GENPREX, INC. Over the years, gene therapy has become pretty significant. Research has shown that genes we are born with can be somehow be flawed and there are potentially more than 7,000 genetic diseases in the world today. These genetic diseases are shown to result in the loss of critical enzymes and proteins in our bodies. Unfortunately, the loss of normal proteins and enzymes can lead to disease conditions that range from levels of mild to deadly.
Although these types of diseases are considered pretty rare, one-in-ten people may experience a rare disease. The total cost of treatment is between
Full Company Report: ? HERE
It is one of the few plays trading at highly discounted levels while developing therapies and technologies for treating a range of disorders, such as inherited diseases and cancers. Rather than treating patients with drugs or surgeries,
With the Gene therapy market expected to grow to
Here are a few reasons why
TOP GNPX HIGHLIGHTS
* GNPX stock is trading near a 52-week low with RSI near 46 - A strong bounce can happen at anytime.
*
*
*
* With the Gene therapy market expected to grow to
Robust Gene Therapy Technologies
Nanoparticle Delivery System is the first systemic gene therapy delivery platform that can be used to enclose plasmids that deliver tumor suppressor genes. It also administers encapsulated plasmids which are taken up by tumor cells and express any missing proteins in the tumor cells.
In addition, the company has REQORSA, a leading immunogen therapy drug for non-small cell lung cancer. The therapy is administered through the company's systemic gene therapy platform for cancer.
Finally,
Lung
REQORSA is the company's lead candidate drug currently in clinical trials for the treatment of non-small cell lung cancer and small cell lung cancer. The novel treatment positions
After successful clinical trials, the candidate drug has already received fast-track designation from the
Multidisciplinary Management Team
The management team boasts of highly experienced personnel as part of its scientific advisory board that reviews research and development activities and provides recommendations to accelerate the development of the moving treatment. There is also a clinical advisory board tasked with overseeing clinical trials to ensure they are up to board or approval by the FDA.
While insiders own about 4.5% of GNPX, their interests are perfectly aligned with that of investors. Likewise, institutions owning about 7.8% of the stock underscore the strong belief in the company's gene therapy pipeline.
In the near term, the investment community could elevate the value of GNPX closer to that of its peers.
ANEW MEDICAL, INC. (OTC: LEAS) was established with the mission to create and deliver groundbreaking therapies that can effectively combat and potentially reverse the advancement of cancer and neurodegenerative conditions by utilizing cell and gene therapy. Their primary objective is to develop cutting-edge treatments for individuals diagnosed with cancer, Alzheimer's disease, Parkinson's disease, and amyotrophic lateral sclerosis (ALS) or commonly known as
ANEW MEDICAL, INC. has been awaiting approval from
On
The merger agreement values the combined company at an estimated pro forma enterprise value of approximately
Voyager's TRACER(TM) platform, known as Tropism Redirection of AAV by Cell-type-specific Expression of RNA, offers a versatile method for discovering AAV capsids. This RNA-based screening platform allows for quick identification of AAV capsids that effectively penetrate the blood-brain barrier and exhibit enhanced tropism in the central nervous system (CNS) across various species, including non-human primates (NHPs). Capsids generated through TRACER have demonstrated exceptional gene expression throughout the CNS, surpassing the capabilities of conventional AAV capsids. They also exhibit specific transduction in cells and tissues, including previously inaccessible areas of the brain. Furthermore, studies have shown that certain capsids possess improved targeting of cardiac muscle and reduced targeting of the dorsal root ganglia. Voyager is actively expanding its collection of AAV capsids optimized for delivering diverse therapeutic payloads to treat a wide range of CNS and other diseases. Through collaborative partnerships, Voyager aims to provide its advanced TRACER capsids to support gene therapy programs targeting various diseases.
"The results presented confirm the efficacy of our TRACER capsid discovery platform in consistently generating capsids capable of crossing the blood-brain barrier," remarked
TRACER AAV Capsid Discovery Platform
Through a process of directed evolution using an AAV9 library, a variant capsid with improved brain tropism and reduced liver targeting in non-human primates and mice following systemic administration was identified (Oral Presentation).
First unveiled at the ASGCT 2022 conference, the novel capsid, VCAP-102, derived from TRACER, has displayed tropism for the central nervous system in both mice and non-human primates (NHPs). A recent study conducted on marmosets demonstrated that intravenous administration of VCAP-102 resulted in 450-fold higher transgene expression compared to a conventional AAV9 benchmark capsid, achieving over 50% cell transduction in multiple brain regions at a dose of 2E12 vg/kg. Further evaluation involving intravenous administration of VCAP-102 in African green monkeys revealed transduction in various regions of the brain, including the frontal, motor, temporal, and cerebellar cortexes, putamen, caudate, and hippocampus. Compared to a conventional AAV9 benchmark capsid, these brain regions experienced 38-fold to 186-fold increases in transduction. Additionally, VCAP-102 demonstrated reduced targeting of the dorsal root ganglia (DRG) and liver. The capsid showed similar levels of transduction in both neurons and astrocytes. Iterative evolution of the VCAP-102 capsid led to the development of a second generation of capsids with further improvements in blood-brain barrier penetration and reduced liver targeting relative to VCAP-102, as observed in cynomolgus macaques.
disease with a single dose. They target the root cause of genetic disease by introducing a functional copy of the affected gene into patients' own hematopoietic stem cells (HSCs), with the goal of durably expressing the therapeutic protein throughout the body, including the central nervous system. Their first-in-class pipeline includes clinical programs for Gaucher disease and cystinosis, as well as preclinical programs for Hunter syndrome and Pompe disease.
On
Cystinosis is a rare and progressive disease that imposes a heavy treatment burden and lacks effective treatment options. The Phase 1/2 clinical trial, which is fully enrolled, is monitoring the long-term safety and efficacy of a gene therapy approach in six adult patients with the most severe form of cystinosis. These patients had previously received the standard treatment, cysteamine. In the trial, the patients' own hematopoietic stem cells (HSCs) were genetically modified to produce a functional version of the cystinosin protein, which is deficient in individuals with cystinosis. Promising preliminary data indicate that after gene therapy, functional cystinosin is being produced throughout the body, as demonstrated by clinical assessments in various tissues such as the eyes, skin, gastrointestinal mucosa, and through neurocognitive tests indicating activity in the central nervous system. Importantly, no adverse events related to the treatment have been reported so far.
In addition to the presented data,
Further data demonstrate that investigational HSC gene therapy has lasting systemic effects on neurocognitive measures, reducing cystine levels in the blood, as well as crystal accumulation in the skin and gastrointestinal mucosa.
Follow-up data indicate that patients who underwent HSC gene therapy can produce functional cystinosin protein throughout their bodies. As a result, cystine levels in the blood have decreased below baseline in all six patients and have remained stable for up to 36 months post-treatment. Skin and gastrointestinal mucosa biopsies of the first four patients, observed for at least 12 months with two patients monitored for up to 24 months, reveal a decrease in tissue cystine crystals below baseline.
Individuals with cystinosis typically experience a decline in visual spatial and visual motor function over time, as measured by standardized tests assessing the brain's ability to interpret and translate visual information into precise motor responses. However, the first four patients treated with gene therapy have shown either improvement or stabilization of scores on the Beery-Buktenica Developmental Test of visual motor integration, even up to 36 months post-treatment, indicating a potential impact on the disease's neuropathology.
This data confirms and extends previous findings, demonstrating the durability of the treatment effect for up to 36 months.
Disclaimer: All content provided by BigTimePennys Report is for informational purposes only. The information provided should not be considered as a substitute for professional advice, analysis, or guidance. Any investment decisions made based on the information provided are solely at the user's own risk. BigTimePennys Report are not liable for any losses or damages incurred as a result of using this information. BigTimePenny is not operated by a licensed broker, a dealer, or a registered investment adviser. The Securities Litigation Reform Act of 1995 provides safe harbor protection for forward-looking statements made by companies or individuals. It should be noted that any forward-looking statements made by BigTimePennys Report, its authors, are not guarantees of future performance or results. These statements are subject to risks, uncertainties, and assumptions that may cause actual results to differ materially from those expressed or implied in the forward-looking statements. The content provided by BigTimePennys Report does not constitute an endorsement, recommendation, or solicitation to buy, sell, or hold any securities or investment products. We have been retained by a third party to assist in the production and distribution of content on
CONTACT: CONTACT: Bigtimepennystocks.com
Editor: mail@bigtimepennystocks.com
.
(C) 2023 M2 COMMUNICATIONS, source
