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.

Clinical Development 69

Clinical Development Production Clinical Trials Treatment 69

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.

Therapies 96

Therapies Clinical Trials Clinical Research Trials 96

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.

Therapies 98

Therapies Small Molecule Production Disease 98

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.

Disease 113

Disease Treatment Small Molecule Therapies 113

Drug Target Review

AUGUST 28, 2024

What are the main challenges currently faced in the treatment of chronic liver diseases, and how does Resolution Therapeutics aim to address these challenges? 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.

Therapies 116

Therapies Disease Engineering Medical Schools 116

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?

Therapies 64

Therapies Treatment Vaccine FDA Approval 64

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.

Therapies 105

Therapies Engineering Production Clinical Trials 105

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.

Therapies 98

Therapies Disease RNA Engineering 98

Broad Institute

JANUARY 14, 2025

The treatment, which uses base editing to make a single-letter change in DNA, reduced levels of the disease-causing prion protein in the brain by as much as 60 percent. A base-editing approach could also likely be a one-time treatment for all prion disease patients regardless of the genetic mutation causing their disease.

Disease 141

Disease DNA Protein Production Treatment 141

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.

Treatment 105

Treatment Immune Response Therapies Clinical Trials 105

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.

Therapies 105

Therapies Disease Research Treatment 105

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.

Therapies 118

Therapies FDA Treatment Production 118

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?

Biochemical Assays 98

Biochemical Assays Treatment Bioinformatics Disease 98

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.

Therapies 59

Therapies Treatment Engineering Molecular Biology 59

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.

Treatment 105

Treatment Organic Chemistry Research Disease 105

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?

Therapies 98

Therapies Trials FDA Clinical Trials 98

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.

Therapies 119

Therapies Pharmaceutical Companies Disease Treatment 119

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.

Therapies 72

Therapies Clinical Supply Clinical Trials Trials 72

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?

Therapies 84

Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

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.

Therapies 74

Therapies Drugs DNA Treatment 74

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.

RNA 134

RNA Vaccine Small Molecule Therapies 134

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.

Treatment 75

Treatment FDA Clinical Trials Therapies 75

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.

Therapies 100

Therapies Licensing Licensing FDA Approval 100

Drug Target Review

MARCH 21, 2024

Bobcat mRNA TM is a proprietary linear mRNA technology designed for the highly efficient and cost-effective production of high-quality mRNA encoding large proteins (over 13 kb). There are a few approved therapies for DMD including four exon-skipping drugs and one AAV-microdystrophin drug, which uses a shortened version of dystrophin.

Therapies 64

Therapies RNA Immune Response Treatment 64

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options.

Small Molecule 145

Small Molecule Drugs Clinical Pharmacology Trials 145

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 (..)

Therapies 80

Therapies Research Hospitals Treatment 80

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.

Production 52

Production FDA Compliance Disease 52

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

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.

Therapies 69

Therapies Immune Response DNA Virus 69

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.

Therapies 52

Therapies Production Treatment 52

Drug Target Review

SEPTEMBER 6, 2024

What trends are driving the increased use of in vivo delivery methods in gene therapy? This growth has been influenced by the increased use of lentiviral vectors for more prevalent diseases, earlier treatment lines and broader applications. How are new therapeutic areas emerging as potential targets for in vivo gene therapy?

Therapies 59

Therapies Vaccine Marketing Disease 59

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.

DNA 100

DNA Therapies Treatment Production 100

Drug Target Review

JANUARY 12, 2024

How does the integration of Real-World Data (RWD) with genomic biomarker data contribute to a more comprehensive understanding of disease progression and treatment response? This one size fits all approach to drug prescribing still pervades in mostly all therapy areas except oncology. Most drugs do not work in all people.

Doctors 111

Doctors Clinical Trials Treatment Therapies 111

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.

Therapies 59

Therapies RNA Protein Expression DNA 59

FDA Law Blog: Drug Discovery

MARCH 26, 2024

Sasinowski — On March 21st, FDA announced the approval of the first nonsteroidal therapy for the treatment of Duchenne Muscular Dystrophy (DMD) (FDA press release available here ). By Charles G. Raver & James E. Valentine & Frank J. Duvyzat (givinostat), a histone deacetylase (HDAC) inhibitor developed by Italfarmaco, S.p.A.,

FDA Approval 59

FDA Approval Therapies FDA Trials 59

ASPET

MARCH 7, 2024

Aldehyde dehydrogenase 2 (ALDH2) is an important player in the redox reaction of ethanol with endogenous aldehyde products released by lipid peroxidation. Therefore, clarifying the relationship between ALDH2 and HCC is helpful for formulating rational treatment strategies.

Research 100

Research Regulations Treatment Therapies 100

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.

Therapies 59

Therapies FDA Production Regulations 59