The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., “Accessing state-of-the-art LNP technology through this collaboration will add momentum to our gene editing efforts for the benefit of patients.”

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pharmaphorum

AUGUST 31, 2022

After a recent approval, there are now three gene therapies available on the US market. In recent years, gene therapy has transitioned from a promising idea to a reality for patients, with many of the severe safety issues that emerged in early iterations of the technology being overcome. from 2021 to 2029.

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Drug Discovery World

MAY 23, 2023

DDW’s Megan Thomas caught up with George White , General Manager, Product Management, Cell & Gene Therapy at Cytiva, to learn more about the company’s insight into the CGT market. Key challenges for cell and gene therapies In order for cell and gene (CGT) therapies to succeed, George White identifies several challenges to overcome.

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Pharmaceutical Technology

MARCH 28, 2023

Vertex Pharmaceuticals has signed a new non-exclusive licensing agreement with CRISPR Therapeutics to expedite the development of its hypoimmune cell therapies to treat type 1 diabetes (T1D). The gene-editing technology allows for precise, directed changes to genomic DNA.

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The Pharma Data

JANUARY 11, 2023

Using nanotechnology that enabled mRNA-based COVID-19 vaccines, a new approach to gene therapy may improve how physicians treat inherited forms of blindness. “Improving the technologies used for gene therapy can provide more treatment options to prevent blindness. .”

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Pharmaceutical Technology

APRIL 28, 2023

The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022.

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Drug Discovery World

SEPTEMBER 6, 2022

In recent years the advanced therapy industry has been the most significant disruptor in medicine. Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. ViaCyte, Beam, and Verve are all due to begin gene editing trials this year.

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Bio Pharma Dive

FEBRUARY 23, 2021

Guide Therapeutics, the company Beam acquired, is working on ways to more efficiently deliver gene editing therapies into the body.

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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.

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Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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Pharmaceutical Technology

NOVEMBER 16, 2022

A custom-made viral vector and cell therapy centre of excellence, ElevateBio BaseCamp has an end-to-end process development and existing Good Manufacturing Practice (cGMP) production expertise for research, clinical and commercial cell and gene therapies and regenerative treatments.

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Pharmaceutical Technology

APRIL 4, 2023

Formerly known as CTX001, exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. If granted, these would shorten the time taken for application review to eight months.

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pharmaphorum

FEBRUARY 23, 2022

As investment in gene therapies continues to grow rapidly, more effectively engaging patients throughout development has become a priority for any company serious about building safer and more meaningful gene therapy programs. The post 2nd Annual Gene Therapy Patient Engagement Summit appeared first on.

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Gene Therapy Gene Gene Editing Clinical Trials 52

pharmaphorum

JUNE 12, 2022

A gene-editing drug developed by CRISPR Therapeutics and Vertex Pharma has continued to show impressive results in clinical trial, with an update at the EHA congress showing long-term effects on the symptoms of both beta thalassaemia and sickle cell disease. billion product if it gets approved for both indications. .”

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Gene Editing Gene Gene Therapy Drugs 59

pharmaphorum

SEPTEMBER 14, 2022

SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related macular degeneration (AMD). The post SparingVision raises €75m for eye disease gene therapies appeared first on.

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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. The move marks a continuation in its ongoing commitment to exploring gene-editing technology. DOWNLOAD THE FULL ARTICLE HERE.

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Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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pharmaphorum

OCTOBER 21, 2020

million) to develop its gene therapy for a rare ocular disease. SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy consisting of one neurotrophic factor and one oxidative stress reducing enzyme which, acting together, aim to slow or stop the degeneration of photoreceptors. million euros ($52.7

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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.

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Drug Discovery World

FEBRUARY 27, 2023

An investigational gene therapy for Sanfilippo syndrome – which leads to a form of childhood dementia – has shown promising early results in a proof-of-concept study. The post Gene therapy for ‘childhood dementia’ shows promise appeared first on Drug Discovery World (DDW).

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Pharmaceutical Technology

APRIL 25, 2023

After missing the previous Q1 deadline, bluebird bio has submitted a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for its sickle cell disease gene therapy lovo-cel, based on an April 24 company announcement. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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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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.

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STAT News

MARCH 22, 2023

DALLAS — It took just three years, after the tool’s invention, for researchers to devise ways of using CRISPR-Cas9 gene editing to treat mice with Duchenne muscular dystrophy. Yet, with one exception, no gene-editing treatments for the rare muscle-wasting disease have entered the clinic and none appear particularly close.

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Medical Xpress

NOVEMBER 4, 2022

The lone volunteer in a unique study involving a gene-editing technique has died, and those behind the trial are now trying to figure out what killed him.

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Pharmaceutical Technology

AUGUST 26, 2022

ElevateBio has signed a long-term strategic collaboration with the University of Pittsburgh, US, to establish a biomanufacturing centre for expediting cell and gene therapy development. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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BioSpace

SEPTEMBER 28, 2021

?Gene therapy research delivers breakthroughs this week with a CRISPR-edited droplet that could lead to a cure for heart disease and the possibility of turning glial brain cells into neurons.

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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.

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Medical Xpress

FEBRUARY 7, 2023

A recent study by University of Illinois Chicago researchers in the College of Business Administration analyzed how the experiences of racism and discrimination in health care significantly affect the adoption of innovative medical technology like gene therapies and the gene editing tool CRISPR.

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BioTech 365

JANUARY 10, 2022

Bayer and Mammoth Biosciences to Collaborate on Novel Gene Editing Technology Bayer and Mammoth Biosciences to Collaborate on Novel Gene Editing Technology Agreement strengthens Bayer’s new cell and gene therapy platform further Supports Mammoth’s vision of unlocking the full potential … Continue reading →

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STAT News

NOVEMBER 4, 2022

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. The clinical trial was to be the first time anyone got a gene editing therapy for muscular dystrophy.

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BioTech 365

OCTOBER 21, 2021

Graphite Bio Announces Participation in Upcoming Jefferies Gene Therapy/Editing Summit Graphite Bio Announces Participation in Upcoming Jefferies Gene Therapy/Editing Summit SOUTH SAN FRANCISCO, Calif.–(BUSINESS –(BUSINESS WIRE)–Graphite Bio, Inc.

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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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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. Common gene therapies use vehicles such as adeno-associated vectors (AAVs), which cannot carry the required payload in this case, says Carney.

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STAT News

JULY 12, 2022

Somewhere in New Zealand, the first patient ever has been dosed with a kind of gene-editing treatment known as a base editor, a newer way of utilizing CRISPR for gene editing. The company studying the treatment, Verve Therapeutics, announced the news Tuesday. Continue to STAT+ to read the full story…

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Drug Discovery World

MARCH 9, 2023

The post FDA clears in vivo gene editing therapy for trials in US appeared first on Drug Discovery World (DDW). In 2022, Intellia reported positive interim results from the Phase I study demonstrating deep, dose-dependent reductions in plasma kallikrein and robust reductions in patient HAE attacks.

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BioSpace

OCTOBER 26, 2021

With the complete acquisition of Life Edit, ElevateBio will be able to integrate that company’s genome editing capabilities with its cell and gene therapies.

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