Broad Institute

MAY 16, 2024

By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor.

Therapies 137

Therapies FDA Approval Engineering Disease 137

Drug Target Review

JULY 11, 2025

Neurological diseases are among the most complex and least understood conditions in medicine. Despite the knowledge that certain genes increase the risk of contracting diseases like Alzheimer’s and Parkinson’s, it is still not fully understood how those diseases develop in the brain. But that is beginning to change.

Molecular Biology 69

Molecular Biology Disease Therapies Bioinformatics 69

Drug Target Review

APRIL 7, 2025

As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. These fields explore highly precise biological processes related to RNA, specific proteins and gene expression mechanisms.

Clinical Development 69

Clinical Development Clinical Trials Clinical Pharmacology Trials 69

BioPharma Drive: Drug Pricing

JUNE 3, 2024

With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.

Science 141

Science RNA Therapies Disease 141

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. We are studying the placement of organelles within cells and how they communicate to help better treat disease,” said Coskun.

RNA 128

RNA Immune Response Therapies Disease 128

The Pharma Data

JUNE 6, 2025

These new findings reinforce the company’s growing leadership in rare hematologic diseases, with a focus on transformative treatments for immune thrombocytopenia (ITP) and hemophilia. the therapy has been granted Fast Track Designation , and the Food and Drug Administration (FDA) is expected to make a regulatory decision by August 29, 2025.

Disease 40

Disease Therapies Treatment International 40

LifeSciVC

JUNE 12, 2025

Founding Vision: Harnessing RNA Editing for Transformative Medicine Korro Bio’s story began in 2018, co-founded by a remarkable group: Jean-François Formela, M.D. Academic specializing in RNA editing). The ability of modifying RNA enabled us to sidestep many of the potential risks associated with permanent DNA editing.

RNA 67

RNA Protein Expression DNA Science 67

Drug Target Review

JUNE 30, 2025

“I was increasingly drawn to understanding the underlying biology of disease at a molecular level – the ‘why’ – and how we might harness science to create better medicines for patients,” Lepore says. From theory to therapy This approach is already delivering promising insights across key therapeutic areas.

Disease 59

Disease Medical Schools Science Therapies 59

Elrig

MARCH 25, 2025

This second ELRIG meeting on Therapeutic Oligonucleotides brings together esteemed scientists from academia, industry, and other members of the drug discovery community to explore the discovery, validation, and targeting of oligonucleotide-based drug candidates, including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA).

RNA 59

RNA Bioinformatics Science Pharmacokinetics 59

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.

Therapies 103

Therapies DNA Engineering Virus 103

Drug Target Review

DECEMBER 6, 2024

This includes verifying the experimental design and understanding how the data was generated whether it was from RNA sequencing, mass spectrometry, or other biological assays. Figure 1: High-level workflow for early drug discovery Once the raw data has been gathered, the next step is to gain a thorough understanding of the data.

RNA 52

RNA Drugs Research Science 52

Broad Institute

JANUARY 8, 2024

Now the researchers describe how they re-engineered both eVLPs and parts of the prime editing protein and RNA machinery to boost editing efficiency up to 170 times in human cells compared to the previous eVLPs that deliver base editors. By Sarah C.P.

Engineering 144

Engineering Research Virus DNA 144

Drug Target Review

DECEMBER 16, 2024

Since their first approvals in 2017, autologous CAR T-cell therapies have revolutionised the treatment of blood cancers, offering hope where previous options had failed. Consequently, these therapies come with significant burdens and are often underutilised despite their transformative patient outcomes.

Therapies 52

Therapies Clinical Trials Treatment Trials 52

The Pharma Data

JUNE 18, 2025

Pushkal Garg to Lead Unified Research and Development Organization, Signaling Strategic Expansion into Next Phase of RNAi Therapeutics Innovation Alnylam Pharmaceuticals , a global leader in RNA interference (RNAi) therapeutics, has announced the promotion of Pushkal Garg, M.D.,

RNA 40

RNA Clinical Development Science Disease 40

Broad Institute

OCTOBER 23, 2024

By Leah Eisenstadt, Broad Communications October 23, 2024 Credit: Courtesy of the Broadbent family Brian and Julia Broadbent are raising their daughters Claire, top left, and Emma, seated, who is the first person to be diagnosed with a rare genetic disorder caused by the long noncoding RNA CHASERR.

DNA 128

DNA RNA Disease Hospitals 128

DrugBank

JANUARY 29, 2025

Gene Therapy Gene therapy operates on the principle of modulating the DNA blueprint of cells to induce a therapeutic response. Each of these mechanisms represents a distinct strategy to address the underlying genetic perturbations contributing to disease pathogenesis.

Therapies 52

Therapies DNA RNA Disease 52

DrugBank

MARCH 12, 2025

Introduction Messenger RNA (mRNA) technology has emerged as one of the most significant medical breakthroughs. However, mRNA technology is not limited to infectious diseases. However, mRNA technology has introduced new possibilities, particularly in developing personalized cancer vaccines and combination therapies.

Vaccine 97

Vaccine Treatment Clinical Trials Therapies 97

The Pharma Data

MARCH 9, 2022

Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets.

Licensing 52

Licensing Licensing Therapies Disease 52

ASPET

OCTOBER 8, 2024

However, the disease eventually progresses as castration-resistant PCa (CRPC). Finally, RNA sequencing revealed a potential link in the downregulation of Ras/MAPK signaling following combination treatment. Androgen deprivation is the standard treatment for prostate cancer (PCa) patients.

RNA 100

RNA Treatment Therapies Disease 100

DrugBank

JULY 24, 2024

This is a game-changer, especially in the fight against cancer and other complex diseases. For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed. This can potentially correct the root causes of some diseases at the genetic level.

Small Molecule 98

Small Molecule Drugs Clinical Trials Therapies 98

Broad Institute

NOVEMBER 7, 2023

While RA therapies targeted to specific inflammatory pathways have emerged, only some patients’ symptoms improve with treatment, emphasizing the need for multiple treatment approaches tailored to different disease subtypes. They also found that patients’ CTAPs were dynamic and could change over time in response to treatment.

Treatment 96

Treatment RNA Hospitals Disease 96

Drug Target Review

JULY 11, 2024

To date, there are less than 30 known surface proteins that form the basis of all approved cancer-targeted therapies of all modalities, including antibody-drug conjugates (ADCs), T-cell engagers, CAR T-cells and radiopharmaceuticals. These could be targets for antibody-drug conjugates (ADCs), bi-specific antibodies, or other therapies.

Therapies 56

Therapies RNA Pharma Companies Disease 56

Crown Bioscience

JULY 15, 2024

The rapid increase of precision oncology approaches in recent years has been fueled in part by technological innovations and an increased interest in personalized therapies.

RNA 52

RNA Therapies Treatment Disease 52

Drug Target Review

JULY 7, 2023

Many disease states are caused by a web of complex interactions rather than single-gene mutations. This gives epigenetic editing the unique ability to treat complex multigenic diseases and opens the door to enhancing cell and regenerative therapies, which require the manipulation of multiple genes for optimal results.

DNA 105

DNA Engineering Therapies Regulations 105

LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Join the club.

Small Molecule 143

Small Molecule Trials Therapies Disease 143

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.

Small Molecule 105

Small Molecule Therapies Regulations Treatment 105

PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. Muscle makes up about 40 percent of body weight. million DNA bases.

FDA Approval 94

FDA Approval Treatment FDA DNA 94

Conversations in Drug Development Trends

AUGUST 14, 2024

Heterogeneity in Hematological Cancers Due to their disease heterogeneity, hematological cancers are well-suited for personalized approaches. Hematological cancer cells can also develop resistance to therapies over time, reducing treatment effectiveness.

Treatment 78

Treatment Therapies Disease Protein Expression 78

FDA Law Blog: Drug Discovery

JANUARY 12, 2025

FDAs interpretation of several threshold criteria for eligibility (serious condition, available therapy, unmet medical need) will continue to rely upon the 2014 Guidance. However, the 2014 Guidance cannot be ignored entirely. However, while FDA reiterated its interpretation that the statutory requirement to take into account.

FDA 64

FDA Clinical Trials Trials Therapies 64

KIF1A

JULY 29, 2023

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. But in the last few years we’ve seen a culture shift that has made rare disease research much more viable.

RNA 98

RNA Clinical Trials Disease Science 98

Broad Institute

AUGUST 19, 2024

Using RNA sequencing and working with the Broad’s Metabolomics Platform and collaborators at St. I’m so proud of this team and the partnership we formed to follow the science and set up some major positive impacts on women’s health,” Blainey said.

Hospitals 121

Hospitals Treatment Medical Schools Science 121

DrugBank

MAY 15, 2024

Today, we're able to identify and target specific molecules involved in disease processes—a method that's much more like using a sniper rifle than throwing darts blindfolded. Among these targets are proteins, receptors, and enzymes that are fundamental to disease mechanisms.

Small Molecule 98

Small Molecule Molecular Biology Drugs Disease 98

Broad Institute

DECEMBER 13, 2023

Related news Researchers map brain cell changes in Alzheimer’s disease Courtesy of the Chen and Macosko labs. The researchers also discovered clues about cellular function and the potential roles of brain structures in disease. Courtesy of the Chen and Macosko labs. Courtesy of the Chen and Macosko labs.

DNA 140

DNA Virus Research Engineering 140

Broad Institute

JANUARY 22, 2024

As part of the Cancer Program at the Broad, the Sellers lab explores the link between genetic alterations and cancer dependencies with the goal of informing new therapies. Initially, there were two approaches: small hairpin RNA (shRNA)-based and now CRISPR-based experiments. There are a number of limitations to those approaches.

Drugs 128

Drugs Therapies Regulations RNA 128

Broad Institute

JUNE 30, 2023

Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize. Common genetic variants associated with cardiometabolic disease can produce phenotype changes of such small effect that they can be difficult to characterize.

Small Molecule 105

Small Molecule Cell Based Assays Disease Assay Development 105

The Pharma Data

SEPTEMBER 30, 2021

“We used NanoSTING because the adjuvant for intranasal vaccination and single-cell RNA-sequencing to verify the nasal-associated lymphatic tissue as an inductive site upon vaccination. a pioneering biotech company developing novel intranasal vaccines and therapies to assist patients defeat debilitating diseases, including COVID-19.

Vaccine 52

Vaccine Disease Immune Response Virus 52

Alta Sciences

MARCH 1, 2024

Norbert Makori and Pierre Jolicoeur: The Benefits of Combining Preclinical and Bioanalytical for Your Gene Therapy Studies pmjackson Fri, 03/01/2024 - 22:20 Recent advances in gene therapy have allowed for diseases to be approached differently. q: How can Altasciences support sponsors’ Gene Therapy Programs?

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Therapies Laboratories Molecular Biology RNA 52