Drug Target Review

DECEMBER 5, 2024

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.

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Drug Target Review

JANUARY 8, 2024

Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. a clinical-stage private biopharma company developing therapies for neurological and psychiatric diseases.

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Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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Drug Target Review

NOVEMBER 6, 2023

2 It is this complexity that necessitates powerful, targeted combination therapies. To bring us closer to curing cancer, a combination of effective drugs with non-overlapping mechanisms of action is required. To bring us closer to curing cancer, a combination of effective drugs with non-overlapping mechanisms of action is required.

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DrugBank

JULY 24, 2024

For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed. Similarly, PROTACs can target and degrade overexpressed proteins, offering a way to overcome drug resistance, a common issue in cancer treatment.

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DrugBank

MAY 15, 2024

Its structure makes it incredibly difficult for drugs to bind effectively, which has stymied drug development for decades. The binding pockets on KRAS are shallow and polar , not ideal for the kinds of interactions needed for strong, effective drug binding.

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Drug Target Review

DECEMBER 14, 2023

Throughout the course of our lives, natural and environmental factors can break DNA. Breaks in both DNA strands are a particularly dangerous threat to genome stability. At a DNA double-strand break (DSB), potentially lost sequence information cannot be recovered from the same DNA molecule.

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Conversations in Drug Development Trends

AUGUST 14, 2024

Historically, these indications have challenged the one-size-fits-all treatment approach due to patient variability, such as genetic differences in drug metabolism and underlying health conditions. Hematological cancer cells can also develop resistance to therapies over time, reducing treatment effectiveness.

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The Pharma Data

SEPTEMBER 14, 2020

Today these medicines, called targeted therapies, are available for treating many forms of cancer. Targeted therapies took center stage when Alice Shaw started her career as a physician-scientist caring for patients with lung cancer. Clinical trial investigators are the eyes and ears in the clinic for pharmaceutical drug developers.

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Conversations in Drug Development Trends

JUNE 21, 2024

Antibody-Drug Conjugates: Advancing Precision Therapy ADCs represent a compelling combination of targeted antibody therapy and potent chemotherapy, aiming to enhance treatment efficacy while minimizing systemic toxicity. Currently, there are 12 approved ADC drugs, and several hundred more are in the development pipeline.

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LifeSciVC

OCTOBER 13, 2022

This summer, approvals on both sides of the Atlantic – for beta thalassemia and cerebral adrenoleukodystrophy in the United States and for severe hemophilia A and aromatic L-amino acid carboxylase deficiency in Europe – show that regulators are likely open to green-lighting a range of emerging gene therapies.

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DrugBaron

JULY 19, 2022

Despite the current hype around so called “advanced therapies”, which range from gene editing to cell therapies, and the inexorable advance of biologic therapeutics such as monoclonal antibodies, even in 2022 the majority of drugs in development and reaching patients are still small organic molecules.

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The Pharma Data

DECEMBER 14, 2020

. The current standard of care for GBM consists of de-bulking surgery followed by combined treatments with fractionated ionizing radiation (IR) and the DNA alkylating agent temozolomide (TMZ). At the stage of GBM relapse and recurrence, no effective therapy strategies currently exist. Contact.

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The Pharma Data

SEPTEMBER 11, 2021

We have the ability to generate precise antibodies to a diverse range of targets, which together with Boehringer Ingelheim’s strength in drug development capabilities, could mean multiple new, more personalized treatments in the future for patients,” said Emily M. Leproust, Ph.D., CEO and co-founder of Twist. About Twist Biopharma.

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PLOS: DNA Science

APRIL 18, 2024

Understanding blood-brain barrier function has been at the heart of research into drug therapies for neurological diseases. That’s why we are excited about the discovery of this variant in fibronectin, which may be a good target for drug development,” said study co-leader Richard Mayeux.

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Conversations in Drug Development Trends

JULY 8, 2024

They are specifically formulated to emit radiation that can be detected by imaging equipment or deliver targeted radiation therapy to specific tissues or organs within the body, serving as a diagnostic and therapeutic tool for various medical conditions.

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Vial

DECEMBER 5, 2023

It has become a fundamental tool for researchers to explore the complexities of genetic information and conduct genetic-informed drug development. Sponsors have used NGS to screen patients for clinical trial eligibility and patient stratification , expanded CDx development, and comprehensive genomic profiling (CGP).

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Drug Target Review

SEPTEMBER 18, 2024

What specific cost-cutting measures can AI provide in the manufacturing process of cell and gene therapy, and how do these compare to traditional methods? The cell and gene therapy (CGT) space provides a great example of the potential of AI. AI can vastly improve cell culture, a critical step in cell therapy production.

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Drug Target Review

SEPTEMBER 10, 2024

The current landscape of protein drug development is characterised by accelerated timelines where new drugs are approved in months rather than years. Hence, in many cases an earlier IND may be prevented by the timely provision of representative Drug Substance (DS) to execute such toxicology studies.

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The Pharma Data

JUNE 28, 2021

Broadens company’s oncology platform of Targeted Alpha Therapies / Acquisition includes actinium-225 labeled differentiated PSMA small molecule for the treatment of prostate cancer. About Targeted Alpha Therapies (TAT) at Bayer Targeted Alpha Therapies (TAT) are an emerging class of radionuclide therapy for various difficult to treat tumors.

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The Pharma Data

OCTOBER 19, 2020

. “Innovation in cancer treatment is realized through collaboration, and the physicians and researchers at Emory’s Winship Cancer Institute have consistently shown their commitment to developing novel cancer therapies using this collaborative approach,” said Chadi Nabhan , M.D., Ramalingam , M.D.,

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Drug Target Review

APRIL 9, 2024

For his postdoctoral stay he joined the group of André Nussenzweig, where he started to work on DNA repair, particularly focusing on the role of histone H2AX. Among other discoveries, the group developed inhibitors of the ATR kinase and showed their potential for cancer therapy.

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Vial

FEBRUARY 19, 2024

Explore the groundbreaking VersAptx bioconjugation platform, designed to elevate cancer therapies and uncover the delicate balance between clinical excellence and commercial viability in the dynamic landscape of pharmaceuticals. Can you elaborate on how the platform allows your company to tailor therapy? Ahmed Hamdy: Absolutely.

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DrugBank

JUNE 27, 2023

The general notion is that patients should be viewed individually, rather than strictly as members of some larger general population, and that their specific genetic background, environment, and lifestyle choices should be considered throughout drug development to the point of treatment and continuing patient care.

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Agency IQ

MARCH 29, 2024

Patients frequently receive up to four therapies at once and go through multiple prolonged courses of treatment to keep recurrence and progression at bay. Commonly combined treatment options include antineoplastics (chemotherapies), radiation therapy, corticosteroids, immunomodulators, proteasome inhibitors, and monoclonal antibodies.

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Conversations in Drug Development Trends

DECEMBER 12, 2022

Genetic testing may help confirm a clinical diagnosis, help predict disease prognosis and progression, facilitate early detection of symptoms, allow for family planning and genetic counseling, inform optimal treatments and therapies, and/or promote enrollment in clinical trials.

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Agency IQ

APRIL 12, 2024

Draft guidance on potency assays for CGT products garners extensive stakeholder input 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. How does “potency” apply to cell and gene therapy (CGT) products?

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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. Nearly three million drug-resistant infections occur annually in the U.S.

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Drug Target Review

JANUARY 15, 2024

Unlike almost every other cell type (except B cells), T cells do not have the exact same chromosomal DNA sequences as other cells in the body. In previous decades, isolation, sequencing and characterisation of DNA encoding a specific TCR was laborious and technically challenging. Engineering soluble T-cell receptors for therapy.

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Broad Institute

OCTOBER 11, 2023

But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way. His mother had a presentation of the disease that suggested her immune system was already on the job.

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Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Peter Marks, head of FDA’s CBER – the organization responsible for regulating gene therapies.

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The Pharma Data

MARCH 22, 2021

Novartis is committed to reimagining prostate cancer through targeted radioligand therapy with 177 Lu-PSMA-617. More than 15 dedicated early to late development and research programs underway to identify the next wave of radioligand therapies for cancer. About 177 Lu-PSMA-617.

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The Pharma Data

DECEMBER 3, 2020

TWO: 4162) announced today that it has entered into a Collaboration and License Agreement with UK-based Sentinel Oncology Limited for advancing the new drug development of SOL-578 , a Checkpoint Kinase 1 (Chk1) inhibitor, under which PharmaEngine will fund the IND enabling studies for SOL-578. TAIPEI , Dec.

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DrugBank

FEBRUARY 26, 2025

Over the past two decades, many pharmaceutical companies have deprioritized antibiotic research due to high development costs and lower profitability compared to treatments for chronic diseases. This stagnation has created a perfect storm, threatening the effectiveness of current therapies and limiting the pipeline of new drugs.

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PLOS: DNA Science

SEPTEMBER 5, 2024

Targeted cancer drugs developed to treat one type of cancer often expand, with further testing, to treat other types, based on molecular similarities. The tool detects cancer cells and pinpoints the origin of the tumor, as well as identifying and assessing gene and DNA patterns to predict patient outcomes and treatment responses.

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Molecule Blog

OCTOBER 1, 2020

While we are finally beginning to understand the key factors that drive aging and are actively developing biomarkers that measure age, we still have a lot of work to do before there is a widely-available therapy that’s proven to increase lifespan. Indication expansion is the use of approved drugs in Longevity applications.

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PLOS: DNA Science

APRIL 27, 2023

” The subtext: Textbooks shouldn’t use only or mostly photos of white people, and interpreting DNA test results shouldn’t be based on research done mostly on white people. ” The term comes from the Orphan Drug Act of 1983, which encouraged drug development for rare conditions.

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