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. billion in funding. It has a vibrant community of life-science companies, from global pharma to smaller start-ups.

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The Pharma Data

JANUARY 27, 2021

Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for PBFT02, an adeno-associated virus (AAV)-delivery gene therapy that is being studied for the treatment of patients with Frontotemporal Dementia (FTD) with granulin (GRN) mutations. chief executive officer of Passage Bio. “FTD

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Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. From a technical standpoint, such diseases should be easier to treat with current gene therapy technology.

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Drug Discovery World

APRIL 29, 2024

Jean-Baptiste Agnus, Chief Business Officer, and Peter Carbone, Chief Quality Officer, KBI Biopharma: Agnus brings more than two decades of experience in the biopharmaceutical industry with expertise in biologics, small molecule APIs, antibody-drug conjugates (ADCs), and cell and gene therapies.

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The Pharma Data

MARCH 20, 2023

2,3 These data are among a Zolgensma data set being presented during the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, which also include, in part, real-world evidence data from the RESTORE registry. 7 Severity varies across a spectrum of types corresponding to the number of copies of the back-up SMN2 gene.

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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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Drug Discovery World

OCTOBER 17, 2023

This is an invaluable approach for precision medicine because it promises a detailed view of desired and undesired effects of treatments with cell and gene therapy agents or antibodies. Spatial omics technologies are ideally suited for characterising cells and their interactions within a tissue. Let’s take CAR T cells as an example.

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Drug Discovery World

NOVEMBER 13, 2023

Thereafter, there will be two luncheon presentations: Joseph Rucker, PhD, Vice President, Research & Development, Integral Molecular, on: ‘A humanised chicken antibody platform that delivers diverse and developable therapeutic candidates’.

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XTalks

AUGUST 2, 2023

Meanwhile, BioMarin’s gene therapy Roctavian is approved by the FDA for the treatment of hemophilia A. Onureg (azacitidine), a leukemia therapy, also saw large growth of 70 percent to $124 million in 2022, and the multiple sclerosis drug Zeposia (ozanimod) generated $250 million in 2022, an 87 percent increase.

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Pharmaceutical Technology

MAY 26, 2023

Enzymatic DNA production company Touchlight have augmented its DNA production capabilities with a newly announced expansion to its London facilities. Tripling its production capacity, Touchlight can now manufacture 8kg plasmid DNA, a key component for mRNA gene therapies and vaccines.

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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.

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