Drugs.com

JUNE 25, 2024

TUESDAY, June 25, 2024 -- An experimental stem cell therapy can essentially cure type 1 diabetes by restoring insulin production in some patients, early clinical trial results show.Seven out of 12 patients no longer needed daily insulin shots after.

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Drug Target Review

SEPTEMBER 1, 2023

Could you explain the fundamental distinction between autologous and allogeneic cell therapies and how they differ in terms of cell sourcing and application? Autologous immune cell therapy involves the grafting of a patient’s own immune cells. Then, cells are infused back to that same patient.” There are pros and cons to each.

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Drug Target Review

OCTOBER 2, 2024

Cell and gene therapies (CGTs) have made significant advancements in treating oncological diseases, with therapies like CAR-T cell treatments transforming cancer care. However, cell-based therapies are intended to leverage those healthy cells via transplant to regenerate damaged tissue.

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Alta Sciences

JULY 18, 2023

Webinar—Nonclinical Safety Assessment for Gene Therapy Products—Key Considerations lperez Tue, 07/18/2023 - 18:35 Image Website_Webinar_AAV-SoMe_v2.jpg jpg Tags Preclinical Research URL [link] Event End Date Thu, 10/19/2023 - 12:00 Event Start Date Thu, 10/19/2023 - 12:00 Weight 1

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Fierce BioTech

JULY 16, 2024

Launching a Product in Europe: Understanding Supply Chain Cost Drivers This paper explores the costs of setting up a supply chain in Europe for a new biopharma therapy. lbarrick Tue, 07/16/2024 - 14:15 This paper explores the costs of setting up a supply chain in Europe for a new biopharma therapy.

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Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

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BioPharma Drive: Drug Pricing

SEPTEMBER 4, 2024

A planned facility in California will boost Novartis’ West Coast supply chain as new uses for the company’s radioligand therapies grow.

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Fierce BioTech

SEPTEMBER 30, 2024

Gene therapy manufacturing: Custom and GMP-grade reagents to accelerate your scale-up plan Download now to learn how gene therapy manufacturers can overcome the unique challenges of scaling production.

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Drug Channels

OCTOBER 4, 2024

Timothy discusses the affordability and patient journey challenges of specialty-lite products for patients, manufacturers, and health care providers. He explains how AssistRx's Advanced Access Anywhere (AAA) solution streamlines processes for specialty-lite products and facilitates enrollment via a digital hub.

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Agency IQ

APRIL 12, 2024

BY RACHEL COE, MSC Late last year, the FDA published a draft update to its 2011 guidance on potency assays for cell and gene therapy products, unveiling a major shift in approach to the issue. Now, the deadline for feedback to that draft guidance has passed, and AgencyIQ has an analysis of the comments here.

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BioPharma Drive: Drug Pricing

AUGUST 24, 2023

Bristol Myers Squibb is among those backing the startup, which claims the manufacturing capacity at its New Jersey plant can surpass that of conventional CDMO facilities.

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Fierce BioTech

APRIL 9, 2024

Commercialization Strategies for Cell and Gene Therapies pesurya Tue, 04/09/2024 - 12:46 Tue, 05/07/2024 - 11:00 Resource Type Webinar Mike Sweeney Joel Wayment Fran Gregory Akshay Peer (Ph.D) Click here to login. Listing Image Combined-Logo-Listing-5072024.png

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The Pharma Data

APRIL 27, 2023

Bristol Myers Squibb Strengthens Cell Therapy Capabilities by Adding New U.S. Manufacturing Facility for Viral Vector Production Bristol Myers Squibb (NYSE: BMY) today announced expansion of its global cell therapy manufacturing network to enable in-house viral vector production through a U.S.-based

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Drug Target Review

AUGUST 31, 2023

Stem cell therapies have already demonstrated their prowess in treating diverse cancers and ailments linked to the blood and immune system. Chronic inflammation involving microglia has been linked to the disease, as the release of inflammatory molecules triggers an increase in β-amyloid production.

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The Premier Consulting Blog

APRIL 18, 2024

Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. This blog will outline three characteristics of a successful nonclinical program to support entry into clinical trials for a cell or gene therapy product.

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The Premier Consulting Blog

APRIL 18, 2024

Designing an adequate nonclinical program to support the safety of cell or gene therapy products is not always straightforward. The cell or gene therapy should be pharmacologically active in the animal model. Biodistribution of the product (transgene/gene or cells) should be evaluated. How long does the study need to be?

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Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. Hearing aids also have all sorts of deficits.

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Drug Target Review

MAY 22, 2024

How does Circio’s circVec technology aim to enhance the potency and reduce the cost of current gold-standard gene therapy? There are three major reasons for why Circio has selected AATD as its lead gene therapy program. Firstly, there have been previous AAV gene therapy clinical studies in this indication that have shown promise.

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DrugBank

JUNE 13, 2024

  Gene Therapy: Reprogramming the Body's Cellular Code    Gene therapy is an exciting field that treats diseases at their genetic roots.    A key part of gene therapy is efficiently delivering the therapeutic genetic material directly into target cells.

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thought leadership

MAY 10, 2024

In January 2024, the FDA Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products issued a guidance document- Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products.

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Fierce BioTech

MAY 2, 2024

In this webinar, you will learn about: The challenges and risks related to the packaging and filling of high-value drug products. West Pharmaceutical Services (West) and Cytiva have teamed up to offer innovative and versatile fill finish solutions to efficiently meet the unique demands of advanced pharmaceutical applications.

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FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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FDA Law Blog: Drug Discovery

NOVEMBER 2, 2023

Lewis, Senior Regulatory Device & Biologics Expert — On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” A considerable roadblock in the development of RMT products is a lack of regulatory predictability.

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Fierce BioTech

SEPTEMBER 11, 2023

Aligning cross-functional teams across the organization to ensure medical, marketing, and market access are all in lock-step is even more crucial when launching a therapy into a rare disease market. Click here to login.

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thought leadership

JULY 2, 2024

The CTR is applicable to clinical trials with Gene Therapy Medicinal Products (GTMPs) as well, but unfortunately it does not cover the environmental requirements that are applicable to GTMPs if they contain or consist of genetically modified organisms (GMOs).

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Agency IQ

NOVEMBER 3, 2023

Probiotics, a type of microbiome-based therapy containing live micro-organisms, are broadly available on drug store and grocery store shelves, but the regulatory classifications for products containing the same micro-organism can differ dramatically.

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Advarra

JANUARY 11, 2024

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.

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Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

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The Pharma Data

OCTOBER 26, 2020

The agreement covers the gene therapy program targeting OTOF (OTOF-GT), the gene encoding the otoferlin protein. The agreement covers the gene therapy program targeting OTOF (OTOF-GT), the gene encoding the otoferlin protein. Mutations in OTOF lead to severe or profound congenital hearing loss. About Sensorion.

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PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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Fierce BioTech

OCTOBER 6, 2023

Bringing a New Heart Failure Therapy to the Clinic swheeler Fri, 10/06/2023 - 14:56 Tue, 11/28/2023 - 11:00 Resource Type Webinar Phil Morton, MD Dr. Takehiko Kaneko, MD Duration 60 minutes Commercializing biopharmaceuticals and advanced therapies requires robust, standardized manufacturing processes.

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Fierce BioTech

FEBRUARY 7, 2024

Accelerate Your Cell Therapy to Clinic with a Scalable CGMP-Ready Process swheeler Wed, 02/07/2024 - 16:13 Thu, 03/28/2024 - 11:00 Resource Type Webinar Duration 60 minutes Accelerate your journey with viral vector-basted cell therapies and the enhancements you can incorporate. Click here to login.

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Drug Channels

OCTOBER 28, 2022

Today’s guest post comes from Brooks Wildasin, Vice President of Product Management at CareMetx. Brooks introduces us to CareMetx’s specialty therapy warranty, a specialty patient outcome-based contract model.

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ASPET

OCTOBER 12, 2023

The CET RAIDR program minimizes development and procurement costs by supplementing the military medical providers' toolbox with post-Phase II therapies that demonstrate established safety and manufacturing processes, leading to a cost-sparing model for niche medicines (i.e., CBRN MCMs).

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Drug Discovery Today

DECEMBER 5, 2020

OVO Biomanufacturing, a spin-out from the University of Warwick and Coventry University, is developing digital solutions to improve the efficiency of viral vaccine and gene therapy manufacture. The technology can be applied to any virus that is grown to produce a vaccine or therapy.

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Fierce BioTech

AUGUST 14, 2023

This webinar will focus on the latest advancements in bioprocessing for cell and gene therapies (CGTs), including the improved consistency and reduced production costs associated with closed manufacturing. Listing Image Cytiva 250x190.png

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