XTalks

DECEMBER 4, 2024

Proceeds from the IPO will propel key initiatives, including the Phase II clinical trial of Jotrol in Parkinson’s disease. While Jupiter faces financial uncertainties and challenges in scaling operations in competitive markets, it remains committed to refining grant applications for proof-of-concept trials.

Bioavailability 111

Bioavailability Genetic Disease Marketing RNA 111

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). Formulation Considerations.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

XTalks

APRIL 1, 2025

Hemophilia A and B are rare genetic bleeding disorders caused by a deficiency or dysfunction of clotting factors VIII (FVIII) and IX (FIX), respectively. Through RNA silencing, it targets the expression of antithrombin, a protein that inhibits blood clotting. The frequent intravenous infusions can be challenging for patients.

FDA Approval 59

FDA Approval RNA Clinical Trials Clinical Trials 59

Pharmaceutical Technology

NOVEMBER 10, 2022

The siRNA therapy is created to reduce lipoprotein(a) [Lp(a)] levels, which is a genetically decided, independent cardiovascular disease risk factor. According to data from the Phase II OCEAN(a)-DOSE trial, olpasiran was found to offer a substantial and lasting decline in levels of Lp(a) levels over 36 weeks.

RNA 130

RNA Sales Clinical Trials Clinical Trials 130

XTalks

JUNE 20, 2022

Alnylam Pharmaceuticals, a leading RNA interference (RNAi) therapeutics biopharmaceutical company, announced it received approval from the US Food and Drug Administration (FDA) for its RNAi therapeutic Amvuttra (vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults.

FDA Approval 98

FDA Approval Protein Genetics RNA 98

Worldwide Clinical Trials

AUGUST 14, 2024

Historically, these indications have challenged the one-size-fits-all treatment approach due to patient variability, such as genetic differences in drug metabolism and underlying health conditions. This resistance may occur due to genetic mutations, epigenetic changes, or a tumor’s microenvironment.

Genetics 195

Genetics Protein Gene Genotype 195

pharmaphorum

JUNE 28, 2021

Preliminary results from a phase 1 trial run by Regeneron and CRISPR specialist Intellia Therapeutics – co-founded by Nobel Prize winner Jennifer Doudna – showed steep reductions in a biomarker of ATTR amyloidosis disease activity with a single dose of the NTLA-2001 drug. In three more patients treated at 0.1mg/kg levels fell by 52%.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105

Worldwide Clinical Trials

MAY 30, 2024

Authors: Rich Worldwide Clinical Trials Exec. This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. Director, Therapeutic Area Medical Lead.

Gene 204

Gene Clinical Trials Clinical Trials Gene Therapy 204

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

XTalks

DECEMBER 22, 2021

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 technology is not new nor has its potential been surprising. RNA in the Making. So why did this perceived RNA ‘revolution’ take so long?

Life Science 98

Life Science RNA Vaccine Vaccination 98

XTalks

OCTOBER 26, 2021

The Phase III trial results show that Givlaari offers sustainable benefit to AIP patients and confirm its favorable safety profile. AHP is a life-threatening genetic condition caused by an inherited deficiency in the metabolic enzyme hydroxymethylbilane synthase (HMBS), also known as porphobilinogen deaminase (PBGD).

Gene Silencing 98

Gene Silencing Gene Clinical Trials Clinical Trials 98

Pharmaceutical Technology

DECEMBER 14, 2022

GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. The alliance will merge the PRISM oligonucleotide platform of Wave and the capabilities of GSK in genetics and genomics.

Life Science 162

Life Science Development Gene Therapy Genetics 162

XTalks

JANUARY 21, 2025

In a clinical trial, participants demonstrated a 98% success rate after six months post implantation, with arteries widened successfully and no stent fractures observed. There, plasma is separated, and tumor DNA is identified by detecting genetic mutations, methylation patterns and fragmentation signals.

FDA Approval 105

FDA Approval Manufacturing Development RNA 105

XTalks

SEPTEMBER 4, 2024

Alnylam Pharmaceuticals announced promising results from its HELIOS-B Phase III clinical trial evaluating vutrisiran, an investigational RNA interference (RNAi) therapeutic for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). As of March 2024, the FDA has approved six small interfering RNA (siRNA) therapies.

Trials 52

Trials RNA Protein Gene Editing 52

pharmaphorum

DECEMBER 13, 2021

In June, BioNTech started the first phase II trial of BNT111, an mRNA vaccine candidate for skin cancer and later announced plans to move mRNA therapeutics against malaria and tuberculosis into the clinic in 2022 as well as plans to set up a sustainable vaccine production and supply network in Africa. mRNA-based vaccine trial development.

RNA 105

RNA Vaccine Vaccination Development 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 Vaccine Vaccination Pharma Companies 111

Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.

Engineer 52

Engineer Genetics Clinical Research Research 52

XTalks

NOVEMBER 7, 2022

Gleich, MD, FACS, Senior Vice President, Medical Department, and Dr. Christopher Huth, PhD, Senior Clinical Trial Manager, Clinical Trial Management. Liquid Biopsy Use in Oncology Clinical Trials. Also, exosomes are mostly being used for the detection of circulating RNA and microRNA. “An percent show less reliability).

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

Pharmaceutical Technology

JULY 5, 2022

It was also found in lab experiments that ZCCHC14, a protein that interacts with zinc, attaches to certain part of HAV’s ribonucleic acid (RNA), enabling the virus to recruit TENT4 from the human cell. In addition, the scientists later found that the HAV needs TENT4A/B for its replication.

Protein 130

Protein RNA Genome Genomics 130

Delveinsight

AUGUST 26, 2021

Shape’s RNA editing technologies can modify the RNA sequence, which makes the body’s protein building blocks. This system is designed to deliver RNA editing technology or other payloads directly to particular body areas, such as the nervous system or muscle. and Leila Zegna, director of the Kabuki Syndrome Foundation.

Vaccine 98

Vaccine Vaccination Gene Therapy RNA 98

Delveinsight

SEPTEMBER 23, 2021

858 Therapeutics closes USD 60 Million series A round to drug RNA modulation. Now, they are ready for their next venture, which is launching RNA biotech 858 Therapeutics with a USD 60 million Series A fundraising. have helped prepare more than 20 drug candidates for clinical trials. Jeffrey Stafford, Ph.D., Stafford and co.

RNA 61

RNA Gene Therapy Gene Protein 61

pharmaphorum

DECEMBER 1, 2020

The companies initially filed the nonclinical data and other technical data, followed by emerging clinical trial results. In the trial, BNT162b2 also showed an overall favourable tolerability with no safety concerns reported to date. The first primary objective analysis was based on 170 confirmed cases of COVID-19.

Vaccine 88

Vaccine Vaccination RNA Clinical Trials 88

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

XTalks

DECEMBER 1, 2021

The country of South Africa has a particularly sophisticated infectious disease monitoring, surveillance and research infrastructure, which is why the country has been at the forefront in both the identification of coronavirus variants as well as COVID-19 vaccine trials.

Gene 105

Gene Genome Genomics Genetics 105

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. Both trials concluded with maintained virologic suppression, and no clinically significant change from baseline in CD4+ cell counts was observed.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

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.

Genetics 111

Genetics Vaccine Vaccination DNA 111

pharmaphorum

MARCH 29, 2022

Less than six months after the late-stage failure of tofersen, Biogen and Ionis have suffered further disappointment in the amyotrophic lateral sclerosis (ALS) pipeline after BIIB078 (IONIS-C9Rx) failed to demonstrate clinical benefit in a Phase 1 trial.

Trials 49

Trials RNA Containment Genetics 49

The Pharma Data

AUGUST 16, 2021

The antiviral agent incorporates RNA-like building blocks into the RNA genome of the virus. If this genetic material is further replicated, defective RNA copies are produced and the pathogen can no longer spread. Molnupiravir is currently being tested in clinical trials.

RNA 52

RNA Drugs Genomics Genome 52

pharmaphorum

NOVEMBER 9, 2020

Top-line results come from a phase 3 trial of the candidate known as BNT162b2 and an evaluation of 94 cases from the trial of 43,538 patients. Results will be discussed with regulators worldwide and the European Medicines Agency has already begun its rolling review of data to hasten a decision on the clinical trial dossier.

Vaccine 98

Vaccine Vaccination Clinical Trials Clinical Trials 98

XTalks

AUGUST 30, 2022

Clinical research experts from the CRO Medpace shared insights about the operational and regulatory considerations for neuroscience trials with direct CNS administration. Such therapies may involve small interfering RNA (siRNA) or oligonucleotides in viral transfection platforms, large molecules and antibodies.

Trials 83

Trials Drug Delivery Drugs Gene 83

pharmaphorum

JANUARY 6, 2021

It’s likely no coincidence that Pfizer’s flagship project in 2020 while the design of the logo was being finalised was unquestionably its high-profile alliance with BioNTech on the RNA-based coronavirus vaccine Comirnaty, which provided further evidence of the potential of harnessing genetic toolkits to tackle health issues.

DNA 116

DNA Branding Vaccine Vaccination 116

XTalks

JANUARY 3, 2024

Amvuttra: Alnylam’s Similar Treatment on the Market In 2022, Alnylam, a US-based biopharma company with expertise in RNA interference therapeutics for genetically defined diseases, received FDA approval for its drug Amvuttra (vutrisiran) for the treatment of hATTR.

FDA Approval 105

FDA Approval Protein RNA Clinical Trials 105

pharmaphorum

FEBRUARY 3, 2021

While achieving the Nobel Prize spotlight would have been enough to impress, CRISPR-Cas9 gene editing is part of a growing list of technologies granted Investigational New Drug (IND) applications with early data from clinical trials supporting its safe use in edited cells re-introduced into a patient.

Gene Editing 98

Gene Editing Gene DNA Engineer 98

The Pharma Data

NOVEMBER 17, 2021

Mount Sinai researchers have developed a new model that uses DNA and RNA sequencing data from hundreds of patients to identify specific genes and genetic alterations responsible for never-before-defined subtypes of a blood cancer called multiple myeloma. doi: 10.1126/sciadv.abg9551. Source link: [link].

Scientist 52

Scientist RNA DNA Genetics 52

pharmaphorum

OCTOBER 6, 2020

In the case of a rolling review, the EMA evaluates trial data as they become available before a complete filing is submitted. The vaccine is based on different technology from AZ’s rival: while AZ’s uses a weakened and genetically modified cold virus to prime the immune system, BNT162b2 is based on a string of RNA.

Vaccine 59

Vaccine Vaccination RNA Immune Response 59

XTalks

JUNE 18, 2024

The company is currently testing its lead candidate, EXG-34217 — an autologous cell therapy in Phase I/II trials for treating telomere biology disorders (TBDs), conditions known to cause bone marrow failure with treatments mainly limited to complicated bone marrow transplants.

Protein 52

Protein RNA Genetics Gene Expression 52

pharmaphorum

OCTOBER 21, 2021

Givlaari (givosiran) has been recommended as a treatment option for people aged 12 and over with AHP, a rare life-threatening genetic condition that causes attacks of severe abdominal pain, vomiting and nervous system disorders, such as seizures, depression and anxiety.

Gene Silencing 98

Gene Silencing Gene RNA Drugs 98