pharmaphorum

JULY 20, 2022

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on. .

RNA 52

RNA Genetic Disease Drugs Protein 52

BioSpace

JUNE 20, 2022

Therorna brought in $42 million to advance its circular RNA platform, while Carbon Biosciences banked $38 million to develop therapies for genetic diseases, beginning with cystic fibrosis.

Genetic Disease 64

Genetic Disease RNA Genetics Development 64

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Moderna will also assume further development, manufacturing, and commercialisation responsibilities on exercising a target option.

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

BioPharma Reporter

FEBRUARY 23, 2023

The messenger RNA (mRNA) specialist Moderna has teamed up with ElevateBio-owned Life Edit Therapeutics to develop gene editing therapies that are delivered into patients in vivo.

Gene Editing 52

Gene Editing Genetic Disease Gene In-Vivo 52

pharmaphorum

OCTOBER 5, 2022

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule. billion deal in 2018. The aim will be to identify promising candidates in areas of “unmet patient need.”

RNA 105

RNA Genetics Dermatology Antibody 105

Delveinsight

AUGUST 26, 2021

Seattle biotech firm Shape Therapeutics has signed a deal potentially exceeding USD 3 billion with pharma giant Roche to bolster the development of gene therapies for Alzheimer’s and Parkinson’s disease. Shape’s RNA editing technologies can modify the RNA sequence, which makes the body’s protein building blocks.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

pharmaphorum

MAY 6, 2021

Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood. Severely-affected patients need regular blood transfusions to maintain their haemoglobin levels.

Genetic Disease 104

Genetic Disease Protein RNA Doctors 104

XTalks

AUGUST 29, 2022

Alex Marson, MD, PhD, and senior author of the study states that the research team has developed a method to insert lengthy DNA sequences into a target site in the genome without the use of viral vectors. The results demonstrate potential as a novel, safe and effective approach to cell therapies.

Gene Editing 98

Gene Editing DNA Gene Genome 98

Pharmaceutical Technology

APRIL 24, 2023

Robert Langer was part of the first cohort of researchers to begin nanoparticle research , as his lab at Cornell University developed tiny particles to deliver large molecules for angiogenesis. Langer’s engineering lab was instrumental in the development of these lipid nanoparticles, playing into his role in co-founding Moderna.

Drug Delivery 147

Drug Delivery Vaccination Vaccine Research 147

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., as Head of Business Development. .–( BUSINESS WIRE )– EdiGene, Inc. as Head of the US Subsidiary, and Kehua Fan, M.D.,

Business Development 40

Business Development Development Gene Editing In-Vivo 40

Roots Analysis

FEBRUARY 21, 2022

Advancement in DNA sequencing technologies have resulted in noteworthy developments in various healthcare-related research fields, such as diagnostics and personalized medicine. As per our analysis, majority of the kits use purified RNA / DNA as the input sample. Diverse Landscape of NGS Library Preparation Kits. Concluding Remarks.

Marketing 52

Marketing Gene Sequencing DNA Genome 52

The Pharma Data

DECEMBER 8, 2020

AbCellera’s AI-powered antibody discovery platform speeds the otherwise lengthy and grueling process by analyzing the database of natural immune systems to find antibodies that can be developed into drugs. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost. Sigilon Therapeutics

RNA 52

RNA Antibody Life Science Protein 52

The Pharma Data

OCTOBER 14, 2020

is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA.

Protein 52

Protein RNA Production Genetics 52

Roots Analysis

AUGUST 12, 2024

Molecular diagnostic tests refer to tests intended to detect specific sequences in human genomic samples, such as DNA or RNA, in order to diagnose a particular disease. Further, such tests also aim to identify single nucleotide polymorphisms, deletions, rearrangements, and insertions in genetic sequences.

Genetics 40

Genetics Gene Expression RNA DNA 40

The Pharma Data

NOVEMBER 29, 2020

The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs. Typically, it is children who suffer from the disease. It is an RNA interference (RNAi) therapeutic. It was accepted for priority review on June 2. The FDA approved the drug on November 24.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

The Pharma Data

JANUARY 17, 2021

It’s the cause of the mutation, not the name of the disease, that matters.”. The treatments all use the antisense technology and automation techniques developed at Ionis Pharmaceuticals , which Crooke founded. That’s not the case with RNA-targeted drugs. “We focus on the organs we really understand,” he emphasized.

Genetics 52

Genetics Clinical Trials Clinical Trials Drugs 52

XTalks

JANUARY 3, 2025

Investigational drug plozasiran , developed by Arrowhead Pharmaceuticals, is under review for FCS treatment. As Tryngolza becomes available, patients and healthcare providers are optimistic. However, Tryngolza is not the only potential solution in the pipeline.

FDA Approval 59

FDA Approval Drugs Clinical Trials Clinical Trials 59

Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. While most gene therapy clinical studies are ongoing, a number of products are in advanced clinical development, and several are approved by FDA. PK Planning for Gene Therapy Development Programs. Route of Administration Considerations.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

XTalks

FEBRUARY 24, 2025

Carlsbad, California-based Ionis Pharmaceuticals announced it will be sharing promising new trial data for its RNA-targeted prophylactic treatment donidalorsen for hereditary angioedema (HAE) at the 2025 American Academy of Allergy, Asthma & Immunology (AAAAI)/World Allergy Organization (WAO) Joint Congress in San Diego, California.

Trials 59

Trials RNA Allergies Drugs 59

XTalks

APRIL 1, 2025

Some individuals with hemophilia A may develop inhibitors against factor VIII, while those with hemophilia B can develop inhibitors against factor IX. Through RNA silencing, it targets the expression of antithrombin, a protein that inhibits blood clotting. Hemophilia affects approximately 33,000 males in the US.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

Pharma Mirror

APRIL 21, 2024

With the rapid development of biotechnology and molecular medicine, the introduction of mRNA as a vaccine or therapeutic agent enables the production of almost any desired functional protein/peptide within the human body.

In-Vitro 130

In-Vitro Drugs Genetic Disease RNA 130

XTalks

DECEMBER 22, 2021

The development and widespread adoption of new technologies is key to revolutionizing the way we diagnose, prevent, treat and manage disease. The RNA Revolution: From mRNA Vaccines to RNA Editing. The age of RNA is officially here, and it’s here to stay as more than a passing life science trend. RNA in the Making.

Life Science 98

Life Science RNA Vaccination Vaccine 98

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR.

DNA 98

DNA Gene Gene Silencing Genome 98

The Pharma Data

SEPTEMBER 22, 2020

Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. This guidance provides clarity on the path to registration for AVXS-101 IT.

Clinical Development 52

Clinical Development Gene Therapy Development Gene 52

The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. Together, the ILLUMINATE studies comprise a comprehensive clinical development program intended to demonstrate the safety and efficacy of Oxlumo across the full spectrum of patients diagnosed with PH1.

FDA Approval 52

FDA Approval Production RNA Medicine 52

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. Epigenome Editing with CRISPR.

DNA 52

DNA Gene Gene Silencing Genome 52

XTalks

DECEMBER 13, 2023

In 2023, biotechnology and pharmaceutical companies leverage cutting-edge technology and significant investments in research and development (R&D) to continue pushing the boundaries of innovation in the healthcare sector, transcending the challenges posed by the pandemic.

Genetics 111

Genetics Vaccination Vaccine DNA 111

XTalks

SEPTEMBER 13, 2021

With RNA therapies being the next hot thing in genetic medicine, Eli Lilly is joining the RNA editing race by partnering with Netherlands-based ProQR Therapeutics NV (Nasdaq: PRQR), a biotech company developing RNA-based therapies for rare genetic diseases with a focus on blinding disorders of the retina.

RNA 105

RNA Genetics Genetic Disease Medicine 105

pharmaphorum

JANUARY 10, 2022

Previously, messenger RNA (mRNA) therapies were a niche part of the global R&D pipeline, now a wide section of the public is at least familiar with the name of this type of therapy. The company has also stated, at the same time as the study was announced, that it will look at developing mRNA technology in oncology and genetic diseases.

RNA 111

RNA Vaccination Vaccine Pharma Companies 111

The Pharma Data

DECEMBER 27, 2020

Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. The drug is a humanized IgG4 monoclonal antibody that blocks CCR5 and is being developed for COVID-19, HIV and metastatic triple-negative breast cancer. It is being developed for relapsed CD19+ leukemias and lymphomas.

Trials 52

Trials Antibody Gene Therapy Gene 52

XTalks

JANUARY 6, 2025

From leveraging artificial intelligence (AI) to streamline diagnostics and treatments to exploring the untapped potential of RNA-based therapeutics, biotechnology is shaping the future of healthcare and beyond. As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas.

Gene Therapy 111

Gene Therapy RNA Gene Editing Gene 111