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

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

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

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.

Vaccination 98

Vaccination Vaccine Gene Therapy RNA 98

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.

Vaccination 88

Vaccination Vaccine 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

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

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

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

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.

Vaccination 59

Vaccination Vaccine RNA Immune Response 59

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

XTalks

AUGUST 29, 2022

CRISPR technology has begun to enter clinical trials due to emerging therapeutic applications, but the technology still has limitations, primarily because it is difficult to safely make large quantities of precisely edited cells. The results demonstrate potential as a novel, safe and effective approach to cell therapies.

Gene Editing 98

Gene Editing DNA Gene Genome 98

The Pharma Data

SEPTEMBER 28, 2020

The large-scale project, undertaken in collaboration with researchers and clinicians from Fred Hutchinson Cancer Research Center (Fred Hutch) and the Target Padiatric AML (TpAML) computational working group aims to enable paediatric oncology physicians and researchers to better analyse patient clinical trial and genetic data.

Genome 52

Genome Genomics Clinical Trials Clinical Trials 52

Delveinsight

JANUARY 7, 2021

The immunotherapy pipeline is led by an in-licensed EP4 antagonist that Ikena is testing combined with Keytruda in phase 1b/2 colorectal and non-small cell lung cancer clinical trials. Eli Lilly backed Terns Pharmaceuticals now has two NASH candidates in clinical trials. IND-enabling studies are underway.

RNA 52

RNA Clinical Trials Clinical Trials Bioavailability 52

pharmaphorum

NOVEMBER 18, 2021

The two companies have been working together since 2019 to develop multiple RNA interference (RNAi) candidates for disorders such as non-alcoholic steatohepatitis (NASH), type 2 diabetes, obesity and rare diseases. billion in cash. The transaction is expected to close in the fourth quarter of this year.

Gene Silencing 52

Gene Silencing RNA Protein Gene 52

XTalks

JANUARY 3, 2025

FCS is a rare genetic disorder that prevents the body from properly breaking down triglycerides (a type of fat in the blood), leading to dangerously high levels. The approval of Tryngolza was based on positive results from the Phase III Balance trial, which showed reductions in triglyceride levels and acute pancreatitis events.

FDA Approval 59

FDA Approval Drugs Clinical Trials Clinical Trials 59

XTalks

APRIL 6, 2022

XTALKS WEBINAR: Using CRISPR interference (CRISPRi) Viability Screens to Map Long Noncoding RNA Dependencies in Tumor Cells. Register for this webinar to get an overview of the role of long non-protein coding RNAs (lncRNAs) in gene regulations. Live and On-Demand: Thursday, April 21, 2022, at 11am EDT (4pm BST/UK).

DNA 105

DNA In-Vitro Doctors Doctor 105

The Pharma Data

JANUARY 13, 2021

13, 2021 — A single-shot COVID-19 vaccine from Johnson & Johnson has shown very strong results in early clinical trials, potentially providing a significant boost to U.S. The vaccine produced an immune response of all 805 clinical trial participants within two months of inoculation, according to results published Jan.

Vaccination 40

Vaccination Vaccine Trials Immune Response 40

pharmaphorum

JUNE 1, 2022

The US biotech has just reported phase 2 trial results with the small, interfering RNA (siRNA) gene-silencing drug showing that it an cause a 90% or greater reduction in Lp(a) levels – a risk factor for cardiovascular disease – that was sustained over 48 weeks of follow-up.

Gene Silencing 59

Gene Silencing Drugs Medicine RNA 59

pharmaphorum

NOVEMBER 7, 2022

Final results of the OCEAN(a)-DOSE trial – published in the New England Journal of Medicine – showed that the small, interfering RNA (siRNA) drug reduced Lp(a) levels compared to placebo at all four doses tested, with doses above 75 mg given every 12 weeks cutting levels by 95% or more after six months.

Drugs 59

Drugs RNA Medicine Genetics 59

pharmaphorum

JULY 28, 2022

The RNA interference specialist says its researchers and collaborators have identified mutations in a gene called INHBE that are associated with protection against abdominal obesity and metabolic syndrome – a condition impacting more than 20% of adults worldwide.

Biobanking 52

Biobanking Gene Gene Silencing Drugs 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

The Pharma Data

MAY 31, 2022

Olpasiran is a small interfering RNA designed to lower the body’s production of apolipoprotein(a), a key component of Lp(a) that has been associated with an increased risk of cardiovascular events. Lp(a) is genetically determined 1,2 and reported to be an independent risk factor for cardiovascular disease (CVD). About Lp(a).

Genetics 52

Genetics RNA Medicine Development 52

The Pharma Data

OCTOBER 19, 2020

With over 300 active clinical trials, Winship researchers are dedicated to taking new cancer treatments from bench to bedside. cancer centers selected as a Lead Academic Participating Site for the NCI’s National Clinical Trials Network (NCTN). Winship is one of 32 U.S. MBA, FACP, Chairman of the Caris Precision Oncology Alliance.

Life Science 52

Life Science Clinical Trials Clinical Trials RNA 52

pharmaphorum

NOVEMBER 23, 2021

The bolt-on deal comes – which includes an upfront payment of $120 million and up to $910 million in milestones – covers rights to ARO-HSD, Arrowhead’s RNA interference drug targeting an enzyme called HSD17B13 that seems to contribute to the pathology of NASH and other liver diseases.

Sales 52

Sales Drugs Trials RNA 52

Delveinsight

JANUARY 14, 2021

The companies schedule to initiate a Phase 2 trial assessing combination therapy for both treatment-experienced and treatment-naïve people living with HBV. People in the trial with HBV treatment experience may also receive Gilead’s Vemlidy (tenofovir alafenamide fumarate, TAF). Gilead Sciences, Inc. and Vir Biotechnology, Inc.

Gene Editing 40

Gene Editing Gene Genetics DNA 40

The Pharma Data

MARCH 28, 2022

“C9orf72-associated ALS is a complex genetic form of ALS and there are multiple mechanisms by which the scientific community believes the C9orf72 gene causes disease. This Phase 1 study was a randomized, placebo-controlled, dose-escalating trial to evaluate BIIB078 administered intrathecally to adults (n=106) with C9orf72-associated ALS.

Drugs 52

Drugs Genetics RNA Development 52

The Pharma Data

DECEMBER 8, 2020

Silverback’s lead candidate is currently in a Phase I trial in adults with HER2-expressing solid tumors. The assumption is SciNeuro will start with Parkinson’s disease and pain in addition to rare genetic diseases, after scaling up staffing. . Laying down a new track for RNA processing, Remix launched with $81 million in financing.

RNA 52

RNA Antibody Life Science Protein 52

The Pharma Data

NOVEMBER 29, 2020

The submission was based on the safety and efficacy data of the pivotal Phase II trials 201 and 12-230. It is an RNA interference (RNAi) therapeutic. The ILLUMINATE-A trial evaluated 39 patients who were randomized 2:1 to receive three monthly doses of Oxlumo or placebo followed by a quarterly dosing schedule.

Genetic Disease 52

Genetic Disease Antibody Licensing Licensing 52

The Pharma Data

JANUARY 17, 2021

n-Lorem selects patients based on their genetic mutation and the organ affected rather than their specific diseases. “We That’s not the case with RNA-targeted drugs. They’re basically the same,” but each one uses a particular nucleotide to bind to specific genetic sequence.

Genetics 52

Genetics Clinical Trials Clinical Trials Drugs 52

Roots Analysis

OCTOBER 13, 2023

Unlike, a small molecule or a biologic based drug, which works on the mechanism of cytotoxicity induction at the site of action, these conjugates insert their payloads directly inside the genetic material of the host cell. This results in altering the genetic makeup of the host cell, eliminating the root cause of the disease.

Drugs 40

Drugs Antibody RNA Genetics 40

The Pharma Data

DECEMBER 8, 2020

chief executive officer at Ionis, said, “In 2020, we pursued an aggressive agenda focused on building our commercial plans and capabilities, progressing the Ionis-owned pipeline, advancing our technology and growing our leadership position in RNA-targeted therapeutics. Monia , Ph.D., It is available here for a limited time.

Medicine 52

Medicine RNA Production Marketing 52

Bioengineer

JULY 23, 2021

“By applying high-resolution imaging, advanced cell and molecular readouts and RNA sequencing methods leveraging the OTME-Chip, we discovered the actual genetic signaling pathways behind the blood cell triggered metastasis of ovarian cancer and a new drug strategy to stop this process.” ” ###.

Engineer 52

Engineer Gene Editing Clinical Trials Clinical Trials 52