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

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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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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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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Drug Discovery World

DECEMBER 13, 2023

Fortis Life Sciences offers custom antibody discovery and manufacturing services for antibodies and single-domain antibodies, supporting the drug discovery market. Our entire manufacturing process is done in-house, making it customisable from start to finish. This article is sponsored by Fortis Life Sciences.

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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 A total of $15.4 References 1.

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Gene Therapy Gene In-Vivo Genetics 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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XTalks

AUGUST 19, 2022

XTALKS WEBINAR: How Agile Clinical Trials Unlock Universal Access to Rare Disease Research. Register for this free webinar to learn why the agile clinical trial model is a great fit for many rare disease categories, how to navigate the regulatory landscape for agile clinical trials in rare diseases, how to design studies and more.

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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 27, 2020

In these unprecedented times, we are especially grateful for the dedication of our research site staff and trial participants, whose commitment to EXCELLENCE facilitated the advancement of our lead program,” said George Yeh, President of TLC. SOUTH SAN FRANCISCO, Calif. and TAIPEI, Taiwan, Dec. About TLC599.

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pharmaphorum

JULY 22, 2021

We have completed phase 2 trial of a multi-antibody drug for influenza, CT-P27, and since 2015 we have been developing a therapeutic antibody to treat the Middle East Respiratory Syndrome (MERS), CT-P38. In just over a year, Celltrion Healthcare says it has identified and delivered a potential treatment for COVID-19. Overcoming obstacles.

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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

The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. “We

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Clinical Trials Clinical Trials Gene Therapy Trials 52

Drug Discovery World

NOVEMBER 23, 2023

Coupled with increasing awareness around ethical considerations and the increasing cost of animal studies, there is growing interest in New Approach Methodologies (NAMs), such as OOC, to provide effective, predictive preclinical models.

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Drug Discovery World

JUNE 14, 2023

In subsequent articles, themes cover future regulatory changes and redefining manufacturing. BioSenic is heavily involved in fundamental mechanistic studies, creating and formulating new ATMPs and conducting clinical trials to demonstrate the benefits for patients. DDW’s Megan Thomas heard from attendees about key trends in the sector.

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Drug Discovery World

OCTOBER 13, 2022

However, this discovery is being accomplished with help from contract development and manufacturing organisations (CDMOs) – compelled by the vaccine’s developers to expand capability to support vaccine development during pandemics. . The value of that versatility is reflected by the number of mRNA-based therapeutics in clinical trials.

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The Pharma Data

JANUARY 12, 2021

INCHEON, South Korea–( BUSINESS WIRE )– Celltrion Group today announced top-line results from its randomised, double-blind, and placebo controlled global Phase II/III clinical trial of CT-P59, an anti-COVID-19 monoclonal antibody treatment candidate. days quicker compared to placebo. days; 95% C.I, days compared to placebo [5.7

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Drug Discovery World

FEBRUARY 26, 2024

Reece Armstrong speaks to Kathryn Golden , SVP Technical Operations and Manufacturing, bit.bio, about novel technologies, and methods to manufacture human cells for therapies so more patients can access these treatments. One of these is cost, driven largely by the bespoke and expensive manufacturing processes.

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Manufacturing In-Vivo Development Production 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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Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

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Drug Discovery World

SEPTEMBER 6, 2022

And whilst clinical trials activity decreased by around 15% compared to 2020, last year still saw a number of regenerative medicines reach the bedside. Last year saw the sector surge, with levels of investment hitting an all-time high at $22.7 billion, compared to $19.9 billion, compared to $19.9 million price point per dose.

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Gene Therapy Gene Manufacturing Medicine 52

The Pharma Data

JANUARY 10, 2021

Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial.

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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.

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Drug Discovery World

JULY 22, 2022

For example, in randomised clinical trials of patients with non-small cell lung cancer (NSCLC), pembrolizumab has been shown to be effective in slowing down disease progression and improving survival rates 1. However, adverse effects such as cytokine release syndrome (CRS) and neurotoxicity still occur. Jespersen et al.

In-Vivo 52

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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.

In-Vivo 52

In-Vivo In-Vitro Antibody Development 52

The Pharma Data

DECEMBER 23, 2020

Additionally, Avacta announced it submitted a Clinical Trial Application in the UK for a Phase 1, first-in-human, open label, dose-escalation and expansion study of its lead pre|CISION prodrug, AVA6000, in patients with locally advanced or metastatic selected solid tumors. Elsewhere around the world: Avacta Group – U.K.

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Drug Discovery World

NOVEMBER 30, 2023

DDW’s Megan Thomas attended PEGS Europe 2023 in Lisbon, Portugal, from Tuesday 14 November to Thursday 16 November 2023. The company’s goal is to accelerate functional characterisation and shortenoptimisation time in antibody discovery and t-cell workflows. GSK has developed high-throughput mammalian and E.

Antibody 59

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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. This blog post will discuss how PK modeling can contribute to sample size estimation, a key aspect of a clinical trial protocol. Approaches to Pharmacokinetic Analysis.

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Drug Discovery World

FEBRUARY 2, 2023

A late-stage clinical trial in head and neck cancer for the treatment of severe oral blistering or oral mucositis caused by radiation therapy is planned. Bryan Oronsky , Chief Development Officer at EpicentRx, discucsses how one drug sourced from the defense and aerospace industry, could have many potential disese targets.

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The Pharma Data

OCTOBER 14, 2020

The AOP Orphan Group manufactures 80% of its drugs in Europe and has more than 20 subsidiaries and representative offices across the whole of Europe as well as in the United Arab Emirates and Israel. All jobs have been maintained and the workforce has grown from about 220 to 350. 1. -- -->. -- [if lte IE 8]--> .

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Clinical Trials Clinical Trials Manufacturing 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 59

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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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Delveinsight

FEBRUARY 11, 2021

However, they have only shrunk tumors in about half of patients who took them, along with chemotherapy, in the clinical trials. The first Fabry disease patient treated with Avrobio’s Plato gene therapy platform in a phase 2 trial has undergone the complete clearance of toxic kidney substrate.

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

The Pharma Data

DECEMBER 27, 2020

PARIS–( BUSINESS WIRE )– Regulatory News: Lysogene (FR0013233475 – LYS) (Paris:LYS), a phase 3 gene therapy platform company targeting central nervous system (CNS) diseases, today reports positive biomarker data from the ongoing AAVance clinical trial with LYS-SAF302 for the treatment of MPS IIIA (NCT03612869). About Lysogene.

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Gene Therapy Clinical Trials Clinical Trials In-Vivo 52

The Pharma Data

JULY 8, 2021

8) There were no clinically meaningful differences in safety results between the tezepelumab and placebo groups in the NAVIGATOR trial.(8) 8) There were no clinically meaningful differences in safety results between the tezepelumab and placebo groups in the NAVIGATOR trial.(8) Amgen (NASDAQ:AMGN) announced that the U.S.

Allergies 52

Allergies Trials In-Vivo Medicine 52

Drug Discovery World

AUGUST 4, 2022

Lack of access to this information can mask potential signals that could predict later issues with formulation, scale-up, manufacturing, transferability or even safety and efficacy. A need for speed and quality. Even for regular mAbs there are challenges. The risk of late-stage failure increases if this information is not obtained early on.

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