Drug Target Review

JANUARY 15, 2024

Could you provide insights into the driving factors behind exploring novel treatments for cancer types? Could you provide further details on how this phenomenon could impact broader treatment outcomes? Immunotherapy has become a cornerstone of cancer therapy and significantly improved patient outcomes.

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Treatment Immune Response Therapies Clinical Trials 106

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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Therapies Engineering Production Clinical Trials 106

Drug Target Review

OCTOBER 11, 2023

Most of these conditions are genetic in origin and the majority have no effective treatment. 1,2 Many, however, do have dysfunctional proteins at their core, and for these diseases, a new option is being explored: autophagy-boosting therapies. Gene therapy, of course, also holds great promise.

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Disease Treatment Small Molecule Therapies 113

DrugBank

JUNE 13, 2024

These innovative technologies are changing how medications reach their targets within the body, leading to more effective treatments with fewer side effects.   Take cancer treatment, for example. Implantable insulin pumps equipped with continuous glucose monitoring (CGM) sensors can update old diabetes treatment options.

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Therapies Drugs DNA Treatment 74

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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Therapies FDA FDA Approval Treatment 119

Olympian Clinical Research

MARCH 6, 2024

Managing rosacea can be challenging, but with the right treatments, many people see significant improvements. Here’s a closer look at the top treatments that can help control this condition and why professional guidance is key. Products should be non-irritating, fragrance-free, and designed for sensitive skin.

Treatment 52

Treatment Therapies Production 52

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) Join CGT industry experts for an insightful discussion on real-world commercialization challenges and mitigation strategies. Click here to login.

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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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Therapies Disease Research Treatment 105

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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Vaccine Therapies Clinical Trials Small Molecule 52

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. Could you give us an overview of the Lineage platform?

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Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

Conversations in Drug Development Trends

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

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Treatment FDA Clinical Trials Therapies 75

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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Therapies Production Engineering Treatment 40

BioPharma Drive: Drug Pricing

OCTOBER 26, 2023

Executives at the biotech say they’re trying to get ahead of the payer and production challenges that will face their gene editing treatment exa-cel, which is now under FDA review.

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Therapies FDA Treatment Production 123

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? As a first step, Circio is working on designing and validating circVec-AAV vectors for the treatment of genetic diseases, and testing these head-to-head against the mRNA-AAV equivalent in vivo.

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Drug Target Review

JULY 12, 2023

Molecular-level biochemical assays like transcriptomics, genomics and proteomics have emerged as valuable tools for identifying potential targets in cancer treatment through deep cyclic inhibition (DCI). How does the DCI mechanism compare to the design of other drugs for cancer treatment?

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Biochemical Assays Treatment Bioinformatics Disease 98

Conversations in Drug Development Trends

JULY 9, 2024

The recent surge in GLP-1 treatments has ignited a profound shift in the pharmaceutical landscape, with more studies being announced than ever before. However, the treatment journey does not end with weight reduction, as maintaining weight loss overall is equally crucial.

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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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Therapies FDA Production Regulations 59

ASPET

OCTOBER 12, 2023

The focus of the CET RAIDR effort is to bridge treatment gaps between threat identification and the implementation of licensed targeted MCMs, thereby strengthening warfighter resiliency. Responsible and relevant care must be more responsive to needs of expansive and novel threats, as showcased by lessons learned from the COVID-19 pandemic.

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Therapies Licensing Licensing FDA Approval 100

Chemical Biology and Drug Design

SEPTEMBER 16, 2024

Baicalein stabilized MAPK9 to induce apoptosis in hepatocarcinoma cells, which would be helpful to understand and use baicalein in hepatocarcinoma therapy. However, there are limited effective treatments available for it. The use of natural products in the management and treatment of HCC is gaining more attention.

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DNA Therapies Treatment Production 100

Drug Target Review

AUGUST 6, 2024

Autoimmune diseases : Rheumatoid Arthritis : ADCs targeting specific immune cells or inflammatory mediators can provide more precise treatment options with potentially fewer side effects. Obesity : By targeting adipose tissue or specific metabolic pathways, ADCs could offer new treatments for obesity and related metabolic disorders.

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Therapies Pharmaceutical Companies Disease Treatment 118

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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Therapies Immune Response DNA Virus 69

Broad Institute

SEPTEMBER 23, 2024

New approach may yield modified messenger RNAs for treating a wide range of conditions By Ari Navetta September 23, 2024 Breadcrumb Home New approach may yield modified messenger RNAs for treating a wide range of conditions The LEGO framework could help scientists create mRNA therapies that last longer and require smaller doses.

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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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FDA Law Blog: Drug Discovery

MARCH 13, 2023

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

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Drug Target Review

JUNE 8, 2023

An important research area at AbbVie involves blood cancers, where our ultimate goal is to transform standards of care across these diseases and advance a dynamic cancer research and treatment pipeline. 2 However, treatment options were still limited and often came with severe side effects.

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Treatment Organic Chemistry Research Disease 106

Vial

DECEMBER 11, 2023

In the decades since its identification in 1989, advances in clinical research for new Hepatitis C therapies and the introduction of direct-acting antiviral agents (DAAs) have led to viral eradication in more than 98% of patients, resulting in HCV crossing from manageable to being the first curable , chronic viral infection.

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Clinical Research Therapies Research Vaccine 52

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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Therapies Production Regulations Treatment 52

SCIENMAG: Medicine & Health

JULY 21, 2023

The preclinical study showed that injecting ovarian tissue-derived differentiated induced pluripotent stem cells can restore hormone production and fertility in mice with premature ovarian failure related to genetic diseases and cancer treatment A new study by investigators from Brigham and Women’s Hospital, a founding members of the Mass General Brigham (..)

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Therapies Research Hospitals Treatment 80

FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” Drug development for these conditions has unique and complex challenges, therefore few treatments are available to patients.”

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The Pharma Data

MARCH 31, 2022

Bayer announced today that it is strengthening the production network of its pharmaceutical division to ensure sustainable competitiveness and support the transformation of its pharmaceutical business based on breakthrough innovation delivering long-term, sustainable business growth.

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Production Pharmaceuticals Science Therapies 52

The Pharma Data

MAY 26, 2023

State-of-the-art cryogenic freezers alongside material transfer equipment have been installed that are designed to retain the integrity of investigational advanced therapy products by minimizing their time-out-of-environment. Catalent helps accelerate over 1,000 partner programs and launch over 150 new products every year.

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Clinical Supply Therapies Clinical Trials Packaging 52

The Pharma Data

MARCH 9, 2022

Investigators at Cedars-Sinai have identified a potential new therapy for COVID-19: a biologic substance created by reengineered human skin cells. Although still in the early stages, the findings open the possibility of having a new therapy for COVID-19 patients. ” ASTEX appears to have stopped this hijacking process.

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Treatment Virus Engineering Therapies 52

The Pharma Data

APRIL 19, 2022

Kite’s Global CAR T-Cell Therapy Manufacturing Network Increasing Capacity by 50% to Meet Patient Demand for New Cancer Therapies. — Scalable and Adaptable Facility Provides Flexibility for Current and Future Cell Therapy Innovation. The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer.

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FDA Approval Therapies FDA Hospitals 52

Agency IQ

FEBRUARY 12, 2024

BY RACHEL COE, MSC | FEB 7, 2024 10:21 PM CST The Bespoke Gene Therapy Consortium A collaborative effort, the Bespoke Gene Therapy Consortium (BGTC) was launched in October 2021 to “accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease.”

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DrugBank

MAY 6, 2024

Unlike conventional chemical-based medicines, biologics offer targeted therapies derived from living cells, which can result in more effective and personalized treatments. This surge in approvals clearly marks a new era in healthcare, emphasizing the rapid progress and heightened focus on biologics in medical treatment.

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The Pharma Data

JUNE 22, 2023

Kite Announces Completion of Marketing Authorization Transfer for Yescarta® CAR T-cell Therapy in Japan SANTA MONICA, Calif.–(BUSINESS has been approved by Japanese regulatory authorities to manufacture Yescarta ® for the Japanese market, and commenced its supply of the product earlier this year. to Gilead Sciences K.K.,

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Conversations in Drug Development Trends

JUNE 21, 2024

Worldwide Clinical Trials attends each year to hear from the research community, connect with our sponsors and sites, and explore potential partnerships to drive forward novel treatments. Previously, T-cell therapies had been primarily used on liquid tumors, as the T-cells were unable to penetrate the solid tumor environment.

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