pharmaphorum

DECEMBER 6, 2020

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has achieved “remarkable” improvements in patients with beta thalassaemia and sickle cell disease in an early-stage trial reported at the American Society of Haematology (ASH) annual meeting.

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Gene Editing Gene In-Vivo Genetics 52

Camargo

JULY 27, 2021

Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases. It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy.

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Gene Therapy Gene Gene Editing Genetics 238

pharmaphorum

JANUARY 10, 2022

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences.

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In-Vivo Gene Editing Gene Therapy Gene 52

pharmaphorum

APRIL 20, 2022

The potential to develop treatments with CRISPR gene editing is hinged on its ability to add, delete or replace elements within a target DNA sequence. Cancer is a genetic disease, caused by certain changes in the way that genes control cell function, such as how they grow and divide. Why cancer? The pipeline.

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Gene Editing DNA Gene Genetics 52

pharmaphorum

FEBRUARY 17, 2022

The drug – which costs $125,000 at US list prices – can be used either alone or in combination with hydroxyurea, a well-established therapy for the disease. CRISPR Therapeutics and Vertex are also in the running with their gene-editing candidate CTX001, in phase 1/2 trials which are due to generate final results later this year.

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Drugs Gene Therapy Gene Editing Gene 59

The Pharma Data

DECEMBER 13, 2020

“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO

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Business Development Development Gene Editing In-Vivo 40

pharmaphorum

SEPTEMBER 14, 2022

The French biotech said the cash injection – which follows a €45 million Series A in 2020 – will also help it bring forward the first development candidate in a collaboration signed with gene-editing specialist Intellia Therapeutics last year, currently in the early-discovery stage.

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pharmaphorum

JANUARY 10, 2022

The company has also stated, at the same time as the study was announced, that it will look at developing mRNA technology in oncology and genetic diseases. This not only allowed Pfizer to establish a COVID-19 vaccine but the company also recently announced that it had begun a study testing an mRNA flu vaccine.

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XTalks

MAY 4, 2021

The CRISPR gene editing system consists of the Cas9 enzyme, which serves as molecular scissors to cleave double-stranded DNA, and a guide RNA template targeted to a specific genomic sequence, which allows for precise editing.

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DNA Gene Gene Silencing Genome 91

XTalks

DECEMBER 13, 2023

Casgevy’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US. In 2022, bluebird won approvals for two gene therapies — Skysona for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD) and Zynteglo for beta-thalassemia.

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Gene Therapy Gene FDA Approval In-Vivo 90

XTalks

MAY 4, 2021

The CRISPR gene editing system consists of the Cas9 enzyme, which serves as molecular scissors to cleave double-stranded DNA, and a guide RNA template targeted to a specific genomic sequence, which allows for precise editing.

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DNA Gene Gene Silencing Genome 52

XTalks

DECEMBER 29, 2022

In 2022 alone, the US regulator approved four new gene therapies, showing the high interest in getting these therapies to market. In 2023, a number of gene therapies are expected to get the FDA green light. Given their precise targeting, gene therapies have largely been focused on oncology indications followed by rare genetic diseases.

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Life Science Gene Therapy Gene Manufacturing 52

XTalks

DECEMBER 22, 2021

The gene-editing tool CRISPR edits DNA using specialized strands of guide RNA and endogenous cellular mechanisms of RNA interference. RNA-based treatments include RNA interference (RNAi) with double-stranded RNA molecules, or small interfering RNA (siRNA) involving single-stranded antisense oligonucleotides (ASOs).

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