Disease, Protein Expression and RNA - Drug Discovery Digest

Disease

Protein Expression

RNA

A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

JUNE 27, 2024

By Greta Friar, Whitehead Institute June 27, 2024 Images of a mouse brain show the effect of a technology called CHARM in turning off the expression of a gene in the brain. CHARMs are an elegant solution to the problem of silencing disease genes, and they have the potential to have an important position in the future of genetic medicines.” “The

Disease

Disease Therapies Engineering Clinical Trials

Addressing increasingly resistant drugs by infectious agents

Drug Target Review

SEPTEMBER 4, 2023

There have been several studies that have reported the ability of infectious disease agents to be drug resistant. Infectious agent includes bacteria, virus, fungi, and parasites, and they are capable of causing disease in a living organism. They could be spread by physical contact, airborne, waterborne, foodborne, or vector-borne.

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Chemical Biology and Drug Design

The Role of Oncology Biomarkers in Personalizing Hematology Treatment Plans

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. Molecular Biomarkers : Encompass various molecules, such as RNA and metabolites, to reflect the physiological state of the cells and their disease pathways.

Treatment

Treatment Therapies Disease Protein Expression

The Future of Drug Discovery: Tackling the Undruggable with New Biotechnologies

DrugBank

JULY 24, 2024

This is a game-changer, especially in the fight against cancer and other complex diseases. These multifunctional small molecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. This can potentially correct the root causes of some diseases at the genetic level.

Small Molecule

Small Molecule Drugs Clinical Trials Therapies

Enhancing gene therapy with Circio

Drug Target Review

MAY 22, 2024

What are the key findings of Circio’s in vivo proof-of-concept for its circVec circular RNA platform technology compared to conventional mRNA-based expression with DNA vectors? Circular RNA (circRNA) has two major advantages versus mRNA in a vector-expression context. DNA vectors in mouse models?

Therapies

Therapies RNA Protein Expression DNA

Biopharma Money on the Move: December 2 – 8

The Pharma Data

DECEMBER 8, 2020

Some of the funds will be used for the development of therapeutics for oncology and other serious diseases through its proprietary ImmunoTAC technology platform. The ImmunoTAC platform pairs proprietary payloads that modulate key disease modifying pathways with monoclonal antibodies directed at specific disease sites.

RNA

RNA Therapies Disease Protein Expression

Second-Generation mRNA COVID-19 Vaccine Candidate, CV2CoV, Demonstrates Improved Immune Response and Protection in Preclinical Study

The Pharma Data

AUGUST 16, 2021

The CureVac-GSK COVID-19 collaboration announced in February 2021 extends the existing strategic mRNA technology partnership both companies started in July 2020, which focuses on the development of new products based on CureVac’s second-generation RNA-technology for different targets in the field of infectious diseases.

Immune Response

Immune Response Vaccine Virus RNA

Marvin Caruthers receives inaugural Merkin Prize in ceremony at the Broad Institute for DNA synthesis technology

Broad Institute

OCTOBER 4, 2023

Today, scientists use such reactions to produce the customizable DNA and RNA molecules that enable genetic sequencing, drug and vaccine development, pathogen tests, cancer diagnostics, and more. Wang concluded her presentation by expressing gratitude to Merkin. I’m really thankful for the support of the Merkin Fellowship,” she said. “It

DNA

DNA RNA Science Protein Expression

Codon Digest: CAR-T Therapy for Neuroblastoma

Codon

APRIL 9, 2023

Small snippets of double-stranded RNA were sprayed onto hot pepper plants to control a pest, called Frankliniella occidentalis. The RNA strands shut down a specific gene in the insects, and killed half after 7 days. RNA may offer a safer form of pest control in the future. The new sequences might help de-extinction efforts.

Therapies

Therapies RNA Engineering Vaccine

Five Promising Treatment Areas in Early-Phase Drug Development in 2024

Alta Sciences

APRIL 17, 2024

Gene Therapy Gene therapy is currently considered for diseases, often rare, that have no other cures. We have conducted over 130 in-life studies in the last five years (over 95% conducted in NHPs), including IND-enabling studies for rare diseases and CRISPR gene editing therapeutics in NHPs.

Drug Development

Drug Development Treatment FDA Approval Drugs

Clinical Catch-Up: January 4-8 | BioSpace

The Pharma Data

JANUARY 10, 2021

Forge Therapeutics received FDA clearance to initiate a Phase I/II trial of its FBX-101 for patients with Krabbe disease. Krabbe disease is a rare and fatal pediatric leukodystrophy caused by mutations in the galactosylceramidase (GALC) gene. ENPP1 deficiency is a rare, chronic, systemic, and progressive disease.

Clinical Trials

Clinical Trials Trials FDA Vaccine

Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Conversations in Drug Development Trends

MAY 30, 2024

The peak number of trials in the last five years and the regulatory approvals of three additional disease-modifying treatments for ALS in the previous eight years signal cause for hope that, as a community, we have the potential to transform this progressive and ultimately terminal neurodegenerative disorder into a treatable condition.

Treatment

Treatment Clinical Trials Small Molecule Therapies

Novartis Provides Update on AVXS-101 Intrathecal Clinical Development Program

The Pharma Data

SEPTEMBER 22, 2020

All patients deserve a gene therapy designed to address the genetic root cause of their disease with a single dose. The company’s commitment to SMA extends beyond gene therapy to branaplam (LMI070), an oral, once-weekly RNA splicing modulator also currently under development, to expand the treatment options for SMA patients. .

Clinical Development

Clinical Development Therapies Clinical Trials Treatment

A therapy candidate for fatal prion diseases turns off disease-causing gene

Addressing increasingly resistant drugs by infectious agents

Trending Sources

The Role of Oncology Biomarkers in Personalizing Hematology Treatment Plans

The Future of Drug Discovery: Tackling the Undruggable with New Biotechnologies

Enhancing gene therapy with Circio

Biopharma Money on the Move: December 2 – 8

Second-Generation mRNA COVID-19 Vaccine Candidate, CV2CoV, Demonstrates Improved Immune Response and Protection in Preclinical Study

Marvin Caruthers receives inaugural Merkin Prize in ceremony at the Broad Institute for DNA synthesis technology

Codon Digest: CAR-T Therapy for Neuroblastoma

Five Promising Treatment Areas in Early-Phase Drug Development in 2024

Clinical Catch-Up: January 4-8 | BioSpace

Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Novartis Provides Update on AVXS-101 Intrathecal Clinical Development Program

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