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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pharmaphorum

OCTOBER 13, 2022

In the last decade, there has also been rapid development and interest in CRISPR/Cas9 technology and other gene-editing tools that could offer transformative avenues to deliver gene therapies for patients and families affected by devastating diseases such as sickle cell disease and thalassemia. DOWNLOAD THE FULL ARTICLE HERE.

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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.

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pharmaphorum

JUNE 23, 2022

Novartis has shouldered its way into the in vivo gene editing category via a deal with US biotech Precision BioSciences, focused on a therapy for sickle cell disease (SCD). It is also working with Eli Lilly on in vivo gene-editing drugs against three targets, including one in Duchenne muscular dystrophy, under the terms of a $1.4

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pharmaphorum

APRIL 25, 2022

With iPSCs exhibiting game-changing potential in the bid to develop an off-the-shelf therapy, the brand-new iPSC Manufacturing Summit is the only summit dedicated to solving the manufacturing, quality and analytical challenges hindering your iPSC-derived treatments from reaching patients. Hear from: Fate Therapeutics. Cartherics.

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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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pharmaphorum

APRIL 23, 2021

CRISPR Therapeutics is to receive a hefty $900m payment from Vertex after the companies amended a collaboration to develop, manufacture and market a gene editing therapy for sickle cell disease and beta thalassemia.

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pharmaphorum

SEPTEMBER 29, 2020

For example, under a traditional pharma/biotech collaboration and licensing model, a biotech partner may have primary responsibility for significant elements of research and early product development, and the pharma partner may lead the majority of later stage development, as well as post-approval commercial manufacture and supply.

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pharmaphorum

OCTOBER 21, 2020

The financing will support manufacturing of a first clinical batch of the product, regulatory activities and a first human study scheduled to start next year. Boissel was previously executive vice president of corporate strategy at gene editing biotech Sangamo Therapeutics.

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pharmaphorum

MAY 7, 2021

Gene editing firm CRISPR Therapeutics has announced a collaboration with US biotech Nkarta to develop natural killer (NK) cell therapies for cancer. It’s at the forefront of gene editing although the technology has spawned rivals including Intellia Therapeutics, Caribou Biosciences, Sangamo Therapeutics and Mammoth Biosciences.

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Pharmaceutical Technology

MARCH 1, 2023

It discovers, develops and manufactures prescription drugs to treat cancer, multiple sclerosis and infertility; and develops liquid crystal mixtures, organic light-emitting diode (OLED) materials, cosmetic active ingredients, pigments for coatings, and high-tech materials.

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Pharmaceutical Technology

MARCH 1, 2023

It discovers, develops and manufactures prescription drugs to treat cancer, multiple sclerosis and infertility; and develops liquid crystal mixtures, organic light-emitting diode (OLED) materials, cosmetic active ingredients, pigments for coatings, and high-tech materials.

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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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XTalks

SEPTEMBER 20, 2024

Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality. Its therapeutic pipeline targets unmet needs in oncology and autoimmune disease.

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The Pharma Data

JANUARY 17, 2021

18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD). NEW YORK , Jan.

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The Pharma Data

AUGUST 4, 2020

UK-based Oxford Biomedica has signed a new development, manufacture and license agreement with Beam Therapeutics for next-generation CAR-T therapeutics. Agreement will utilise Oxford Biomedica's LentiVector platform. The agreement also puts in place a three year clinical supply agreement for the two companies.

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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. The potential for CRISPR gene editing to offer a therapeutic option to patients is clear, based on its mechanism, but there are already gene editing therapeutics on the market.

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Pharmaceutical Technology

MAY 2, 2023

Under the deal’s structure, BMS has assumed sole responsibility for further worldwide development, manufacturing, and commercialisation of the therapy. This news comes a few weeks after BMS signaled an increased focus on cell therapy manufacturing with a takeover of former Novartis plant to boost viral vector production for T-cell therapies.

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pharmaphorum

OCTOBER 8, 2021

The company is also seeking if there is evidence of clonal expansion of the abnormality in in other words passed on to daughter cells – and if it is linked to the gene-editing used to modify the ALLO-501A cells.

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Delveinsight

JANUARY 14, 2021

Last year, the company disclosed its gene-writing service, which combined various gene editing, manufacturing, and synthesizing technologies to provide more tailored therapeutic instructions to genetic code. Verve Therapeutics has an aim to bring one-and-done gene editing treatments to heart disease.

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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. billion in funding.

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pharmaphorum

AUGUST 25, 2022

Explore approaches to boost safe gene-editing, approach regulatory affairs, turbocharge scale-up, optimize analytics and more to produce best-in-class therapies for immuno-oncology and regenerative medicine. Sana Biotechnology , Takeda and many more.

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Roots Analysis

JANUARY 16, 2023

At-home Self-testing Kit Manufacturers – Current Market Landscape. Gene Editing beyond CRISPR Market, 2022-2035. Cell and Gene Therapy Bioassay Services Market, 2021-2030. Currently, more than 650 at-home self-testing kits are available worldwide and are approved by regulatory authorities. Our Social Media Platform.

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Advarra

NOVEMBER 29, 2022

Bottlenecks in the manufacturing process, supply chain issues such as accessibility of good manufacturing practice (GMP)-grade reagents, and shortages of qualified scientists and engineers have caused many therapies to fail at critical stages of the clinical development pipeline. But review issues are not the only problems.

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Delveinsight

MARCH 2, 2021

As per the company, its manufacturing platform supports large-scale production and cryopreservation of off-the-shelf, allogeneic NK cell therapies and proprietary CAR-NK and NK-specific gene-editing technologies.

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XTalks

FEBRUARY 10, 2023

The most common factors associated with delays were insufficient non-clinical development and chemistry, issues with manufacturing and controls (CMC), clinical trial design problems, inadequate site and vendor feasibility, and regulatory authority submission delays. Among 20 recent trials, 17 experienced delays in meeting key milestones.

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The Pharma Data

NOVEMBER 3, 2020

LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples.

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The Pharma Data

SEPTEMBER 1, 2021

” With CRISPR-Cas9 gene editing, cells have the potential to be programmed to make all sorts of drugs, meaning that if one arthritis drug works better than another in a particular patient, the researchers could engineer cartilage cells to make personalized treatments.

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The Pharma Data

DECEMBER 27, 2020

Our work began modestly, but we hit our stride in 2020 and reached important clinical, manufacturing, and business milestones this year, which created substantial value and advanced us toward our ultimate goal. Our goal of building Lineage into the preeminent cell therapy company is ambitious but attainable.

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Roots Analysis

OCTOBER 11, 2023

To balance these factors, we can expect to see an increased collaboration between manufacturers, suppliers, and regulatory agencies to ensure safe, and contamination-free bio innovations. The following figure presents some of the key market drivers and restraints in the agricultural biologicals domain.

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Pharmaceutical Technology

MAY 26, 2023

Tripling its production capacity, Touchlight can now manufacture 8kg plasmid DNA, a key component for mRNA gene therapies and vaccines. As per the company, dbDNA is suitable for rapid, scalable manufacture of GMP DNA and can incorporate gene sequences of sizes ranging from 500bp to 20kb, which are typically unstable as pDNA.

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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.

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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 treatment used a new version of CRISPR to turn on a backup copy of the dystrophin gene that mysteriously lies dormant in our muscles.

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Cloudbyz

MARCH 6, 2024

This shift signifies not just a change in the way drugs are manufactured and administered, but also a fundamental transformation in the approach to treating diseases at their root cause. In this blog post, we delve into the rise of biologics and explore how they are reshaping the pharmaceutical industry and improving patient outcomes.

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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. 3. Manufacturing & Supply Chain: Addressing Drug Shortages. And each drug shortage often has a unique reason.

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XTalks

DECEMBER 13, 2023

XTALKS WEBINAR: Cryopreservation Best Practices for Cell & Gene Therapy Source Material Live and On-Demand: Monday, January 22, 2024, at 11am EST (4pm GMT/UK) Register for this free webinar to learn about cryopreservation, a vital step in the manufacturing of today’s cell and gene therapies. How do Casgevy and Lyfgenia Work?

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The Pharma Data

DECEMBER 11, 2020

of Children’s Hospital at TriStar Centennial in Nashville, Tennessee, and colleagues found that gene editing using CRISPR-Cas9 is safe and feasible and can provide clinical benefit to patients with sickle cell disease and transfusion-dependent ß-thalassemia. ASH: CRISPR-Cas9 Gene Editing Promising in TDT, SCD.

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