Genetically modified herpes combats advanced cancers
Pharma Times
SEPTEMBER 21, 2022
A new genetically engineered virus has delivered a one-two punch in initial phase 1 trial
Engineer Genetics Trials 
Experiment Reveals Human Genes Can Be Controlled With Electricity
AuroBlog - Aurous Healthcare Clinical Trials blog
AUGUST 7, 2023
New research from ETH Zürich in Switzerland could see future wearable devices (with perhaps a few implants and a touch of genetic engineering) boost our health directly. Fitness trackers help you stay healthy by keeping count of your steps and monitoring your heart rate, driving you on to hit those cardio goals.
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CIGB-128 by Center for Genetic Engineering and Biotechnology for Brain Tumor: Likelihood of Approval
Pharmaceutical Technology
JUNE 10, 2023
CIGB-128 is under clinical development by Center for Genetic Engineering and Biotechnology and currently in Phase I for Brain Tumor. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. It is a combination of interferon alfa-2b with interferon gamma.
CIGB-128 by Center for Genetic Engineering and Biotechnology for Brain Tumor: Likelihood of Approval
Pharmaceutical Technology
JUNE 10, 2023
CIGB-128 is under clinical development by Center for Genetic Engineering and Biotechnology and currently in Phase I for Brain Tumor. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. It is a combination of interferon alfa-2b with interferon gamma.
Novavax’s Covid-19 vaccine gets Taiwan FDA EUA for use in adults
Pharmaceutical Technology
JUNE 24, 2022
The protein-based vaccine is engineered from the genetic sequence of the SARS-CoV-2 virus’ initial strain. The totality of preclinical, manufacturing and clinical trial findings that were submitted for analysis formed the basis for the EUA. The vaccine showed an encouraging safety and tolerability profile in both trials.
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Ginkgo buys StrideBio’s AAV discovery and engineering platform assets
Pharmaceutical Technology
APRIL 6, 2023
Ginkgo Bioworks has strengthened its end-to-end R&D capabilities in gene therapy with the purchase of adeno-associated virus (AAV) capsid discovery and engineering assets from StrideBio, for an undisclosed sum. AAV is used in hundreds of active clinical trials, and is described as the preferred viral vector for gene therapy.
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China NMPA grants approval for CanSinoBIO’s Covid-19 vaccine as booster
Pharmaceutical Technology
SEPTEMBER 5, 2022
In March last year, CanSinoBIO obtained approval for the clinical trial application to analyse Convidecia Air. Administered as a single dose, the genetically engineered vaccine has the replication-defective adenovirus type 5 vector that expresses the spike S protein of the SARS-CoV-2 virus.
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Synlogic’s SYNB1934 receives orphan drug status from US FDA
Pharmaceutical Technology
MAY 10, 2023
SYNB1934 consumes Phe in the gastrointestinal (GI) tract by leveraging genetic engineering of the drug or drug-carrying capsule, probiotic escherichia coli (E coli) Nissle. This designation also comes at a pivotal time as we prepare to initiate our Phase 3 trial for PKU – Synpheny-3 – in the first half of 2023.”
Scientists discover non-invasive approach for cancer therapy
Pharmaceutical Technology
APRIL 11, 2023
The finding was made as part of a study published in the journal Nature Biomedical Engineering. Kelley added: “Engineering-based tools allow you to do things that open up new areas of biology. CTRL Therapeutics will file an application with the US Food and Drug Administration (FDA) to advance the new platform into clinical trials.
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Capsida Biotherapeutics and Prevail to develop CNS gene therapies
Pharmaceutical Technology
JANUARY 5, 2023
Under the multi-year strategic partnership, Prevail will detect and advance capsids, which are clinically translatable, along with its cargo to develop the transformative genetic medicines by using Capsida’s novel adeno-associated virus (AAV) engineering platform.
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US FDA grants orphan drug status to IN8bio’s INB-400 and INB-410
Pharmaceutical Technology
APRIL 26, 2023
This marks the first-ever designation for genetically modified gamma-delta T cell therapies. INB-400, an autologous, genetically engineered gamma-delta T cell therapy, is the company’s DeltEx chemotherapy-resistant autologous and allogeneic drug-resistant immunotherapy (DRI) technology.
Tessera Cuts Dozens of Jobs Following Positive Preclinical Results
BioSpace
APRIL 25, 2024
The genetic engineering startup, recently honored by BioSpace readers for its work environment, is downsizing as it seeks to launch its first clinical trials.
Biogen’s ambitions in gene therapy hit by another failed pivotal trial
pharmaphorum
JUNE 15, 2021
Just over two years later, both of those candidates have failed late-stage clinical trials, leaving Biogen’s $800 million investment in Nightstar looking like a poor deal. The post Biogen’s ambitions in gene therapy hit by another failed pivotal trial appeared first on.
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Engrafting Bacteria for Therapeutic Advantage in Kidney-Related Diseases
BioSpace
DECEMBER 6, 2021
Proof of concept for the Genetically Engineered Microbial Medicines (GEMM) platform came in a recent first-in-human, Phase I trial.
ESMO: TIL therapy improves on Yervoy in melanoma trial
pharmaphorum
SEPTEMBER 11, 2022
It is the first time that a cell therapy for solid tumours has been tested in a phase 3 trial, and the first time that the approach has been directly compared with standard second-line immunotherapy in melanoma. ” The post ESMO: TIL therapy improves on Yervoy in melanoma trial appeared first on.
Trials 105
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Enabling the next wave of innovative drug therapies with speciality enzymes
Pharmaceutical Technology
JUNE 29, 2022
In September 2021, GlobalData figures revealed there to be 1,320 industry-sponsored regenerative medicine and advanced therapy trials ongoing worldwide. Novozymes has a long legacy of enzyme discovery and the ability to genetically engineer these speciality enzymes to be superior. And it’s within the family – Novozymes!”.
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Expanding ketamine’s horizons to treat rare neurological disorders
Pharmaceutical Technology
NOVEMBER 29, 2022
The company now plans to move this approach into a Phase III trial. PharmaTher also has an agreement in place with Case Western Reserve University to develop ketamine as a treatment for Rett syndrome, a rare genetic neurological disorder. PharmaTher is not the only player in this space.
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Ctexli (Chenodiol) Gains FDA Approval as First CTX Treatment
XTalks
FEBRUARY 25, 2025
CTX is a rare, progressive genetic disorder caused by mutations in the CYP27A1 gene, which disrupts the livers ability to produce chenodeoxycholic acid, a bile acid. The FDAs approval was supported by data from the Phase III RESTORE study a 24week, doubleblind, placebocontrolled, randomized crossover withdrawal trial.
FDA Approval 59
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GSK partners LifeMine on fungi-derived medicines
pharmaphorum
MARCH 24, 2022
LifeMine has developed an industrialised, genomics-based discovery engine that it hopes will take a broad, systematic approach to identifying new compounds from fungi and screening them for activity against disease targets. Greg Verdine – LifeMine.
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First launch for Daiichi Sankyo’s oncolytic virus Delytact in Japan
pharmaphorum
NOVEMBER 1, 2021
Delytact (teserpaturev) is a genetically engineered oncolytic herpes simplex virus type 1 (HSV-1) that was approved for marketing in Japan earlier this year, and received pricing approval in August at 1.43 million yen (around $12,500) per dose, according to a Pharma Japan report.
City of Hope researchers develop new brain cancer therapy
Pharmaceutical Technology
NOVEMBER 29, 2022
These viruses are engineered genetically for killing cancer. The researchers plan to seek the US Food and Drug Administration approval to commence clinical trials of the viral platform comprising chemokines and cetuximab, in humans. GBMs are called “cold” tumours as they lack helpful immune cells.
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QN-023a by Hangzhou Qihan Biotechnology for Relapsed Acute Myeloid Leukemia: Likelihood of Approval
Pharmaceutical Technology
MARCH 2, 2023
Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval. The therapeutic candidate comprises allogeneic NK cells genetically engineered to express chimeric antigen receptors (CAR-NK) targeting cells CD33.
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Diabetes Clinical Trials with Dr. Stayce Beck
Clinical Trial Podcast
FEBRUARY 3, 2024
To learn more about clinical trials in diabetes, I invited Dr. Stayce Beck, Global Vice President of Clinical Affairs at Dexcom Inc. in Chemical Engineering from the University of Texas at Austin. The post Diabetes Clinical Trials with Dr. Stayce Beck appeared first on Clinical Trial Podcast & Blog. on the podcast.
Generating Over a Billion Cells with CRISPR for Next Generation Cell Therapies
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. The goal of the team was to significantly increase yields of correctly engineered immune cells with CRISPR to effectively manufacture cell therapies. From the Bench to Patient Therapeutics.
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Twin Firsts for Tecelra — First Engineered Cell Therapy for Solid Tumors and First New Treatment in a Decade for Synovial Sarcoma
XTalks
AUGUST 8, 2024
Tecelra is the first cell therapy engineered for a solid tumor cancer in the US, offering a new option for patients with specific genetic markers who have previously undergone chemotherapy. Clinical trial results for the therapy are expected to be shared in late 2024. The trial showed a 43 percent response rate with 4.5
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Pharma Research Trends: Leveraging Innovation for Smarter Marketing Strategies
Pharma Marketing Network
NOVEMBER 23, 2024
By leveraging research into specific genetic markers and tailored therapies, campaigns can address niche markets with unparalleled relevance. The Role of Real-World Evidence Real-world evidence (RWE) bridges the gap between clinical trials and everyday use. Authentic, research-backed messaging builds trust and credibility.
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AstraZeneca-Oxford COVID-19 Vaccine Shows High Efficacy
XTalks
NOVEMBER 23, 2020
Severe cases of the infection did not occur among trial participants, nor were any hospitalizations reported. The global trials are assessing the safety and efficacy of the vaccine in individuals aged 18 years or over from diverse racial and geographic groups who are healthy or have stable underlying medical conditions.
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UCSF launches $250M initiative to develop living therapeutics
Scienmag
JUNE 14, 2021
Investments have catalyzed research, clinical trials and revolutionary treatments across many disciplines UC San Francisco is launching a new initiative to propel the development of living therapeutics – a category of treatments broadly defined as human and microbial living cells that are selected, modified, or engineered to treat or cure disease (..)
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NICE recommends first CAR-T cell therapy on NHS in England
pharmaphorum
JANUARY 26, 2023
Yescarta – a CD19-directed genetically modified autologous T-cell immunotherapy investigated in the ZUMA-1 trial – is the first chimeric antigen receptor (CAR) T-cell therapy recommended for routine use on the NHS in England , meaning that for the first time eligible patients will be able to access CAR-T cell therapy in the long-term.
Q&A: A decade on, what’s next for CAR-T therapies?
Pharmaceutical Technology
APRIL 5, 2023
And my estimate is that between 25,000 and 30,000 patients have been treated with those therapies globally, which does not include patients treated in clinical trials. We have the emergence of this new pillar of medicine, which is out of the lab and beyond clinical trials now. We must approach this on several fronts.
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Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug
XTalks
OCTOBER 20, 2023
Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.
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Blood test detects childhood tumors based on their epigenetic profiles
Scienmag
MAY 28, 2021
These “liquid biopsy” analyses exploit the unique epigenetic landscape of bone tumors and do not depend on any genetic alterations, […]. The scientists analyzed short fragments of tumor DNA that are circulating in the blood.
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Life Science Trends to Look Out for in 2024
XTalks
DECEMBER 20, 2023
This includes analyzing how drug combinations may impact individual patients or groups of patients before even entering a clinical trial. With a staggering 90 percent of drugs failing in clinical trials, AI has the potential to help improve these statistics. These companies are at various stages of research and clinical trials.
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How Centralized IBC Review Can Benefit Gene Therapy Research
Advarra
AUGUST 8, 2023
Gene therapy involves the transfer of engineered genetic materials to human research subjects. These studies were previously considered to be highly experimental and limited to early phase trials at a handful of highly specialized academic medical centers.
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Pfizer’s haemophilia B gene therapy meets phase 3 primary endpoint
pharmaphorum
JANUARY 3, 2023
Haemophilia is a rare genetic bleeding disorder that causes the blood to take a long time to clot because of a deficiency in one or several blood clotting factors. SPK-9001 is a novel, investigation vector containing a bio-engineered adeno-associated virus (AAV) capsid (protein shell) and a high-activity human coagulation FIX gene.
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Kymriah Approved for Additional Indication for Adults with Relapsed/Refractory Follicular Lymphoma
XTalks
JUNE 2, 2022
XTALKS WEBINAR: Next-Gen Cell Therapies: Listening and Engaging with Patient Perspectives as an Essential Element in Clinical Trials. CAR-T is a type of immunotherapy in which the patients’ own T-cells are engineered to target and remove cancer cells. Kymriah Clinical Trial Results. x 10 8 CAR-positive viable T.
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Filsuvez Topical Gel Gets FDA Approval for Epidermolysis Bullosa
XTalks
JANUARY 2, 2024
XTALKS WEBINAR: Strategic Design Decisions in Rare Disease Trials Live and On-Demand: Thursday, February 8, 2024, at 11 am EST (4 pm GMT/UK) Register for this free webinar to discover invaluable strategies to effectively navigate rare disease trials, enhancing endpoint adaptation, data quality and clinical interpretation.
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World Cancer Day 2024: Trends in Oncology Research and More
XTalks
JANUARY 26, 2024
By targeting and identifying specific genetic mutations, this technology could lead to the development of more precise diagnostic tools, enabling early intervention and treatment. By tailoring treatments based on an individual’s genetic makeup, it may allow for more effective and targeted therapies.
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Top 30 New Medical Devices of 2024
XTalks
JANUARY 21, 2025
From non-invasive cancer diagnostics to life-saving cardiovascular implants, the latest medical devices cleared or approved by the FDA in 2024 reflect the remarkable strides in science and engineering. There, plasma is separated, and tumor DNA is identified by detecting genetic mutations, methylation patterns and fragmentation signals.
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How Potential Changes to the NIH Guidelines Could Impact IBC Review
WCG Clinical
NOVEMBER 17, 2023
Since then, however, certain genetic engineering technologies (e.g., For example, two trials testing genetically identical genome-edited cell therapies – one engineered with a viral vector, and one without – can differ in their IBC review requirements because of how the cells were modified rather than what they have become.
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Synlogic preps phenylketonuria drug for phase 3 after mid-stage win
pharmaphorum
OCTOBER 19, 2022
PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down phenylalanine (phe), an amino acid that is commonly found in many foods. Left untreated, high levels of the amino acid become toxic to the brain and may lead to serious neurological and neuropsychological issues.
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Good Gut Bacteria Can Aid in Healing Intestines Damaged by IBD
XTalks
JULY 31, 2020
The study developed and researched microbe-controlled and genetically-engineered mice to understand the healing response in the intestine when receiving specific signals from bacteria. If we can influence these interactions, we may be able to control many diseases that are impacted by our microbiome or diet,” she says.
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How Advanced Diagnostics are Shaping the Future of Precision Medicine and Gene Therapy
Cloudbyz
JULY 31, 2024
These technologies provide insights into genetic, proteomic, and metabolomic profiles, enabling the discovery of biomarkers associated with diseases like cancer, cardiovascular diseases, and neurological disorders. For instance, in oncology, patients with specific genetic mutations (e.g.,
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Novavax Announces COVID-19 Vaccine Clinical Development Progress
The Pharma Data
NOVEMBER 30, 2020
CoV2373 is a stable, prefusion protein antigen derived from the genetic sequence of the SARS-CoV-2 coronavirus spike (S) protein and adjuvanted with Novavax’ proprietary Matrix?M. pivotal Phase 3 trial update. Novavax completed enrollment of 15,000 participants in a pivotal Phase 3 clinical trial being conducted in the U.K.
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