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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Chemical Biology and Drug Design

FEBRUARY 2, 2024

Their use in combination therapy decreases the concentration of the reference drug used. Abstract Existing chemotherapy for neglected tropical diseases (NTDs) can often be toxic and ineffective, highlighting the necessity for new treatments. Terpenes are secondary metabolites with pharmacological potential.

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Drug Target Review

AUGUST 22, 2023

This Q&A explores how pre-clinical research is being used to identify potential therapies for Huntington’s disease, a devastating condition that currently lacks disease- modifying treatments. What makes the potential therapeutic for Huntington’s disease unique compared to other treatments?

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Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? Part of the challenge is that patients present with very different kinds of clinical phenotypes, meaning the populations are heterogeneous. All stakeholders have benefited from these innovations.

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PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. A Scant Protein with Outsize Importance Slowing the muscle decline of DMD presents two huge challenges: the ubiquity of muscle and a giant gene. The gene presents a complex landscape. million DNA bases.

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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.

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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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ASPET

OCTOBER 12, 2023

Supramolecular nanostructured based delivery systems are emerging as a meaningful approach in treatment of cancer, offering controlled drug release and improved therapeutic efficacy. Different SNs approaches and recent literature reviews on peptide delivery are also presented to the readers.

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Drug Target Review

NOVEMBER 29, 2023

Our primary focus is to design and develop RNA therapies for liver diseases. Our team of chemists then uses this information to develop novel therapies. By doing this, we get a superior understanding of which therapies will and will not work in human biology before a clinical trial. As a result, waiting lists continue to grow.

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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. She expressed enthusiasm for the prospect of leveraging hematopoietic stem cell therapy to devise an innovative therapeutic approach for this devastating disorder.

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Drug Target Review

FEBRUARY 28, 2024

Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Preclinical research on γδ T cells has made great strides since the cells were first identified in the 1980s, with γδ T-cell therapies from several companies, including IN8bio, now in or nearing clinical trials for various cancers.

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Chemical Biology and Drug Design

DECEMBER 15, 2023

Due to the complex pathological processes underlying neurodegeneration, at present, there is no viable therapy available for neurodegenerative disorders. Abstract Neurodegenerative disorders, which affect millions worldwide, are marked by a steady decline of neurons that are selectively susceptible.

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Drug Target Review

DECEMBER 13, 2024

These types differ in their etiology, natural history, and present distinct challenges in disease management. 2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates.

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PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

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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

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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Chemical Biology and Drug Design

NOVEMBER 23, 2023

Nowadays, gene therapy is being implemented for its treatment as several of these genetic defects have been identified. The present article describes the basics regarding the disease, its causes and factors responsible, and the gene therapy solutions available to treat this disease.

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Drug Target Review

OCTOBER 25, 2023

Their findings may result in new treatments for reproductive conditions. He stated that this could inform new treatments for reproductive conditions like recurrent pregnancy loss and preeclampsia. Preeclampsia may present at any gestation but is more commonly encountered in the third trimester.

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Drug Target Review

JULY 3, 2023

There is a significant unmet need for effective treatments to alleviate suffering and prevent premature death across the spectrum of genetic diseases. While existing treatments can help manage symptoms, reduce complications, and slow disease progression in certain cases, they do not offer lasting cures for most genetic disorders.

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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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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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ASPET

NOVEMBER 17, 2023

Ketamine is a new, potent and rapid-acting antidepressant approved for therapy of treatment-resistant depression, which has a different mechanism of action than currently-available antidepressant therapies. It owes its uniquely potent antidepressant properties to a complex mechanism of action, which currently remains unclear.

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ASPET

JANUARY 23, 2024

The current pharmacological treatments for SCD-associated priapism are limited and focused on acute interventions rather than prevention. Thus, there is an urgent need for new drug targets and preventive pharmacological therapies for this condition.

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Chemical Biology and Drug Design

APRIL 26, 2023

Despite the severity, no causal treatment is currently available. The administration of synthetic polymers that present autoantigens in a multivalent manner have been established as promising therapeutic strategies in other autoimmune diseases and may be applied to IgAN.

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Drug Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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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. The Natural History Studies Grants Program funds natural history studies that collect gather data on how rare diseases progress over time without treatment.

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Drug Target Review

JANUARY 15, 2024

Monoclonal antibodies (mAbs) targeting tumour-specific antigens play an essential role in the treatment of many cancers. An important limitation of antibodies against tumour antigens is that these agents direct responses to molecular targets present on the surface of the cell.

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Fierce BioTech

JULY 17, 2024

The cell and gene therapy industry is experiencing rapid growth and evolution, presenting both exciting opportunities and unique challenges for researchers and logistics providers alike. | How is the cold chain adapting to the cell and gene therapy boom?

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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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Chemical Biology and Drug Design

MAY 11, 2023

Abstract Parkinson's disease is among the most common forms of neurodegenerative illness, with present treatment being primarily symptomatic and frequently coming with substantial adverse effects. Crocin, a naturally occurring carotenoid molecule, was found to have neuroprotective potential in the therapy of this disorder.

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SCIENMAG: Medicine & Health

JUNE 18, 2023

Receiving rapid access to testosterone therapy reduced feelings of gender dysphoria and led to a clinically significant reduction in depression as well as a 50% reduction in suicidality among transgender and gender diverse adults, according to research being presented Sunday at ENDO 2023, the Endocrine Society’s annual meeting in Chicago, Ill.

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Chemical Biology and Drug Design

FEBRUARY 23, 2024

In the present work, we report that isoliquiritigenin, a bioactive component of licorice roots, attenuates neuroinflammation of a mice model with subarachnoid hemorrhage through inhibition of NF-κB-mediated NLRP3 inflammasome activation. Isoliquiritigenin (ISL) is a bioactive component extracted from Genus Glycyrrhiza.

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Drug Target Review

JANUARY 2, 2024

Through a series of experiments, they successfully developed a personalised stem cell therapy using a data-driven, single-cell technique based on swift subcellular proteomic imaging. Our recent work proposes the use of an intracellular toolkit to map organelle bio-geography in stem cells that could lead to more precise therapies.”

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Drug Target Review

MARCH 1, 2024

How significant are the results in terms of advancing TCR-T cell therapy and AMP immunotherapy for the treatment of solid tumours? Long-term protection against tumour recurrence in AMP-treated mice was associated with antigen spreading to additional tumour-associated antigens not targeted by the treatment.

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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? 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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The Pharma Data

MAY 27, 2022

In the pivotal clinical study CARTITUDE-1, 98 percent of patients with relapsed or refractory multiple myeloma responded to a one-time treatment with ciltacabtagene autoleucel and 80 percent of patients who responded experienced a stringent complete response . In December 2017, Janssen Biotech, Inc. to develop and commercialise cilta-cel.[1].

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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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