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. . Vertex is also offering up to $1.3

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pharmaphorum

SEPTEMBER 29, 2020

In 2015, Adaptimmune and Universal Cells signed an agreement to drive the development of technologies leveraging gene-edited Induced Pluripotent Stem Cell (iPSC) lines, towards the development of allogeneic, or ‘off-the-shelf’, T-cell therapies.

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Pharmaceutical Technology

MARCH 1, 2023

Nedisertib is under clinical development by Merck and currently in Phase I for Neuroendocrine Tumors. Merck also provides a wide range of products including lab water systems, gene editing tools, cell lines, antibodies and end-to-end systems. The company serves healthcare, performance materials, and life sciences markets.

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Pharmaceutical Technology

MARCH 1, 2023

Nedisertib is under clinical development by Merck and currently in Phase I for Head And Neck Cancer Squamous Cell Carcinoma. Merck also provides a wide range of products including lab water systems, gene editing tools, cell lines, antibodies and end-to-end systems.

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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.

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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., a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics. Financial details were not disclosed.

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Worldwide Clinical Trials

MAY 30, 2024

Differences in regulatory sentiments and industry sponsors’ subsequent clinical development strategies historically restricted access based on geography. Additional research is underway using gene editing via CRISPR, primarily investigating genes with known relationships to ALS, such as SOD1 and C9orf72.

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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 He received his B.S. degree from Henan Normal University, M.S. degree from Chinese Academy of Sciences and Ph.D.

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The Pharma Data

JANUARY 12, 2021

As a result, the company settled on four areas of focus – iPSC, allogenic CAR-T, genome mutations and gene editing. Each of the companies met strict criteria that includes assets in clinical development, an industry-leading platform and in-house manufacturing capabilities. The platform is already bearing fruit.

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Pharmaceutical Technology

MAY 26, 2023

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. Rapid production will potentially enable researchers to overcome an industry bottleneck.

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Pharma Marketing Network

DECEMBER 21, 2020

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. Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e.,

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The Pharma Data

JANUARY 10, 2021

EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. Previously, the Company received Rare Pediatric Disease designation from the FDA for EDIT-301.

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Delveinsight

SEPTEMBER 14, 2021

VOR33 was created through Vor’s genome eHSC platform combined with multiple therapeutic modalities to create treatment-resistant transplants that can improve the potential of targeted cancer therapies through cell therapy and gene editing. REX-001 is an MCT product in clinical development as patient-specific cellular immunotherapy.

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The Pharma Data

DECEMBER 2, 2020

ERS Genomics – Ireland’s ERS Genomics Limited, and Germany’s Vivlion GmbH, announced a non-exclusive license agreement granting Vivlion access to ERS Genomics’ CRISPR/Cas9 patent portfolio, to enhance Vivlion’s gene editing reagents and screening services. for its cGMP-compliant CHOSOURCE platform. in Mainland China.

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Delveinsight

MARCH 2, 2021

To sum it, Chinook will manage clinical development and commercialization of product candidates while Evotec will be eligible for an undisclosed upfront payment, research funding, progress-dependent milestone payments, and tiered royalties on net sales for targets identified through the collaboration.

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The Pharma Data

NOVEMBER 3, 2020

According to the FDA, the purpose of Fast Track designation is to get important new drugs to patients earlier by facilitating the development, and expediting the review, of drugs to treat serious conditions and fill an unmet medical need. LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies.

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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.

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The Pharma Data

JANUARY 17, 2021

To complement the internal capabilities, Aruvant is partnering with world-leading contract development manufacturing organizations. The strategic long-term manufacturing agreement with Lonza is a critical step to advancing the clinical development of ARU-1801 for sickle cell patients.

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XTalks

FEBRUARY 10, 2023

Jeff Vassallo, Senior Director of Clinical Trial Management, at the global clinical contract research organization (CRO) Medpace. These details must be thoughtfully considered early in clinical development to ensure studies are completed within the projected timeline and budget,” added Dr. Vassallo.

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The Pharma Data

DECEMBER 27, 2020

Notably, we never modify the DNA of these cells, which avoids some of the safety concerns that have been reported with certain gene-editing technologies. From these cells we apply proprietary methods to manufacture pure populations of only the cell types which we wish to use in patients. Forward-Looking Statements.

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XTalks

AUGUST 2, 2023

With Blackstone Life Sciences, up to €300 million (around $315 million USD) will be invested to accelerate pivotal studies and clinical development programs for formulating the subcutaneous delivery of the anti-CD38 antibody Sarclisa (isatuximab) for the treatment of multiple myeloma.

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XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

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XTalks

JANUARY 6, 2025

As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas. While these therapies span all stages of clinical development, they are particularly concentrated in earlier phases, indicating strong future growth.

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