XTalks

APRIL 24, 2023

Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies. Dr. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals.

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Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

APRIL 13, 2023

Takeda has announced that it will be pivoting away from its discovery and preclinical programmes in adeno-associated virus (AAV) gene therapies. Takeda’s announcement underlines the risk associated with gene therapy R&D at the preclinical stage and the fact that many current AAV programs are unlikely to reach late-stage trials.

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Drug Discovery World

AUGUST 7, 2023

The collaboration will work on developing exosome-encapsulated AAV (exoAAV) vectors as a novel gene delivery technology aimed at improving treatments for heart disease. Susmita Sahoo, Associate Professor of Medicine, Cardiology at Icahn Mount Sinai has been exploring the use of exosomes in gene therapy for several years.

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Gene Therapy Gene Research In-Vivo 52

pharmaphorum

JULY 9, 2021

Orchard Therapeutics’ gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance. The post NICE rejects Orchard’s gene therapy for rare childhood disease MLD appeared first on.

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Gene Therapy Gene In-Vivo Marketing 105

Drug Discovery World

JANUARY 27, 2023

The post $5 million raised towards gene therapies for muscular diseases appeared first on Drug Discovery World (DDW). As proof of principle for the platform, success in treating Duchenne would open up opportunities for Myosana to target a large range of neuromuscular and cardiac diseases.”

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Gene Therapy Gene In-Vivo Development 52

XTalks

OCTOBER 20, 2023

Intellia said NTLA-2001 is the first investigational in vivo CRISPR-based gene editing therapy cleared to enter late-stage clinical development. As an in vivo therapy, it can edit genes inside the body rather than in cells extracted from patients. ATTR amyloidosis is a rare, progressive and fatal disease.

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XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

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Gene Therapy Gene Gene Editing In-Vivo 59

Pharmaceutical Technology

MAY 19, 2023

Myeloid Therapeutics has raised $73m to support the continued clinical development of its lead cell therapy programme, MT-101, in Phase I/II trials for T cell lymphoma. MT-302 is a TROP2-targeting in vivo chimeric antigen receptor (CAR) that has been designed to express in the myeloid compartment.

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Drug Discovery World

MARCH 22, 2024

This has been an interesting week for cell and gene therapies, with two landmark FDA approvals, two significant fundraising efforts and potentially ground-breaking study results in glioblastoma. News round-up for 18-22 March by DDW Digital Content Editor Diana Spencer.

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Gene Therapy In-Vivo FDA Approval Drugs 52

Pharmaceutical Technology

APRIL 14, 2023

Ginkgo Bioworks (Ginkgo) and the Wisconsin Alumni Research Foundation (WARF) have collaborated to discover next-generation GD2 CAR T-cell therapies to treat solid tumours. All the new GD2 CAR designs developed through the partnership will be validated via in vitro screens and in vivo murine models.

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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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Gene Therapy Gene Gene Editing In-Vivo 40

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9

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Gene Therapy Gene Manufacturing Medicine 52

Drug Discovery World

FEBRUARY 23, 2024

In a breakthrough for advanced therapies, this week saw the FDA approve the first ever cell therapy for solid tumour cancers, but there were other significant developments in the cell and gene therapy space. News round-up for 19-23 February by DDW Digital Content Editor Diana Spencer.

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Gene Therapy Insulin FDA Approval Drugs 52

Pharmaceutical Technology

MAY 12, 2023

The in vivo study, conducted in mice, involved a phosphatidylserine-containing nano-formulation of mRNA supplied by BioNTech, distinct from traditional lipid nanocrystals (LNCs). The latter partnership was recently expanded to focus on in vitro and in vivo delivery of mRNA after BioNTech pulled out of the deal with Matinas.

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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. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. g/dL and 45.4%

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The Pharma Data

JANUARY 4, 2021

Collaboration with Biogen to develop gene therapy for an undisclosed target to treat inherited eye disease, plus option for additional target. 05, 2021 (GLOBE NEWSWIRE) — ViGeneron GmbH , a gene therapy company, today announced a global collaboration and licensing agreement with Biogen Inc.

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Gene Therapy Gene Development In-Vivo 52

Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

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Gene Editing Gene Gene Therapy In-Vivo 165

Pharmaceutical Technology

MAY 18, 2023

Forbion European Acquisition has entered a definitive business combination agreement with biotechnology company enGene to create a combined biotechnology company to develop next-generation non-viral, locally administered gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene In-Vivo Life Science 130

BioSpace

NOVEMBER 18, 2021

Avrobio’s ex vivo lentiviral gene therapy platform led to durable responses measured in years, and significant reductions in disease-related biomarkers that far exceed today’s standard of care.

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In-Vivo Gene Therapy Gene Trials 98

Scienmag

NOVEMBER 12, 2020

Gong Chen at Jinan University, Guangzhou, China recently reported the first non-human primate study demonstrating successful in vivo neural regeneration from brain internal glial cells for stroke repair. Credit: Jinan University Stroke is a leading cause of death and severe long-term disability with limited treatment available.

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Gene Therapy In-Vivo Gene Research 98

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”

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Gene Editing Gene In-Vivo Gene Therapy 147

The Pharma Data

JANUARY 17, 2021

LONDON–( BUSINESS WIRE )– Ixaka Ltd , an integrated cell and gene therapy company focused on the natural power of the body to cure disease, launches today. The new business will continue to develop Ixaka’s proprietary technologies – concentrated multi-cell therapies (MCTs) and targeted nanoparticle (TNP) therapeutics.

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Gene Therapy Gene In-Vivo Genetics 40

Drug Discovery World

MARCH 7, 2024

Ginkgo Bioworks has acquired Patch Biosciences, with the intention to strengthen its gene therapy services, cell therapy services, and RNA therapeutics services. The collaboration achieved its goals of enhancing the AAV production titres of Biogen’s gene therapy manufacturing processes.

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Pharmaceutical Technology

MAY 18, 2023

Takeda oncology drug discovery unit head Kathy Seidl stated: “We are encouraged by KSQ’s CRISPRomics platform and its ability to perform in vivo genetic screens for the discovery and validation of tumour targets, which have the potential to modulate the innate and adaptive immune system. “We

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Roots Analysis

FEBRUARY 27, 2024

How is the genome editing market landscape evolving: Currently, there is an evident increase in demand for complex biological therapies (including regenerative medicine products), which has created an urgent need for robust genome editing techniques. The global genome editing market is anticipated to grow at a CAGR of 12.6%

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Genome Genomics Gene Gene Editing 40

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. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

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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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Gene Editing Gene Gene Therapy In-Vivo 145

Drug Discovery World

AUGUST 15, 2023

Reliable analytical methods such as digital PCR to characterise lentiviral vectors and the cells transduced with them are critical to helping ensure the safety and efficacy of these biologics. The post New whitepaper explores digital PCR for lentiviral vector characterisation appeared first on Drug Discovery World (DDW).

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Gene Therapy In-Vivo Gene DNA 52

Pharmaceutical Technology

MAY 24, 2023

The TME modulators have been designed for addressing one of the main causes of acquired resistance to immunotherapies, including checkpoint inhibitors or cellular therapies such as CAR-T. It is building many MDSC-targeted, bi-functional ADCs for in vitro and in vivo characterisation, with lead selection expected by the end of next year.

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Gene Therapy In-Vivo In-Vitro Immune Response 130

Drug Discovery World

MAY 1, 2023

Download Emulate’s Introduction to Organ-on-a-Chip Technology eBook to learn about: What Organ-Chips are, how they work, and why they are transforming the way drugs are discovered and developed How Organ-Chips overcome the main challenges of conventional in vivo and in vitro models The wide variety of insights and endpoint analyses Organ-on-a-Chip (..)

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In-Vivo In-Vitro Gene Therapy Gene 52

Drug Discovery World

MAY 29, 2023

Gene delivery technologies company VectorBuilder has opened a new office in Edinburgh, hoping to bolster its presence in the United Kingdom. The new location is part of Edinburgh Technopole, a Pioneer Group in Midlothian Science Zone.

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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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Delveinsight

FEBRUARY 11, 2021

Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer. Immune-stimulating antibodies, which target the receptor CD40, are emerging as promising therapies for pancreatic cancer. Avrobio gene therapy eradicates toxic substrate in Fabry patient.

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Gene Therapy Gene Immune Response In-Vivo 52

Drug Discovery World

MARCH 13, 2023

Perhaps most interestingly this week, after previous caution over in vivo gene therapy trials, the FDA has given the go ahead to Intellia Therapeutics to study NTLA-2002 in hereditary angioedema (HAE).

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Gene Therapy Drugs In-Vivo Clinical Trials 52

Drug Discovery World

DECEMBER 6, 2023

“We are impressed by the unique delivery and editing technologies that Eligo has developed, and we believe this will set the stage for Eligo to forge new paths in the application of in vivo gene-editing technologies.” The investment will fuel pre-IND and IND activities to achieve early human data readouts in a PhIb/IIa clinical trial.

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Gene Editing Gene In-Vivo Genetics 52

Drug Discovery World

MARCH 10, 2023

Perhaps most interestingly this week, after previous caution over in vivo gene therapy trials, the FDA has given the go ahead to Intellia Therapeutics to study NTLA-2002 in hereditary angioedema (HAE). The post This week in drug discovery (6-10 March) appeared first on Drug Discovery World (DDW).

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In-Vivo Clinical Trials Clinical Trials Drugs 52

Pharmaceutical Technology

SEPTEMBER 29, 2022

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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