pharmaphorum

JANUARY 5, 2023

and OriCiro Genomics K.K. However, its messenger RNA (mRNA) platform – building on continuous advances in basic and applied mRNA science – permits the development of therapeutics and vaccines for other infectious diseases, immune-oncology, rare diseases, cardiovascular diseases, and auto-immune diseases as well. Biotech Moderna, Inc.

Gene Sequencing 76

Gene Sequencing DNA Vaccination Vaccine 76

Drug Discovery World

OCTOBER 5, 2023

The presentations will be followed by a panel discussion addressing new drug discovery and development concepts and tools in cancer research, and how these new approaches – e.g. engineered proteins, antibodies, micro RNA, nanocarriers, antibody drug conjugates – can help address unmet need in oncology. More to be announced.

Research 52

Research Antibody Medicine RNA 52

Drug Discovery World

NOVEMBER 1, 2022

He published ‘maps” depicting how various cell types use alternative splicing of messenger RNA to construct genetic templates that produce an ever-changing set of proteins in the cell. . The SLED package released RNA-producing protein that made the melanoma cells die. . Splicing-linked expression design. Initial success in the lab.

Gene Expression 52

Gene Expression Gene Protein Packaging 52

Drug Discovery World

MAY 23, 2024

Reece Armstrong speaks to Samuel Deutsch , Chief Scientific Officer of Nutcracker Therapeutics about the RNA landscape and how it can benefit the development of immunotherapies. RA: What role does RNA play in the development of effective immunotherapies? SD: This is definitely a challenge.

RNA 59

RNA Protein Manufacturing Production 59

XTalks

AUGUST 5, 2020

In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.

Gene Expression 112

Gene Expression Development RNA Drugs 112

Delveinsight

SEPTEMBER 14, 2021

ADARx bags USD 75 Million to advance its RNA tech pipeline. ADARx Pharmaceuticals, a biotechnology company developing RNA targeting therapeutics , announced the completion of a USD 75 million Series B financing to progress its drug development pipeline. SR One Capital Management and OrbiMed Advisors co-led Series B. Simeon George, M.D.,

RNA 67

RNA Clinical Trials Clinical Trials Drugs 67

Drug Discovery World

JANUARY 23, 2023

Following the release of Clarivate’s report ‘RNA Technology Companies to Watch’, DDW’s Diana Spencer speaks to Strand Therapeutics’ Jacob Becraft and HAYA Therapeutics’ Samir Ounzain to find out how this technology could revolutionise disease treatment.

RNA 98

RNA Genome Genomics Engineer 98

XTalks

JUNE 18, 2024

These platforms — Bobcat mRNATM, controllable self-replicating RNA (c-srRNA) and ZSCAN4 delivered by an RNA virus — represent the forefront of genetic therapy, harnessing the power of mRNA to combat diseases at their genetic roots. The essence of the c-srRNA platform lies in its innovative use of temperature as a regulatory mechanism.

Protein 52

Protein RNA Genetics Clinical Trials 52

XTalks

NOVEMBER 30, 2023

Over 26 weeks of Sunlenca combined with other antiretroviral drugs, 81 percent of participants achieved HIV RNA suppression, reaching levels low enough to be considered undetectable. The noteworthy candidate, EBT-101, is designed to eliminate HIV proviral DNA using CRISPR-Cas9 along with two guide RNAs (gRNAs).

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

Drug Discovery World

JANUARY 21, 2024

He has founded and led three bio-AI technology companies, each of which led revolutions in medicine, genomics, agriculture, and conservation. Accelerating biotherapeutics development through technology platforms Anubhav Tripathi is a Professor of Medical Science and Engineering at Brown University.

Protein 52

Protein Vaccination Vaccine Development 52

Roots Analysis

OCTOBER 30, 2023

In the past few years, Next Generation RNA therapeutics have emerged as one of the key therapeutic modalities in the modern healthcare industry. These RNA based therapeutics play a crucial role in protein production and regulation of gene functions.

RNA 40

RNA Vaccination Vaccine Development 40

The Pharma Data

MARCH 23, 2022

Polymerase is a viral protein that directs how Ebola virus replicates its genome as it infects new hosts. Back in 2018, researchers tested a broad-spectrum antiviral candidate called remdesivir/VEKLURY, which acts as a nucleotide decoy to get incorporated into the viral RNA genome and stop viral polymerase.

Protein 52

Protein Genome Genomics Research 52

Drug Discovery World

OCTOBER 16, 2023

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. We also recently opened the Scientific Centre of Excellence for Genomic Insight at our Cambridge site.”

Drugs 75

Drugs Life Science Gene Therapy Genome 75

Drug Discovery World

AUGUST 1, 2023

1 August was first chosen as World RNA Day in 2018 as a play on AUG (adenine, uracil and guanine), a triple sequence of RNA (called a codon) that initiates protein synthesis by the cell. Another potential clinical pathway is to use engineered exogenous circRNAs to produce therapeutic proteins within target cells.

RNA 52

RNA Drugs Gene Silencing Vaccination 52

Drug Discovery World

SEPTEMBER 14, 2023

Moreover, in July 2023, AstraZeneca and Vaxess Technologies commenced a collaboration for the evaluation of a novel RNA-based pandemic influenza prototype vaccine in patch format. In July 2023, AstraZeneca and Ionis Pharmaceuticals expanded their existing collaboration on eplontersen to also include Latin America.

Drugs 59

Drugs Vaccination Vaccine Medicine 59

The Pharma Data

OCTOBER 19, 2020

By leveraging the comprehensive genomic, transcriptomic and proteomic profiling available through the Caris Molecular Intelligence® platform, physicians from Winship will be able to further prioritize therapeutic options and determine which clinical trial opportunities may benefit their patients.

Life Science 52

Life Science Clinical Trials Clinical Trials RNA 52

Drug Discovery World

APRIL 6, 2023

These topics will be represented across a range of session types, including methods workshops, minisymposia, poster and plenary sessions, as well as keynotes, awards and lectures.

Protein 52

Protein Antibody Clinical Trials Clinical Trials 52

XTalks

MARCH 8, 2022

With exciting new applications being developed in the areas of gene therapy, CRISPR and RNA editing, the contributions of women to these fields was highlighted in a piece we published last year on women scientists who paved the way forward in genetics.

Life Science 105

Life Science Gene Therapy DNA Vaccination 105

Drug Discovery World

JULY 11, 2023

Now, with the growth of publicly available genomics, transcriptomics, and proteomics databases, the ability to quickly carry out large-scale DNA, RNA, and protein screenings, and the availability of massive sets of de-identified patient data, the amount of high-value, analysable data has reached enormous proportions.

Big Data 40

Big Data Development Life Science Drugs 40

Drug Discovery World

JULY 2, 2024

Lausanne company HAYA Therapeutics, which focuses on precision medicines targeting the dark genome, raised CHF 25m ($27m) in 2021 and featured in Clarivate’s 2023 report ‘RNA Technology Companies to Watch’.

Life Science 59

Life Science Drugs Development Research 59

Delveinsight

FEBRUARY 25, 2021

Instead of snipping the genome, base editing lets for edits of individual letters in a genetic sequence. The startup spun out of Georgia Tech in 2018 and is based on James Dahlman’s research, a professor in the university’s department of biomedical engineering and school of medicine.

DNA 52

DNA In-Vivo Genetics Medicine 52

The Pharma Data

JANUARY 17, 2021

That’s not the case with RNA-targeted drugs. Working closely with Ionis, “We connect the need to the drug development engine. “It’s the efficiency of antisense technology, coupled with its other characteristics, that makes n-Lorem’s approach feasible.”. Crooke, therefore, created n-Lorem Foundation to do that charitably.

Genetics 52

Genetics Clinical Trials Clinical Trials Drugs 52

Drug Discovery World

JUNE 27, 2024

SeekRNA uses a programmable ribonucleic acid (RNA) strand that can directly identify sites for insertion in genetic sequences, simplifying the editing process and reducing errors. Using the accuracy from this insertion sequence family, seekRNA can be modified to any genomic sequence and insert the new DNA in a precise orientation.

Gene Editing 59

Gene Editing Gene DNA Genetic Engineering 59

Drug Discovery World

OCTOBER 4, 2022

As it stands, there are currently 19 gene therapies approved for clinical use, alongside 18 RNA therapies and 59 non-genetically modified cell therapies, according to the Q2 report by the American Society of Cell & Gene Therapy.

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 52

pharmaphorum

FEBRUARY 3, 2021

These treatments are examples of patient-specific, engineered chimeric antigen receptor (CAR)-T cell therapies that harness the immune system to target and eliminate cancer cells. Thus, the availability of universal, “off-the-shelf” engineered T cells presents an attractive alternative.

Gene Editing 89

Gene Editing Gene DNA Engineer 89

Drug Discovery World

SEPTEMBER 6, 2022

The mysteries of cancer are starting to yield to the range of advanced therapies available and cell therapies – especially engineered cell therapies – are at the leading edge of that progress.” – Carlo Russo, CEO of Genenta.? . “In Research into genomic medicines is only at its infancy. New indications .

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

Roots Analysis

MARCH 16, 2023

The IVT mRNAs are structurally similar to natural mRNAs, these synthetic mRNAs can be used to express proteins through genetic engineering. Cancer Immunotherapies : RNA molecules, such as siRNA, microRNA, and mRNA, have shown tremendous potential for immunomodulation and use as a cancer immunotherapy.

Vaccination 52

Vaccination Vaccine DNA Protein 52

XTalks

DECEMBER 13, 2023

Related: Top 30 Pharma Companies in 2023: Statistics and Trends 1) Moderna Compound annual growth rate: 415 percent Moderna, headquartered in Massachusetts, is a prominent biotechnology company specializing in RNA therapeutics, particularly mRNA vaccines. Alpine Immune Sciences has recently announced an augmentation in its R&D investment.

Genetics 111

Genetics Vaccination Vaccine DNA 111

Drug Discovery World

JULY 26, 2023

With 100-fold the number of genes in the human genome 1 , this microbial collection is a rich genetic signature of clinical significance that we have only recently gained the tools to explore. Genome editing tools such as CRISPR may be employed to create “live biotherapeutics” (drugs composed of living bacteria).

Medicine 52

Medicine Genome Genomics Bioinformatics 52

Drug Discovery World

FEBRUARY 6, 2024

Scientific sessions Tuesday’s presentations spanned a range of topics, including functional genomics for target identification, next-generation proteomics, self-driving laboratories, the future of cell culture, and ‘lab on a particle’ technology.

Life Science 64

Life Science Genome Genomics RNA 64

The Pharma Data

DECEMBER 10, 2020

ImmunityBio has engineered the vector to overcome this problem and has shown that its second-generation adenovirus vector can safely and effectively deliver its cargo even in patients with pre-existing adenovirus immunity. This can significantly limit the effectiveness of first-generation platforms. hAd5-COVID-19 Oral Formulation.

Vaccination 52

Vaccination Vaccine Immune Response Protein 52

XTalks

AUGUST 29, 2022

CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. It is difficult to control where the viral vectors insert genes in the genome, and it is difficult to manufacture large quantities of clinical-grade viral vectors.

Gene Editing 98

Gene Editing DNA Gene Genome 98

Drug Discovery World

DECEMBER 15, 2022

Inducing double strand breaks at unintended locations in the genome can lead to off-target genetic modifications that are difficult to predict and potentially deleterious. Complicating matters even more, researchers currently lack effective and reliable methods for detecting and measuring off-target genomic events.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

AUGUST 10, 2022

Finally, in 2017, after years of improving T cell engineering techniques, the first chimeric antigen receptor (CAR) T cell therapy received FDA approval. If the gene does not integrate into the genome, the cell will not become a CAR T cell. The gene could also bury itself in a silenced region of the genome, leaving the gene dormant.

Manufacturing 98

Manufacturing Gene Genome Genomics 98

The Pharma Data

JANUARY 19, 2021

In the paper, researchers from Johns Hopkins University and elsewhere found a natural long-form transactivating CRISPR RNA (tracr-L) in Streptococcus pyogenes that functions to downregulate its endogenous CRISPR-Cas9 system. But altering the tracr-L with genetic engineering to make it function more like a guide RNA increased CRISPR-Cas9 cuts.

Bacteria 52

Bacteria Gene Editing Research RNA 52

XTalks

MAY 4, 2021

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Epigenetic Editing with CRISPR. DNA methylation occurs endogenously in all mammalian cells in response to various stimuli.

DNA 98

DNA Gene Gene Silencing Genome 98

Drug Discovery World

JANUARY 23, 2023

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector. 2016/17 saw the emergence of this novel CRISPR-Cas9-based approach.

Gene Editing 52

Gene Editing Gene Engineer DNA 52