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

Drug Discovery World

JANUARY 5, 2024

In non-human primate studies, the therapy has demonstrated effective, durable, and well tolerated in vivo CAR-T cell generation. In non-human primate studies, the therapy has demonstrated effective, durable, and well tolerated in vivo CAR-T cell generation.

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In-Vivo Development Licensing Licensing 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

Drug Discovery World

MARCH 7, 2024

The collaboration achieved its goals of enhancing the AAV production titres of Biogen’s gene therapy manufacturing processes. The collaboration achieved its goals of enhancing the AAV production titres of Biogen’s gene therapy manufacturing processes.

Gene Therapy 52

Gene Therapy RNA Engineer Gene 52

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 Vaccination Vaccine In-Vivo 147

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

Drug Discovery World

AUGUST 15, 2023

Reliable analytical methods such as digital PCR to characterise lentiviral vectors and the cells transduced with them are critical to helping ensure the safety and efficacy of these biologics. The post New whitepaper explores digital PCR for lentiviral vector characterisation appeared first on Drug Discovery World (DDW).

Gene Therapy 52

Gene Therapy In-Vivo Gene DNA 52

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

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

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

Drug Discovery World

AUGUST 14, 2023

A Mutual Recognition Agreement (MRA) 2 for inspections of manufacturing sites for certain human medicines, signed and implemented by the United States and EU in July 2019, served as an important milestone in harmonising certain regulatory aspects in developing a global drug. billion in 2020 to $15.5 billion in 2025 at a rate of 28.7%

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Gene Therapy Marketing Gene DNA 52

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 Manufacturing Protein Bacteria 40

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

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 Contract Manufacturing In-Vitro 99

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

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

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

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

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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Vaccination Vaccine In-Vivo Manufacturing 52

Drug Discovery World

JUNE 14, 2023

In subsequent articles, themes cover future regulatory changes and redefining manufacturing. The ISCT 2023 Annual Meeting took place in Paris, France from May 31 to June 3 2023. DDW’s Megan Thomas heard from attendees about key trends in the sector. First, the importance of cell collections. Next, attendees discuss the trend of allo vs auto.

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In-Vivo Gene Manufacturing Development 52

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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In-Vivo Manufacturing Engineer Genotoxicity 45

Drug Discovery World

APRIL 10, 2023

India is traditionally known as ‘the pharmacy of the world’ due to its huge pharmaceuticals manufacturing capacity and high quantity of exports, particularly generic medicines and vaccines. It is emerging markets like India that analysts expect to spearhead the global growth of the pharmaceutical sector 1.

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Genome Genomics Drugs Life Science 52

Drug Discovery World

SEPTEMBER 6, 2022

This area of medicine, which turns our own bodies into agents of combat to fight disease, has quickly become one of the most promising fields in treating deadly diseases such as cancer, central nervous system disorders and even musculoskeletal conditions. . Last year saw the sector surge, with levels of investment hitting an all-time high at $22.7

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

Drug Discovery World

AUGUST 22, 2023

In many cases, it is also challenging to apply traditional bioequivalence methods or to ensure clarity about the comparison model (in-vitro/in-vivo) needed to gain health authority approval. Off-patent medicines today account for about 80% of global prescriptions at an estimated 20% of the total cost. What are complex generics?

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Production Bioequivalency In-Vivo In-Vitro 52

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

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 FDA Approval Gene In-Vivo 98

Drug Discovery World

JULY 22, 2022

In vivo models, particularly HIS models, allow preliminary investigation of cell-based therapeutic strategies—often in combination with other therapeutic modalities—to overcome the limitations and challenges of T cell-based immuno-oncology treatments and identify effective options for clinical investigation. Jespersen et al.

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In-Vivo Engineer In-Vitro Antibody 52

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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In-Vivo Contract Manufacturing Bioinformatics Clinical Development 52

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 Bioequivalency 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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Antibody Clinical Trials Clinical Trials Trials 105

Drug Discovery World

AUGUST 3, 2022

CDMO in peptides and oligonucleotides manufacturing, Bachem, has started building a new future for oligonucleotides: solving the industry’s main challenges around capacity, sustainability and cost-effectiveness. Currently, only a small number of CPP-based oligonucleotide delivery examples have been reported for in vivo application.

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Bioavailability In-Vivo RNA In-Vitro 52

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

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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Vaccination Vaccine In-Vivo In-Vitro 97

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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Licensing Licensing Clinical Trials Clinical Trials 52

Drug Discovery World

MARCH 12, 2024

Secondly, the programme has supported a focus on scientific excellence, which enabled us to release our study on the manufacturing of Velban from our engineered yeast cell factories as a proof-of- concept documenting Biomia’s biosynthesis platform. RA: Biomia is developing drug candidates for pain, addiction and mental health conditions.

Medicine 57

Medicine Production Drugs In-Vivo 57

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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Vaccination Vaccine Protein Antibody 40

Drug Discovery World

SEPTEMBER 14, 2023

Business updates Pfizer reported second quarter 2023 revenues totalling $12.7 billion, a decrease of $15.0 billion, or 54%, compared to the prior year quarter, reflecting an operational decline of $14.7 Vaccines were up 9.1%

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Drugs Vaccination Vaccine Medicine 57