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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Generic Drugs Development Drugs Manufacturing 87

Pharmaceutical Technology

SEPTEMBER 14, 2022

As an autologous cell therapy, manufacturing the infusion product is a highly specialised process involving tumour harvest and the extraction and large-scale ex vivo expansion of TILs. Tumour-infiltrating lymphocyte (TIL) therapy has been investigated as a treatment modality for melanoma for almost 20 years. versus 21.4% respectively.

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Immune Response In-Vivo Production Manufacturing 246

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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Gene Editing Gene Gene Therapy In-Vivo 145

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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In-Vitro In-Vivo Sales Clinical Trials 279

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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Gene Editing Gene In-Vivo Gene Therapy 147

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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Gene Therapy Gene Development Manufacturing 130

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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Manufacturing In-Vivo Development 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). 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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Gene Editing In-Vivo Gene Sequencing Gene 97

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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RNA Vaccine Vaccination In-Vivo 147

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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Development In-Vivo Drugs Clinical Trials 130

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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Bioequivalency Production Genotoxicity In-Vivo 169

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.

Gene Editing 130

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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Drugs In-Vivo In-Vitro Contract Manufacturing 99

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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Engineer In-Vivo FDA Approval Manufacturing 105

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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In-Vivo Gene Manufacturing Marketing 52

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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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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Gene Therapy FDA Approval Gene Clinical Trials 95

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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Life Science In-Vivo Manufacturing Gene Therapy 59

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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Production Bioavailability In-Vivo In-Vitro 133

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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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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Gene Editing Manufacturing Gene In-Vivo 52

pharmaphorum

NOVEMBER 24, 2021

The US biotech will cover R&D costs as well as development, regulatory, manufacturing and sales for projects that progress. The platform has already attracted other biopharma groups including Roche , which paid $200 million upfront last year for rights to an individualised cancer vaccine with another $515 million at the back end.

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The Pharma Data

DECEMBER 9, 2020

The Chinese biopharmaceutical industry is growing in leaps and bounds, but there is still a huge unmet need when it comes to getting patients access to the breakthrough therapeutic modalities and platforms like RNAi, cell and gene therapy and others. A solution could be at hand with Overland Pharmaceuticals.

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In-Vivo In-Vitro Antibody Development 52

WCG Clinical

MAY 28, 2024

The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies. These therapies are manufactured by extracting T cells from the patient’s blood, genetically modifying them in the lab, and then reinfusing them back into the same donor patient. In these cases B cell depletion is a feature, not a bug.

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In-Vivo Clinical Trials Clinical Trials FDA Approval 40

XTalks

DECEMBER 13, 2023

XTALKS WEBINAR: Cryopreservation Best Practices for Cell & Gene Therapy Source Material Live and On-Demand: Monday, January 22, 2024, at 11am EST (4pm GMT/UK) Register for this free webinar to learn about cryopreservation, a vital step in the manufacturing of today’s cell and gene therapies. How do Casgevy and Lyfgenia Work?

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Gene Therapy Gene FDA Approval In-Vivo 98

pharmaphorum

JANUARY 17, 2023

In addition, the construction of a specialised manufacturing facility for mRNA vaccines was agreed. In addition, the construction of a specialised manufacturing facility for mRNA vaccines was agreed. The intracellular barriers include endosomal escape, RNA sensors, and endonucleases.

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XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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Gene Therapy Gene Trials Clinical Trials 95

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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The Pharma Data

OCTOBER 15, 2020

He is currently a professor at the University of Copenhagen where his lab focuses on PNAs in regard to drug discovery, gene targeting, antisense principles, cellular and in vivo delivery and administration of biopharmaceuticals. Nardi previously served as vice president of Worldwide Manufacturing at Iradimed Corporation.

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The Pharma Data

JUNE 7, 2023

Acuitas’ LNP technology will support Bayer’s in vivo gene editing and protein replacement programs by specifically delivering RNA payloads to the desired target organ, the liver. a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics. Source link: [link]

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Gene Therapy Gene Gene Editing In-Vivo 40

Roots Analysis

JANUARY 14, 2024

With the increased interest and gradual shift of investment from small molecule drugs to biologics and the establishment of several biologics manufacturing companies / biologics CMOs, more than 250 biologic therapies and vaccines have been developed, globally. They are different from small molecules in terms of their size and complexity.

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Contract Manufacturing Protein Manufacturing Production 40

XTalks

FEBRUARY 27, 2024

TIL vs CAR T-Cell Therapies While CAR T-cell therapies involve inserting a CAR engineered to target a specific cancer cell protein into a patient’s T cells, TIL-based T-cell therapies like Amtagvi involve separating TILs from a patient’s tumor (in autologous therapies) and expanding them ex vivo.

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FDA Approval Manufacturing In-Vivo Trials 52

Roots Analysis

AUGUST 8, 2023

Introduction to Liposome Liposome was initially identified and described as swollen phospholipid systems, called Banghasomes, by British hematologist Alec D. Bangham, in 1961 at the Babraham Institute, University of Cambridge. The various therapeutic applications of liposomes in drug delivery have been highlighted in the figure.

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Drug Delivery Systems Drug Delivery Drugs In-Vivo 52

XTalks

AUGUST 17, 2020

This typically involves taking T cells from the body, engineering chimeric receptor antigens (CAR) and inserting them into the cells, expanding the cells ex vivo and injecting them back into the body. Cell-Based Immunotherapy vs. Immune Biologics. Immunotherapy involves leveraging components of the immune system (i.e.

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Protein Engineer Immune Response In-Vivo 98

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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Gene Editing Gene In-Vivo Genetics 52