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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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. Moderna will also assume further development, manufacturing, and commercialisation responsibilities on exercising a target option.

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Pharmaceutical Technology

OCTOBER 14, 2022

The two companies will aim to improve the manufacturing and analytic procedures used to develop personalised cell and gene therapies to treat cancer patients. Together, our objective is to advance the development and deployment of cell therapies, such as CAR-T and tumour-infiltrating lymphocyte technologies, to treat solid tumours. "We

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Drug Patent Watch

JANUARY 14, 2025

Case Study 1: Overcoming Formulation Challenges Let’s kick things off with a look at how generic drug manufacturers are tackling formulation challenges head-on. Generic drug manufacturers are taking a similar approach by developing multiple drugs simultaneously[1]. The results?

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BioPharma Reporter

APRIL 4, 2023

Sensible Biotechnologies will partner with Ginkgo Bioworks to develop an in vivo microbial mRNA manufacturing platform â aiming to address a major bottleneck in mRNA manufacturing.

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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). Other companies – notably Vertex Pharma/CRISPR Therapeutics and bluebird bio – are taking an ex vivo approach to SCD and thalassaemia and are much closer to market.

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Pharmaceutical Technology

AUGUST 17, 2022

oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. . These programmes will include therapies and vaccines in infectious disease and oncology areas.

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Pharmaceutical Technology

MAY 31, 2023

In vivo and in vitro pre-clinical studies demonstrated that CFT8919 provides wider coverage of on-target resistant mutations and intracranial activity, indicating its potential to be effective against brain metastases. Betta will be responsible for the development, manufacture and commercialisation of CFT8919 in the licensed territories.

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Pharmaceutical Technology

FEBRUARY 13, 2023

Best practice is to also consider the ‘developability’ of the lead compounds in conjunction with any evidence of potential in-vivo activity, by taking a holistic approach. With all this in mind, how do drug developers know which strategy and approach is right for their molecule at this early stage?

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Camargo

DECEMBER 22, 2021

In addition to prioritizing programs most closely aligned with sponsor objectives, a portfolio analysis can help prevent a program from failing due to misalignment of the various factors that affect drug development, including clinical, nonclinical, regulatory, scientific, manufacturing, and market dynamics. Clinical Pharmacology.

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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). Blood concentrations of Lp(a) are determined almost entirely by inheritance.

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Gene Editing Gene Gene Therapy In-Vivo 130

pharmaphorum

AUGUST 10, 2020

Originally intended as a treatment for Ebola virus, Veklury is a nucleotide analogue with broad-spectrum antiviral activity both in vitro and in vivo in animal models against several emerging viruses. Pfizer will provide contract manufacturing services at its facility in McPherson, Kansas, to supply the drug to Gilead.

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pharmaphorum

APRIL 25, 2022

At the moment, it can take weeks to complete the CAR-T process, which includes harvesting of T cells from the patient an shipment to a manufacturing unit, engineering, activation, and expansion of the cells, quality control checks and shipment back to the hospital for re-infusion into the patient.

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pharmaphorum

OCTOBER 8, 2021

This year, we’ll be specifically focusing on next generation cell therapies and discussing: Novel in vivo delivery. Be part of the conversation as we explore novel in-vivo delivery and leverage innovative developments from early discovery through to manufacturing to bring to market the next generation of cell therapies.

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Roots Analysis

SEPTEMBER 20, 2023

The focus of stakeholders has now shifted to optimizing the cell therapy manufacturing process. The focus of stakeholders has now shifted to optimizing the cell therapy manufacturing process. It is worth noting that manufacturing process biologics and cell therapies is considerably complex when compared to small molecule drugs.

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Pharmaceutical Technology

FEBRUARY 23, 2023

Biopharmaceutics is a scientific discipline that examines the interrelationship of the physicochemical properties of the drug, the dosage form in which the drug is given, and the route of administration on the rate and extent of systemic drug absorption ( Applied Biopharmaceutics and Pharmacokinetics, Shargel, Wu-Pong and Yu, 5th Edition ).

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XTalks

AUGUST 19, 2022

The cells are prepared outside of the body ( ex vivo ) and then infused into the patient. Bluebird has a total of three gene therapies in its pipeline. Along with beti-cel, there is one being developed for sickle cell (approved in Europe as Skysona or eli-cel) and another being jointly developed with Bristol Myers Squibb for melanoma.

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pharmaphorum

MAY 27, 2021

Life sciences tools company Esco Lifesciences Group has scored $200m in a series A and crossover round co-led by Novo Holdings and Vivo Capital. Singapore-based Esco manufactures laboratory and biopharma equipment and IVF medical devices for supply in over 100 countries.

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Camargo

FEBRUARY 10, 2021

Drug development is an extremely cumbersome process, requiring the testing of an agent from in vitro studies to in vivo studies to in silico modeling. Given that it can take up to 20 years for a final product to be approved, it is unsurprising that drug attrition rates are very high. Approaches to Pharmacokinetic Analysis.

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The Pharma Data

JANUARY 17, 2021

Joe Dupere, CEO of Ixaka, commented: “Ixaka’s broad offering of integrated cell and gene therapy capabilities, encompassing cell-based products and an innovative in vivo gene delivery platform, provides a strong foundation for our ambitions to become a leader in cell and gene therapies. A total of $15.4

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Gene Therapy Gene In-Vivo Genetics 40

XTalks

SEPTEMBER 20, 2024

Vittoria’s investigational CAR-T therapy leverages gene editing to modulate the CD5 signaling pathway and a shorter manufacturing time to enhance efficacy and improve manufacturing efficiencies and vein-to-vein time. Such challenges highlight a significant unmet need and call for further advancements in the field.

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The Pharma Data

DECEMBER 20, 2020

. PARTNERSHIP LEVERAGES COMPLEMENTARY STRENGTHS OF IN SILICO, IN VITRO AND IN VIVO ANTIBODY DISCOVERY IN A RANGE OF INDICATIONS. Alloy’s first platform, the ATX-Gx(TM) mouse platform, is a suite of transgenic mice designed for best-in-class in vivo discovery of fully human monoclonal antibodies.

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The Pharma Data

MARCH 2, 2021

As a world-leading, research-driven pharmaceutical company, more than 51,000 employees create value through innovation daily for our three business areas: Human Pharma, Animal Health, and Biopharmaceutical Contract Manufacturing. Senior Vice President and Global Head of Cardiometabolic Diseases Research, Boehringer Ingelheim.

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pharmaphorum

DECEMBER 6, 2020

CTX001 is an ‘ex vivo’ application of gene-editing, in which the technology is used to modify a patient’s own cells outside the body. Similarly, three SCD patients suffered none of the characteristic painful attacks – known as vaso-occlusive crises (VOCs) – in the three to 15-month follow-up period after the treatment.

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The Pharma Data

DECEMBER 13, 2020

The FDA also recommended that Gamida Cell generate additional manufacturing-related data prior to requesting a pre-Biologics License Application (BLA) meeting. Specifically, the FDA requested that Gamida Cell demonstrate analytical and clinical comparability from the company’s planned commercial manufacturing sites. About Omidubicel.

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XTalks

FEBRUARY 10, 2023

The most common factors associated with delays were insufficient non-clinical development and chemistry, issues with manufacturing and controls (CMC), clinical trial design problems, inadequate site and vendor feasibility, and regulatory authority submission delays. Among 20 recent trials, 17 experienced delays in meeting key milestones.

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FDA Law Blog

OCTOBER 10, 2023

Implant devices are used in a range of settings such as dental and orthopedic procedures. Having a clear understanding of FDA’s data expectations for 510(k) applications related to implants is crucial for successful marketing clearance. The intended duration of implantation is another crucial factor in performance testing.

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Delveinsight

FEBRUARY 11, 2021

Avrobio designed the Plato platform to optimize vector copy numbers, personalize the conditioning regimen, aid automated manufacturing and otherwise promote and industrialize its lentiviral vector-based approach to treating genetic diseases. Enhancing the response to immuno-oncology in pancreatic cancer.

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The Pharma Data

JANUARY 12, 2021

We are planning to manufacture doses for around two million patients in 2021 and hope to improve access to this treatment option for those that require.”. CT-P59 treatment groups also reported with significantly shortened time to clinical recovery ranging from 3.4 days quicker compared to placebo. days; 95% C.I, days compared to placebo [5.7

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The Pharma Data

APRIL 22, 2023

“To our knowledge, this study is the first to show in vivo [in a living organism] protection against severe COVID-19 by an AI-designed T-cell vaccine,” said Girish Kirimanjeswara, associate professor of veterinary and biomedical sciences, Penn State. The results were published on April 11 in Frontiers in Immunology.

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The Pharma Data

DECEMBER 10, 2020

.–( BUSINESS WIRE )– ImmunityBio , a privately-held immunotherapy company, today announced its COVID-19 vaccine candidate protected nasal and lung airways of non-human primates against coronavirus (SARS-CoV-2) in a challenge study. This blocking of viral replication was observed in both the lung and nasal passages.

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The Pharma Data

DECEMBER 27, 2020

Changes in heparan sulfate (HS) concentration in cerebrospinal fluid (CSF) are being monitored in patients treated with LYS-SAF302 to provide evidence of in vivo biological activity of the drug and demonstrate proof of concept. Average reductions were highly statistically significant. About Lysogene. Sarepta Therapeutics, Inc.

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Roots Analysis

SEPTEMBER 13, 2023

The synergistic behavior achieved from the process circumvents the inherent limitation of the individual counterparts, such as short in vivo half-life, low solubility and immunogenicity. It consists of somatostatin-derived peptide and DOTA complexed with radioactive isotope 77 Lu.

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The Pharma Data

OCTOBER 22, 2020

The drug is now widely available in hospitals across the country, following early investments to rapidly expand manufacturing capacity to increase supply. Previously authorized by the FDA for emergency use to treat COVID-19, Veklury is now the first and only approved COVID-19 treatment in the United States.

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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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Camargo

SEPTEMBER 30, 2021

The drug development process can be broken down into three main disciplines: chemistry, manufacturing, and control (CMC); nonclinical; and clinical. CMC programs include drug substance manufacturing process scale-up, stability testing, early formulation development, and, later on, manufacturing process validation, among other considerations.

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pharmaphorum

JULY 22, 2021

As soon as the COVID-19 outbreak began, we implemented an emergency preparedness plan which leveraged this experience and our unique antibody discovery, development and manufacturing processes to identify a potential treatment.”. In just over a year, Celltrion Healthcare says it has identified and delivered a potential treatment for COVID-19.

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