Pharmaceutical Technology

APRIL 13, 2023

The company is aiming to refocus its efforts on its core therapeutic areas and current late-stage programmes such as the oral TYK2 inhibitor TAK-279, which is under development for several autoimmune disorders.

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Drug Discovery World

MAY 9, 2023

Jantzen Sperry , PhD, Director of Scientific Operations at Certis Oncology looks at how the adoption of orthotopic PDX models can elevate the impact of cancer research and improve translation in oncology drug development. Fewer than 5% of oncology drugs that enter clinical trials in the US receive US FDA approval.

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Drug Discovery World

APRIL 2, 2024

A new Case Study is now available to download for free: ‘In Vivo Evaluation Of Bispecific-Cell Engager Efficacy & Safety ‘ Discover a clinically predictive and reproducible platform developed by The Jackson Laboratory for simultaneous efficacy and safety assessment. Download now.

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Camargo

NOVEMBER 11, 2020

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. Unpacking the (Black) Box: Antares Licenses Urology Product with Boxed Warning. of hyponatremia, or low blood sodium levels. In October, the U.S.

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Worldwide Clinical Trials

NOVEMBER 27, 2023

In trials, Casgevy was shown to help prevent episodes of debilitating pain, known as vaso-occlusive crises, that often plague patients affected by sickle cell disease. In June 2023, the U.S. Food and Drug Administration (FDA) announced its acceptance of the Biologics License Application (BLA) for exa-cel.

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Pharmaceutical Technology

MARCH 29, 2023

The trial, which is investigating healthy adults with a minimum body mass index of 30 kg/m2, will see VK2735 administered as an oral tablet once daily for 28 days. The Phase I study is an extension of a previously completed Phase I trial that evaluated the subcutaneous administration of VK2735.

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Drug Discovery World

SEPTEMBER 14, 2023

The collaboration contemplates conducting preclinical studies and a Phase I clinical trial evaluating the safety and efficacy of the combination with the objective of further enhancing IMA203 T cell responses. Terms of the agreement Under the terms of the agreement, Immatics will receive an upfront payment of $120 million.

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Development Vaccination Vaccine In-Vivo 52

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). billion in potential milestones if the project advances through development and onto the market. billion agreement that started in 2020.

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pharmaphorum

JANUARY 13, 2021

IO Biotech, an oncology specialist formed and backed by Denmark’s Novo Holdings, has raised €127 million ($155 million) to further develop its cancer vaccine technology that has boosted efficacy of PD-1 immunotherapy in early trials. The post IO Biotech raises $155m to develop breakthrough cancer vaccine appeared first on.

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XTalks

APRIL 24, 2023

Rare disease clinical trials are complex due to the additional scientific, medical, operational and regulatory requirements of newly emerging advanced therapies, such as gene therapy,” says Dr. “Thankfully, we can anticipate that many more of these new treatments are expected in the future.”

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Drug Discovery World

JUNE 30, 2023

Research tools, safety testing and regenerative medicines – these endless stem cell applications are powering precision, speed, and new modalities in drug development. Stem cells for use in drug discovery Stem cells are fast becoming an invaluable tool in the drug discovery process.

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Drug Discovery World

MARCH 8, 2023

BAT8008 was developed by using Bio-Thera Solutions’ ADC linker-payload that includes a cleavable but systemically stable linker, a small molecule topoisomerase I inhibitor and high DAR. The small molecule topoisomerase I inhibitor payload carried by BAT8008 has a strong cell membrane penetration ability.

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Pharmaceutical Technology

JULY 14, 2022

AVR-RD-05 makes use of the own hematopoietic stem cells (HSCs) of a patient that are transduced ex vivo with a lentiviral vector encoding the human IDS enzyme. Next year, Avrobio is expected to initiate a collaborator-sponsored Phase I/II clinical trial for Hunter syndrome in partnership with the University of Manchester, UK.

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Gene Therapy Gene Drugs In-Vivo 130

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

Drug Discovery World

FEBRUARY 20, 2024

One example is that the model can circumvent the difficulties in demonstrating in vivo drug efficacy which arise because of mouse specific metabolism issues. Edinburgh-based PhaSER Biomedical (PhaSER) has received a Bill & Melinda Gates Foundation grant totalling $2.3m A key stage of compound progression. The post $2.3m

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Pharmaceutical Technology

SEPTEMBER 14, 2022

The company is currently progressing its lead drug candidate, RYZ101, into clinical trials for various solid tumour indications. A Phase Ib clinical trial of the drug is underway, enrolling subjects with neuroendocrine tumours, and Phase III trials are anticipated to commence next year.

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Roots Analysis

FEBRUARY 27, 2024

The biopharmaceutical pipeline includes close to 500 gene therapies , several of which are being developed based on CRISPR technology. Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated. Recently, in July 2019, a first in vivo clinical trial for a CRISPR-based therapy was initiated.

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Drug Discovery World

SEPTEMBER 29, 2023

Immunotherapy has taken centre stage this week, with several announcements relating to regulatory approval, investment and research results for antibody and CAR-T therapies, perhaps most significantly, encouraging early-stage data for the first in vivo CAR-T therapy. The top stories: €27.3M

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In-Vivo Drugs Antibody Clinical Trials 52

Pharmaceutical Technology

APRIL 28, 2023

Earlier in the year, the company had announced a move towards developing treatments for hemoglobinopathies like sickle cell disease and beta thalassemia and a focus on in vivo discovery. The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022. g/dL and 45.4%

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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

Pharmaceutical Technology

MAY 24, 2023

Our board and management team believe that the combined company will be well-positioned to develop powerful new therapies with the potential to overcome resistance to current immunotherapies, an area of significant unmet need.” Morphogenesis’ lead personalised cancer vaccine, IFx-Hu2.0,

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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. The proposed transaction will conclude in the second half of 2023.

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Gene Therapy Gene In-Vivo Life Science 130

Drug Discovery World

APRIL 15, 2024

Melissa Little, CEO of the Novo Nordisk Foundation Centre for Stem Cell Medicine “In the stem cell space, 2023 has seen significant increases in the delivery of pluripotent stem cell-derived cell types into first-in-human clinical trials. Gene therapy will move to ex vivo gene modification and the delivery of gene-modified cell types.

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Pharmaceutical Technology

OCTOBER 5, 2022

In 2010, two patients with end-stage refractory chronic lymphocytic leukaemia (CLL) were administered Novartis’ autologous anti-CD19 chimeric antigen receptor T-cell (CAR-T) therapy, Kymriah (tisagenlecleucel), as part of a Phase I trial.

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Drug Discovery World

JANUARY 31, 2024

Breye Therapeutics, a clinical-stage biopharmaceutical company developing novel oral therapies for retinal vascular diseases within ophthalmology, has started a Phase Ib/IIa clinical trial to investigate danegaptide, following oral administration, in patients suffering from diabetic macular oedema (DMO).

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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. While the main thrust of the new alliance is finding in vivo therapies, Bayer said it is also working with Mammoth on ex vivo projects “on a nonexclusive basis.”

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In-Vivo Gene Editing Gene Therapy Gene 52

Drug Discovery World

FEBRUARY 14, 2024

AlphaMedix is a targeted alpha therapy currently in Phase II clinical development, which consists of an SSTR-targeting peptide complex radiolabeled with lead-212 (212Pb) that serves as an in vivo generator of alpha particles. In the Phase I study, treatment was well-tolerated, with a response rate of 62.5%

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XTalks

MAY 19, 2022

Researchers at the Georgia Institute of Technology have recently developed a new implantable vascular monitoring system consisting of a stent and soft sensors. XTALKS WEBINAR: Cardiac Safety Solutions in Decentralized Clinical Trials (DCT). The smart stent developed by Dr. Yeo and his research team is wireless and batteryless.

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Drug Discovery World

APRIL 8, 2024

Aviv Regev, Head and Executive Vice President of Research and Early Development at Genentech, opened the session with a discussion of how advances in genomic sequencing are fostering the development of data-rich cell atlases and what that data could mean for new insights into cancer biology and novel therapeutics.

In-Vivo 52

In-Vivo In-Vitro Antibody Protein 52

Drug Discovery World

MARCH 15, 2024

million to accelerate development of its lead drug candidates in the area of mitophagy, MTX325 and MTX652, through clinical trials. from our investors, we can now make the next vital steps, progressing with essential clinical trials.” Mission Therapeutics has raised £25.2 ($32.1) Thanks to this additional £25.2m

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Clinical Trials Clinical Trials In-Vivo Trials 52

Drug Discovery World

JANUARY 18, 2024

Poolbeg Pharma, a biopharmaceutical company focused on the development and commercialisation of medicines targeting diseases with a high unmet medical need, has announced promising in vivo results for POLB 001 in addressing cancer immunotherapy-induced cytokine release syndrome (CRS). million cases of cancer by 2030 1,2.

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Drug Discovery World

MARCH 26, 2024

DDW’s Megan Thomas caught up with Oliver Rausch, Chief Scientific Officer at Storm Therapeutics, following the ESMO Targeted Anticancer Therapies Congress 2024, a global meeting focusing on promising new anticancer targets and agents, focusing on those in early phase clinical development. MT: What data did you present at the conference?

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Drug Discovery World

OCTOBER 25, 2023

iOnctura, a clinical-stage biotech developing selective cancer therapies against targets that play critical roles in multiple tumour survival pathways, has published new research data in the peer-reviewed journal Cancer Research, that supports a strategy of combining autotaxin inhibitor IOA-289 with TGF-β pathway inhibitor IOA-359.

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Drug Discovery World

APRIL 10, 2024

iECURE has been given the go-ahead to begin US trials of its investigational gene editing-based therapy in newborn males with neonatal onset Ornithine Transcarbamylase (OTC) deficiency. The post FDA approves first in vivo gene insertion programme in infants appeared first on Drug Discovery World (DDW).

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The Pharma Data

MARCH 8, 2021

This program included assays such as Big Blue and PIG-a designed to provide a robust measure of a drug or chemical’s ability to induce mutations in vivo. The totality of the data from these studies indicates that molnupiravir is not mutagenic or genotoxic in in vivo mammalian systems. “We

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Camargo

DECEMBER 22, 2021

Informed by today’s competitive drug development landscape, as well as by the sponsor’s strategic goals and capabilities, a portfolio analysis involves assessing product concept and differentiation, strategic planning, factoring in unmet patient needs, and estimating timelines and expenses. Clinical Pharmacology. Clinical Safety and Efficacy.

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pharmaphorum

JANUARY 29, 2021

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

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