Pharmaceutical Technology

MARCH 2, 2023

QN-023a is under clinical development by Hangzhou Qihan Biotechnology and currently in Phase I for Relapsed Acute Myeloid Leukemia. The company develops gene-editing organ transplantation technology. Hangzhou Qihan Biotechnology overview Hangzhou Qihan Biotechnology is a healthcare company.

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Gene Editing Genetic Engineering Engineer Clinical Trials 100

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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Gene Editing Genetics Drugs Gene 52

Pharmaceutical Technology

MARCH 2, 2023

QN-019a is under clinical development by Hangzhou Qihan Biotechnology and currently in Phase I for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia). The company develops gene-editing organ transplantation technology.

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Gene Editing Clinical Trials Clinical Trials Genetics 100

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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Gene Therapy Gene Production Clinical Trials 52

The Pharma Data

JANUARY 12, 2021

Marianne De Backer, Head of Business Development & Licensing in Bayer’s Pharmaceuticals Division, pictured above. Also in December, the company partnered with Atara Biotherapeutics to develop off-the-shelf T-cell immunotherapies for high mesothelin-expressing tumors. Photo courtesy of Bayer. We have set the bar high.

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Gene Therapy Gene Gene Editing Licensing 52

Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD).

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Gene Editing Gene Gene Therapy In-Vivo 130

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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Gene Editing DNA Cosmetics Life Science 100

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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Gene Editing DNA Cosmetics Life Science 100

Drug Discovery World

MAY 6, 2024

A more established Amsterdam-based enterprise is gene therapy company uniQure, developer of etranacogene dezaparvovec, the first gene therapy for haemophilia B, which was approved for use in the US in November 2022 and in the EU in February 2023. The CDD will sponsor and manage KWF-supported trials.

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Drugs Life Science Gene Editing Gene 59

pharmaphorum

SEPTEMBER 18, 2020

Vertex is already the top player in CF with a quartet of exon-skipping medicines that tackle the mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that underlies the disease, but there are still a sizeable group of patients with no treatment options.

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Gene Editing Gene Vaccination Vaccine 105

pharmaphorum

SEPTEMBER 29, 2020

As of 2018, there were over 1,100 cancer therapies in development, and as of 2020, 362 of them were cell and gene therapies. Many novel therapies have been successfully developed through such partnerships. It enables the companies to work closely together, throughout the continuum of research, development and commercialisation.

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Research Gene Editing Gene Development 132

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 Prior to that, he held various oncology research and development positions at Merrimack Pharmaceuticals and Archemix.

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Business Development Development Gene Editing In-Vivo 40

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. greater safety, transient and reversible effect on gene expression).

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Genetics Medicine Gene Therapy Marketing 52

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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Manufacturing Gene Therapy Trials Gene 52

Drug Discovery World

FEBRUARY 28, 2024

CRISPR and brain cancer Meanwhile, elsewhere in the US, another exciting development – using CRISPR as a therapeutic approach – has possibilities for tackling brain cancer. Scientists have used CRISPR gene editing to target and rapidly destroy glioblastoma cells in an approach that could apply to other highly mutated cancers.

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Development Research Drugs Antibody 64

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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RNA Clinical Trials Clinical Trials Drugs 67

Drug Discovery World

JULY 3, 2023

In terms of the Bispecific and Multi-Specific Antibody Therapeutics programme, Dr Miguel Gaspar, Director, AstraZeneca, covered a breakout on T Cell redirection with bispecific antibodies, both in terms of strategies and clinical examples.

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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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Clinical Trials Clinical Trials Gene Editing Genome 52

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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Drug Delivery Research Drugs Gene Editing 40

Drug Discovery World

OCTOBER 16, 2023

Dr Salako says there are some clear fields of work associated with different areas, which include: Hertfordshire as a cell and gene therapy hub, with a number of companies in this space clustered around Stevenage. This includes support for export and expansion into new territories as well as for innovation and development.

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Drugs Life Science Gene Therapy Genome 75

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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Clinical Trials Clinical Trials Trials Gene Therapy 52

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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Manufacturing Production Development Clinical Trials 52

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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Drugs Gene Editing Genome Genomics 52

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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Medicine Development Clinical Trials Clinical Trials 52

Drug Discovery World

MAY 30, 2023

FAPi PET is being developed as a highly sensitive imaging agent and can be used as a predictive biomarker for FAP targeted therapeutics, several of which are currently in clinical development. We expect this field to grow significantly in the coming years as these and other earlier stage assets are developed.

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Research Drugs Protein RNA 52

Drug Discovery World

NOVEMBER 30, 2023

Lonza’s integrated CMC strategy and timeline was presented, as well as case studies highlighting key approaches and technologies, including: vector design & integration, cell line selection, downstream process, analytical method and formulation development. kb Influenza A hemagglutinin gene was used as a model system.

Antibody 59

Antibody Protein Engineer In-Vivo 59

XTalks

AUGUST 2, 2023

Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. 2022 was the first year on market for cell-based gene therapies Abecma (Idecabtagene vicleucel) and Breyanzi (lisocabtagene maraleucel) , which generated $388 million and $182 million, respectively. Both are anti-CD20 agents.

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Sales Manufacturing FDA Approval Life Science 98

Drug Discovery World

OCTOBER 7, 2022

To achieve this, we have brought our drug discovery sites together under a single leadership team, able to move at speed to partner with academia and industry partners to deliver novel therapies into clinical development. . RA: What’s the future outlook for Cancer Research Horizons? .

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HR Research Drugs Development 52

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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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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DNA Manufacturing Gene Therapy Gene 130