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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Broad Institute

AUGUST 16, 2023

Nuclear DNA influences variation in mitochondrial DNA By Allessandra DiCorato August 16, 2023 Breadcrumb Home Nuclear DNA influences variation in mitochondrial DNA Whole genomes from hundreds of thousands of people reveal new complexity in how the nuclear and mitochondrial genomes interact, which may influence how cells produce energy.

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Drug Target Review

JULY 5, 2023

Induced pluripotent stem cell (iPSC) therapies have the potential to treat a wide variety of devastating diseases. While iPSC therapies hold great promise, several challenges remain, including poor infiltration of cytotoxic lymphocytes into solid tumours and insufficient cytotoxicity of myeloid cells.

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Drug Discovery Today

FEBRUARY 24, 2022

Global cell and gene therapy development and manufacturing business expands into Bruntwood SciTech’s Alderley Park to boost manufacturing capacity.The 17,000 sq ft high-specification lab space is currently under construction, expanding plasmid DNA and viral vector capabilities in Europe.The facility will support therapeutic developers with rapid access (..)

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ASPET

JUNE 30, 2023

Significance Statement Drugs that target cellular DNA damage repair pathways are now being used as new ways to maximise the effect of multimodality treatments such as radiotherapy. Here, we develop a mathematical model to investigate the effects of ceralasertib and olaparib, two drugs that target DNA damage response pathways.

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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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Fierce BioTech

NOVEMBER 1, 2023

Plasmid DNA Design Considerations for Cell and Gene Therapy Cell and Gene therapy organizations must balance the need for rapid clinical progression and stringent quality expectations throughout development.

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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. This approach targets the fundamental instructions within a cell's DNA, either by correcting faulty genes or introducing entirely new ones to combat disease.

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Broad Institute

JUNE 10, 2024

The advance, from the lab of Broad core institute member David Liu , could one day help researchers develop a single gene therapy for diseases such as cystic fibrosis that are caused by one of hundreds or thousands of different mutations in a gene.

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Chemical Biology and Drug Design

SEPTEMBER 18, 2023

The presence of partially occupied water molecules in the layered organization in the crystal structure mimics the release of reactive oxygen species (ROS) for DNA damage resulting in cytotoxicity.

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Broad Institute

MAY 21, 2025

Scientists create next generation of tools in battle against brain disease By Corie Lok May 21, 2025 Breadcrumb Home Scientists create next generation of tools in battle against brain disease The findings contained in eight studies could lead to targeted gene therapies for brain disorders. Adapted from an Allen Institute press release.

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ASPET

SEPTEMBER 16, 2024

Standard of care GBM therapies include radiation and cytotoxic chemotherapy that lead to DNA damage. Subsequent activation of DNA damage response (DDR) pathways can induce resistance. Elimusertib, a novel ATR kinase inhibitor, can prevent repair of damaged DNA, increasing efficacy of DNA damaging cytotoxic therapies.

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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. There are challenges in delivering the gene/DNA to the site of action in the brain, and scientists are working to resolve the same. Scientists are trying to use viral vectors, miRNA, siRNA, and CRISPR technology.

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Broad Institute

OCTOBER 23, 2024

In recent years, genetic editing technologies and other gene therapy approaches have given scientists the ability to alter the genes inside living cells. That is in part because of the ongoing challenge of understanding the DNA switches, called cis-regulatory elements (CREs), that control the expression and repression of genes.

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Chemical Biology and Drug Design

SEPTEMBER 16, 2024

This study identified MAPK9 as the directly binding target of baicalein by the DNA-programmed affinity labeling method and tested the binding affinity as 89.7 nM Baicalein stabilized MAPK9 to induce apoptosis in hepatocarcinoma cells, which would be helpful to understand and use baicalein in hepatocarcinoma therapy.

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Drug Discovery Today

JULY 7, 2022

Pfizer and Touchlight agree to patent license for Pfizer to utilise rapid, scalable, enzymatic doggybone DNA (dbDNA) in Pfizer’s clinical and commercial manufacture of its mRNA vaccines, therapeutics, and gene therapiesAgreement includes upfront payment, potential development and commercial milestone payments, and royalties upon commercializationAccess (..)

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Drug Target Review

JULY 7, 2023

The reasons for this are multifaceted, including concerns over the safety of directly altering DNA sequences and subsequent regulatory restrictions that have arisen as a result. The epigenome (meaning ‘above the genome’) is a system of reversible marks regulating how the DNA is read, translated and used. What is epigenetic editing?

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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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PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. But even if gene therapy could reach sufficient muscle cells to improve quality of life, another hurdle looms: the dystrophin gene that’s deleted in DMD is huge, the largest in a human genome at 2.2

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ASPET

MARCH 20, 2024

DNA topoisomerase IIβ (TOP2β/180; 180 kDa) is a nuclear enzyme that regulates DNA topology by generation of short-lived DNA double-strand breaks primarily during transcription. TOP2β/180 can be a target for DNA damage-stabilizing anticancer drugs, whose efficacy is often limited by chemoresistance.

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DrugBaron

JANUARY 8, 2021

Yesterday, Sarpeta (NASDAQ: $SRPT) announced that its gene therapy for Duchenne Muscular Dystrophy failed to improve muscle function in a study of 40 boys, despite achieving impressive expression of micro-dystrophin (at least in the short term). Surely, then, a correct set of DNA plans will ensure a correctly functioning cell?

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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. Gene therapy trials explore using lentiviral vectors to deliver a functional copy of the HBB gene to hematopoietic stem cells, the progenitors of all blood cells.

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Drug Target Review

SEPTEMBER 28, 2023

After some time in that role and launching several products, I received a call from Bill Banyai and Bill Peck, or ‘The Bills’ as we call them, who were building a company around technology that creates DNA by ‘writing’ it on a silicon chip. We’ve miniaturised the chemical process to making DNA and synthesize or ‘write’ DNA on a silicon chip.

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Broad Institute

NOVEMBER 13, 2023

Liquid biopsies enable clinicians to find and analyze tumor DNA in a patient’s blood sample to detect cancer early, monitor cancer recurrence, assess the patient’s response to treatment, and measure other clinically important features in real time, without invasive procedures.

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Drug Target Review

AUGUST 3, 2023

Part of the problem is that only a fraction of the population, as little as 1-2 percent, has been DNA tested in a healthcare setting (though more have used at-home direct-to-consumer genetic testing kits). We also permit people who have taken consumer DNA tests, such as with Ancestry or 23andMe, to upload their results onto our site.

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KIF1A

APRIL 6, 2024

When a mutation causes a deletion or duplication of larger areas of DNA, we call it Copy Number Variation. DNA deletion and duplication may impact large sections of genes, or multiple neighboring genes. The altered expression of so many genes has profound consequences for the nervous system. She graduated from UC San Diego with a B.S.

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Covalent Modifiers

AUGUST 14, 2023

Acquired cysteines are both driver mutations and sites targeted by precision therapies. For both cancer and healthy genomes, we find that cysteine acquisition is a ubiquitous consequence of genetic variation that is further elevated in the context of decreased DNA repair.

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The Premier Consulting Blog

MARCH 7, 2024

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

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Broad Institute

JANUARY 8, 2024

In the new work published today in Nature Biotechnology , the team adapted engineered virus-like particles (eVLPs) that they had previously designed to carry base editors — another type of precision gene editor that makes single-letter changes in DNA. Nature Biotechnology. Online January 8, 2024.

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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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NIH Director's Blog: Drug Discovery

MARCH 8, 2016

Instead of the black, printed stripes of the Universal Product Codes (UPCs) that we see on everything from package deliveries to clothing tags, they used short, unique snippets of DNA to label cells. DNA barcoding has already empowered single-cell analysis, including for nerve cells in the brain. PRISM consists of two key components.

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Alta Sciences

APRIL 16, 2024

Inside The Altascientist: The Benefits of PCR for Your Gene Therapy Programs pmjackson Tue, 04/16/2024 - 19:04 The gene therapy landscape continues to accelerate in preclinical and clinical research, with programs constantly in development for targeted, personalized medicines. But how are PCR analysis techniques applied?

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Codon

JUNE 11, 2024

And unlike traditional DNA sequencers, which parse genetic material by breaking it up into fragments and interpreting it chunk-by-chunk, a nanopore device unspools a long strand of DNA and reads it all at once. A scientist can isolate DNA and load up a flow cell in fifteen minutes. Nanopore devices work incredibly fast.

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Chemical Biology and Drug Design

DECEMBER 13, 2024

DNA G-quadruplexes (G4) have emerged as potential regulatory targets for c-Myc expression. These findings provide valuable insights for the development of G4-targeting drugs for cancer therapy. Previous studies identified trovafloxacin, a topoisomerase II inhibitor, as a novel c-Myc G4 stabilizer.

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Broad Institute

JULY 10, 2024

Prime editing efficiently corrects cystic fibrosis mutation in human lung cells By Allessandra DiCorato July 10, 2024 Breadcrumb Home Prime editing efficiently corrects cystic fibrosis mutation in human lung cells The approach targets the most common genetic cause of the disease and could enable a one-time treatment as effective as existing daily therapies. (..)

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PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. DEB has been a candidate for a gene therapy since 2002.

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DrugBank

MARCH 12, 2025

  The Expanding Role of mRNA in Cancer Therapy One of the most exciting applications of mRNA therapeutics lies in cancer treatment, where leveraging the immune system to target tumors offers a novel approach. This approach can be personalized for each patient by sequencing the tumor's DNA and RNA to identify unique mutations.

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