Drug Discovery World

APRIL 17, 2024

DS: CAMP4 leverages research from co-founder Richard Young on how regulatory RNA (regRNA) molecules control the expression of genes. JMB: Regulatory RNAs (or regRNAS) are molecules that directly control the expression of nearby protein-coding genes. DS: How easy was it to transfer theoretical research into a viable business?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

Drug Discovery World

DECEMBER 19, 2023

Developer Immusoft has received FDA Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy, designated ISP-001, in this indication. MPS I is a rare, genetic disease that affects the body’s ability to produce the enzyme alpha-L-iduronidase (IDUA).

Engineer 113

Engineer Trials Genetic Disease Gene Therapy 113

Scienmag

OCTOBER 14, 2020

Known as myofibrillar myopathies, these rare genetic diseases lead to progressive muscle wasting, affecting muscle function and causing weakness.

Genetic Disease 67

Genetic Disease Genetics Protein Research 67

Pharmaceutical Technology

AUGUST 17, 2022

oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. . By self-circularisation, Orna’s oRNA technology makes circular ribonucleic acids (oRNAs) from linear RNAs.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

Drug Discovery World

AUGUST 28, 2023

It is the method for removing unwanted debris, e.g., old proteins, and breaking them down so their constituent parts can be used again. The ones we have focussed on, because the data is so promising and the opportunity so big, are neurodegenerative disorders, in particular Parkinson’s disease and Amyotrophic Lateral Sclerosis (ALS).

Genetics 52

Genetics Protein Genetic Disease Clinical Trials 52

Pharmaceutical Technology

MARCH 1, 2023

Tevard Biosciences and Vertex Pharmaceuticals have announced a four-year global research partnership to develop new tRNA-based therapies to treat Duchenne muscular dystrophy (DMD) patients. The company will be responsible for advancing the research and discovery of new tRNA-based therapies, while Vertex will fund all the programme costs.

Development 130

Development Gene Therapy Genetics Genetic Disease 130

Camargo

JULY 27, 2021

The technology of gene therapy is possible due to extensive DNA research and our resulting understanding of many diseases on the genetic level, and it encompasses several mechanisms such as introducing new genes (gene addition) and inactivating or replacing mutated genes (gene editing).

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

Drug Discovery World

FEBRUARY 28, 2024

Each of the three vaccines in Tri-Ad5 targets different proteins associated with precancer and cancer cells. The vaccine combination is studying whether activation of dendritic cells and training the immune system to recognise those proteins will destroy the precancer cells before the cancer occurs.

Vaccination 52

Vaccination Vaccine Trials Protein 52

Delveinsight

JANUARY 12, 2021

Birinapant is a clinical-stage SMAC mimetic that degrades Inhibitors of Apoptosis Proteins (IAPs) by binding to them, ultimately leading to cell death in tumor cells. Bluebird bio recently announced its plans to split its genetic disease and oncology businesses. and Europe.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

XTalks

AUGUST 6, 2020

TSC is a rare genetic disease that affects approximately 1 in 6,000 people. The disease is caused by mutations on the TSC1 and TSC2 genes, which produce the proteins hamartin and tuberin, respectively.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

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

Drug Discovery World

NOVEMBER 14, 2023

Takeda’s Adzynma has become the first recombinant protein product approved in the US for prophylactic or on-demand enzyme replacement therapy (ERT) for congenital thrombotic thrombocytopenic purpura (cTTP). Without treatment, cTTP is ultimately fatal.

FDA Approval 52

FDA Approval Gene Clinical Trials Clinical Trials 52

The Pharma Data

MARCH 16, 2021

“Von Hippel-Lindau disease is a rare genetic condition for which there is no systemic treatment option available and is associated with a high risk of cancer development in multiple organs. Proteins known as hypoxia-inducible factors, including HIF-2?, Research into VHL biology that led to the discovery of HIF-2?

Drugs 52

Drugs Trials Genetics Protein 52

XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

The Pharma Data

OCTOBER 18, 2020

This study is the first to show that by increasing NAD + , NR helped protect telomeres which are important in addressing life-threatening telomere-related diseases, paving the way for future clinical research. The role of ChromaDex in this study was to provide NR through the ChromaDex External Research Program (CERP).

DNA 52

DNA Scientist Genetic Disease Research 52

The Pharma Data

NOVEMBER 8, 2020

Reese, executive vice president of Research and Development at Amgen. Progeria, also called Hutchinson-Gilford Progeria Syndrome (HGPS), is an ultra-rare and fatal genetic disease of accelerated aging in children. Bristol Myers Squibb’s Lisocabtagene Maraleucel (CAR-T) for R/R Large B-Cell Lymphoma.

Trials 52

Trials Protein Gene Genetic Disease 52

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. Whilst research into mRNA has been ongoing for decade, interest has sparked in recent years and research has begun to expand into areas of vaccinology, infectious diseases and other indications.

RNA 52

RNA Protein Vaccination Vaccine 52

The Pharma Data

OCTOBER 27, 2020

AavantiBio’s strategic partnership with University of Florida’s Powell Gene Therapy Center provide their foundational research in rare genetic disorders. The company’s lead program is aimed at Friedrich’s Ataxia, a rare inherited genetic disease that causes cardiac and central nervous system dysfunction.

Gene 52

Gene Gene Therapy Medicine Genetics 52

Drug Discovery World

MAY 11, 2023

In The Evolving Role of the CRO, panellists showed that research partner organisations and their service provision to R&D companies is essential. However, she also presented thoughts in a technical update, Targeted Protein Degradation – Progress, Direction and Prospects.

Life Science 52

Life Science Genetics Vaccination Vaccine 52

XTalks

MAY 12, 2023

The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. What’s been shown in mouse models of Rett syndrome which also have the genetic disease, is that trofinetide helps strengthen those connections between the neurons. Daybue, also called trofinetide, is a tripeptide.

FDA Approval 97

FDA Approval Genetics Trials Drugs 97

The Pharma Data

DECEMBER 20, 2020

Athenex and Almirall have a license deal with Almirall having the license to research, develop and commercialize the drug in the U.S. CF is a rare, life-shortening genetic disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. and Europe, including Russia.

In-Vitro 52

In-Vitro FDA Approval Manufacturing Licensing 52

XTalks

SEPTEMBER 17, 2020

While the table lists acceptable surrogate endpoints for trials of more common conditions, such as asthma, type 2 diabetes and various cancer types, the document also provides key information to developers of certain rare disease therapies. Dietary changes, including restriction of salt and animal protein, are also recommended.

Trials 98

Trials Gene Hormones Clinical Trials 98

pharmaphorum

SEPTEMBER 9, 2020

At the time, the Sanger Institute was working on the Deciphering Developmental Disorders study (DDD), which looked at the approximately 1-2% of the genome that is responsible for making proteins in patients with rare diseases. Everything the project does has to be rubber-stamped by the patients,” he says. We have a very powerful voice.

Genome 103

Genome Genomics Genetics Genome Project 103

Drug Discovery World

FEBRUARY 28, 2023

mRNA enables the delivery of a transiently expressed genetic molecule that is translated into a target protein using the machinery of the host cell. Therefore, the risk for off-target insertional mutagenesis is greatly reduced. In addition, it may be easier to target the therapeutic molecule to the correct cell or tissue.

Genetics 52

Genetics Genetic Analysis Vaccination Vaccine 52

pharmaphorum

SEPTEMBER 9, 2020

At the time, the Sanger Institute was working on the Deciphering Developmental Disorders study (DDD), which looked at the approximately 1-2% of the genome that is responsible for making proteins in patients with rare diseases. Everything the project does has to be rubber-stamped by the patients,” he says. We have a very powerful voice.

Genome 80

Genome Genomics Genetics Genome Project 80

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. million and $38.8 million, respectively, as compared to $17.6

Genetics 111

Genetics Vaccination Vaccine DNA 111

The Pharma Data

DECEMBER 3, 2020

December 5, 2:00 PM – 3:30 PM; Platform A: Translational Research / Genetics. Translational Research / 2B. New preclinical data provide additional evidence of the potential for TANGO antisense oligonucleotides (ASOs) to provide a gene-specific, disease-modifying treatment for Dravet syndrome. Lori Isom, Ph.D.,

Medicine 52

Medicine Genetics Clinical Trials Clinical Trials 52

pharmaphorum

JANUARY 10, 2022

The breakthrough was hailed as a revolution for disease treatment and particularly a major success against the pandemic. However, research into the action of mRNA had been ongoing since the early 1960s, with the potential for therapeutic purposes being investigated since the 1970s. Where next? Biotech leading the charge.

RNA 111

RNA Vaccination Vaccine Pharma Companies 111

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. The research was published earlier this month in the journal Cell.

DNA 98

DNA Gene Gene Silencing Genome 98

Drug Discovery World

OCTOBER 12, 2023

She says: “From turning genes off to look at the effect on cellular function, making single base changes within a coding or non-coding region to see if it impacts protein function, to testing drug candidates on genetic variants, we now have unprecedented genetic control to understand the link between genetics, disease and treatment.”

Drugs 59

Drugs Life Science Genome Genomics 59

The Pharma Data

OCTOBER 14, 2020

Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases.

Protein 52

Protein RNA Production Genetics 52

The Pharma Data

JULY 6, 2021

Haemophilia A is a serious, inherited bleeding disorder in which a person’s blood doesn’t clot properly, as they either lack or do not have enough of a clotting protein called factor VIII. 11 People with haemophilia A either lack or do not have enough of a clotting protein called factor VIII.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 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. The research was published earlier this month in the journal Cell. pyogenes dCas9.

DNA 52

DNA Gene Gene Silencing Genome 52

Drug Discovery World

MARCH 6, 2024

Guiding neurological drug discovery and development to success The 20 years since the Human Genome Project has seen transformational advances in the molecular understanding of cancers and rare genetic diseases, leading to genetically informed, personalised selection of therapies and massively improved outcomes for many patients.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

XTalks

DECEMBER 22, 2021

RNA, and its protein-generating form messenger RNA (mRNA) discovered in 1961 , has quickly transitioned from being an obscure, finicky molecule that is difficult to work with, to becoming a significant cornerstone of therapeutic innovation in pharma and biotech. percent, according to a report by BCC Research. RNA in the Making.

Life Science 98

Life Science RNA Vaccination Vaccine 98

The Pharma Data

OCTOBER 28, 2020

The interim analysis did not include data from other exploratory outcome measures such as seizure frequency, sleep diaries, EEG patterns, UBE3A protein levels in the CSF, ambulation by wearable device, and adaptive behaviors. This condition is typically not inherited but instead occurs spontaneously. About Ultragenyx.

Protein 40

Protein Production Development Drugs 40

The Pharma Data

AUGUST 3, 2021

SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. Nor can there be any guarantee that such products will be commercially successful in the future.

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52