FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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Conversations in Drug Development Trends

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Below, we discuss some of these challenges in cell therapy trials.

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Science Daily: Pharmacology News

OCTOBER 16, 2024

Scientists have discovered a new process in our immune systems that leads to the production of an important family of anti-viral proteins called interferons.

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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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The Premier Consulting Blog

NOVEMBER 6, 2024

Inhaled combination products (ICP) have emerged as a significant advancement in the treatment of respiratory diseases such as asthma, chronic obstructive pulmonary disease (COPD), and other pulmonary conditions. However, these products present unique challenges from a Chemistry, Manufacturing, and Controls (CMC) standpoint.

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Codon

FEBRUARY 13, 2024

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.

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Broad Institute

JANUARY 14, 2025

His lab had just developed base editing, a gene-editing approach that makes single-letter changes in DNA and can shut down protein production using strategies including installing a stop signal in the genetic code. Theres still a long way to go to make this a therapy, Minikel said. But its really exciting to see how much is possible.

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Broad Institute

MAY 16, 2024

Gene therapy could potentially treat a range of severe genetic brain disorders, which currently have no cures and few treatment options. Since we came to the Broad we’ve been focused on the mission of enabling gene therapies for the central nervous system,” said Deverman, senior author on the study. “If

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BioPharma Drive: Drug Pricing

AUGUST 11, 2023

Filed in Maryland district court, the lawsuit claims the biotech unjustly profited from using HeLa cells to develop AAV vectors for its gene therapy products.

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Drug Target Review

NOVEMBER 8, 2023

Problem w/ CTs and foundational understanding of Vittoria: can you explore the current limitations of cell therapies and the challenges faced by patients and providers? Currently, only a small percentage of cancers can be effectively treated with cell therapies, and there is little diversity in the currently approved products.

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Drug Patent Watch

DECEMBER 17, 2024

Contract Development and Manufacturing Organizations (CDMOs) are at the forefront of this shift, playing a crucial role in bringing innovative therapies to market. These modular setups allow for customizable production lines, opening doors to unlimited opportunities in drug development[2]. Here are some key strategies to consider.

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Drug Target Review

AUGUST 28, 2024

Once a patient develops advanced cirrhosis/end-stage liver disease there are no specific therapies to significantly avoid major decompensations and death in the next few years. Could you describe the platform of macrophage biology and cell engineering used by Resolution Therapeutics in developing their cell therapies?

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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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PPD

OCTOBER 17, 2024

Cell and gene therapy (CGT) studies are rapidly gaining momentum in the Asia-Pacific region, fueled by growing patient demand and a thriving ecosystem of innovation. In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches.

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Drug Target Review

JANUARY 4, 2024

What distinguishes Alder Therapeutics’ approach to regenerative cell therapy development from traditional methods, and how does it aim to reduce risks in the preclinical phase? Traditional approaches to regenerative cell therapy development are defined by several challenges. For manufacturing, it’s no different.

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LifeSciVC

APRIL 24, 2024

Additionally, for illustrative reasons this is geared towards a single target / product focus vs. broader platform diligence, though many of these mental models will apply for selecting targets and indications for a platform. with gene editing or gene therapy, enzyme replacement therapy), agonism (e.g., in liver, in CNS)?

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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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SugarCone Biotech

MARCH 2, 2025

Less clear is whether we can productively and safely inhibit TGF- activity at all given the toxicity issues associated with TGF- inhibition. Where TGF- is present there is no or limited IFN- secretion by T cells and that means no PD-L1 expression within the tumor microenvironment, aka the TME.

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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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Drug Target Review

FEBRUARY 9, 2024

What key findings about stem cell behaviour, differentiation and integration within host tissues impact the development of stem cell therapies? Despite their potential, ADSC therapy faces several challenges in preclinical studies. Is there a certain disease or condition that you believe stem cell therapy holds the most promise for?

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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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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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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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Drug Patent Watch

DECEMBER 9, 2024

High-Throughput Screening: Modern Technology Meets Natural Products Advanced technologies now allow researchers to rapidly test thousands of natural compounds against specific disease targets. The development of Taxol involved multiple patents, including those for the isolation method, synthetic production, and various formulations.

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BioPharma Drive: Drug Pricing

JULY 26, 2023

The startup is the latest young company to emerge with plans to make production of cell-based medicines easier for researchers and biotechs.

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Drug Channels

OCTOBER 24, 2024

Shabbir explains the barriers that providers face when dealing with branded portals for multiple products. He then maintains that patients can access new therapies more quickly when the manufacturer relies on a brand-agnostic hub connected to a large network of providers and integrated with the systems those providers use daily.

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PPD

JANUARY 15, 2025

As a result, drug developers make better decisions more quickly (removing 50% of study timeline whitespace) to bring new therapies to market faster. Accelerate customer speed to market With a modern and integrated user experience, AI solutions put the right data and insights into the right hands in real time.

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FDA Law Blog: Drug Discovery

MARCH 13, 2023

To further expand patient input into product development, the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products or “OTP” (formerly the Office of Tissues and Advanced Therapies, or “OTAT”) is holding a free public workshop titled, Clinical Trials: The Patient Experience.

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Drug Channels

APRIL 8, 2022

To learn more about CoAssist and AssistRx specialty therapy initiation and patient support services, click here to schedule a meeting with AssistRx at the Asembia Specialty Pharmacy Summit on May 2-5, 2022. He goes on to describe four new categories of patient solutions and pharmacy models specific to this growing market segment.

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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 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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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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Alta Sciences

JANUARY 17, 2025

WebinarPreclinical Studies of Gene Therapy Products: Latest Trends lperez Fri, 01/17/2025 - 22:48 Image Website_webinar_Preclinical Studies Gene Therapy_v2.jpg jpg Tags Preclinical Research URL [link] Event End Date Thu, 03/06/2025 - 12:00 Event Start Date Thu, 03/06/2025 - 12:00 Weight 1

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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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DrugBank

JANUARY 29, 2025

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. In sickle cell disease, for instance, a missense mutation in the HBB gene , encoding the beta-globin subunit of hemoglobin, leads to the production of abnormal hemoglobin S.

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PPD

MARCH 11, 2025

It involves addressing the specificity of research questions, the clinical and investigational product context, and underlying causal frameworks; selection of a suitable data source; and a deep understanding of RWD provenance, granularity, completeness, structure and curation.

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Alta Sciences

NOVEMBER 21, 2024

Intra-Cerebroventricular (ICV) Route in Mice for Administration of Gene Therapy Products lperez Thu, 11/21/2024 - 14:35 Publication Poster_Altasciences_ACT_2024—ICV_Route_in_Mice_for_Administration_of_Gene_Therapy_Products.pdf Tags Preclinical Research Category Poster Weight 1

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