Daily Archives: June 18, 2021

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Pivoting from conflicting andcontroversial pressover its recently approved Alzheimers disease treatment,Biogenhas announced yesterday that its gene therapy drug timrepigene emparvovec has failed to show a clinically meaningful benefit for a rare inherited eye disease in a Phase III trial.

The late-stage STAR study enrolled 169 adult males with choroideremia, an inherited retinal disease characterized by progressive vision loss and ultimate blindness. The investigators evaluated the efficacy and safety of a single subretinal injection of the investigational gene therapy timrepigene emparvovec. This drug was designed to deliver a functional human choroideremia gene into photoreceptor cells and the retinal pigment epithelium to address the conditions underlying genetic causative mechanisms.

In astatement on the STAR findings, Biogen stated the study failed to meet the primary endpoint of the proportion of patients who experienced a 15-letter improvement in the best corrected visual acuity (BCVA) at one year in the intervention arm versus the control group. The primary endpoint was assessed using the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart.

Also, Biogen reported that the trial failed to show efficacy regarding the key secondary endpoints. However, the safety findings from the study were generally consistent with previous research trials.

We extend our deepest gratitude to all those who contributed to the STAR study, including the participants, investigators, site staff and the broader choroideremia community, said Katherine Dawson, M.D., Biogens Head of the Therapeutics Development Unit.

While we are disappointed by the results of the STAR study, we are hopeful that the clinical insights gleaned from this study may help to shape therapeutic innovation for inherited retinal diseases including choroideremia, so that in the future there may be treatment options for the community affected by these debilitating disorders.

The company noted it would continue to assess the STAR studys complete data set before confirming its future plans for clinical development of timrepigene emparvovec.

These topline results from the STAR trial follow a controversial approval of Biogens Alzheimers drug Aduhelm (aducanumab). Asreported by the companyon June 7, the U.S. Food and Drug Administration (FDA) granted accelerated approval to the therapy to address the accumulation of amyloid beta plaques in the brain associated with the neurologic disease.

The approval was based on clinical trial data showing the therapy could reduce these plaques, but these studies did not demonstrate an overall clinically meaningful reduction in cognitive decline. Instead, the plaques serve as a biomarker for cognitive dysfunction in patients with Alzheimers disease, and both Biogen and some regulators at the FDA surmise the reduction in these plaques could possibly provide a beneficial effect.

But as reported in opinion pieces in theNew York TimesandBloomberg, amongother publications, the approval of the dementia drug is based on spotty evidence, which serves to threaten the FDAs reputation.

As such, three-panel members of the FDAs Peripheral and Central Nervous System Drugs Advisory Committee have resigned from their posts in protest over the Aduhelm approval.

My rationale was that the FDA needs to re-evaluate how it solicits and uses the advisory committees because I didnt think that the firm recommendations from the committee in this case were appropriately integrated into the decision-making process, said one of the resigning members, Aaron Kesselheim, a professor of Medicine at Harvard Medical School, in aninterview withReuters.

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After Controversial AD Drug Win, Biogen's Retinal Gene Therapy Flops - BioSpace

Written by AZoNanoJun 15 2021

The rare lung disease is so complicated that its acronym is difficult to pronounce. However, for infants who were unfortunate enough to be born with this disorder, the outcome is generally fatal.

Thedisease is known as alveolar capillary dysplasia with misalignment of the pulmonary veins, or ACDMPV for short. According to a study, the disease is associated with mutations in the FOXF1 gene. Medical experts throughout the world have recorded around 200 cases, but an unestimated number of infants could have died without the disorder ever being diagnosed, stated the National Organization for Rare Disorders.

The disease is induced by genetic changes that inhibit the formation of proper blood vessels in the lungs. Within a few days or weeks post birth, infants turn blue because of the absence of oxygen while blood pressure increases inside their lungs. The few infants who get to live do so by receiving exceptionally rare infant-sized lung transplants.

Now, a new study headed by experts from Cincinnati Childrens and the University of Cincinnati has reported about helping mice (with a FOXF1 mutation that is identical to human ACDMPV patients) live longer with this fatal disease. This was done by applying high-tech nanoparticles to send a STAT3 gene into the lungs to trigger the growth of blood vessels.

STAT3 is a crucial downstream target of the FOXF1 gene and its delivery can rectify the vascular deficiency in ACDMPV mice. The study results were published online in the Circulation journal on June 11th, 2021.

If these findings can be matched in human analyses in the future, this success can potentially boost the speed of development for other nanoparticle-based treatments for many medical conditions, stated the study co-authors.

Nanoparticle carriers have shown minimal toxicity and have accelerated the development of novel therapies for human cancers, diabetes, and chronic inflammatory disorders. We have developed a unique nanoparticle delivery system that can deliver genes capable of stimulating micro-vessel growth in the newborn lung.

Vlad Kalinichenko, MD, PhD, Study Senior Author and Member of the Center for Lung Regenerative Medicine and Perinatal Institute, Cincinnati Childrens Hospital Medical Center

Kalinichenko added, This study shows that a single injection of the nanoparticles with the STAT3 gene vector was sufficient to increase alveolar-capillary density, prevent excessively high blood pressuresand dramatically improve survival.

Around 70% of mice born with ACDMPV die in less than 28 days of birth without treatment. The new therapy reduced this mortality rate to 35%, stated Fei Sun, PhD, the first author of the study and a member of the Center for Lung Regenerative Medicine at Cincinnati Childrens.

This nanoparticle method is different from gene replacement therapies that can cause permanent changes in the body. It involves materials that do not remain in the body for more than seven days. Yet, in the mice examined so far, a single therapy early after the birth was sufficient to divert a whole stream of later-developing issues that take place with ACDMPV.

The treatment works by sending an engineered nanoparticle composed of many fatty acids, polymersand a small amount of cholesterol that transports the non-integrating STAT3 gene, which consequently encourages the growth of blood vessels in the lung tissue.

Kalinichenko and collaborators also noted the molecular processes involved as part of their ongoing analyses of lung development. The nanoparticle was designed with support from Zicheng Deng and Andrew Dunn, who are both graduate students mentored by Donglu Shi, PhD, from the Materials Science and Engineering Program at the University of Cincinnati.

With the presence of more blood vessels, the rapidly growing lungs in the newborns developed in a closer-to-normal fashion, without triggering harmful molecular remodeling signals that can lead to permanent malformations and even death from lung failure.

The study explains how the therapy enhanced various measures of heart, lungand blood vessel health, such as the ratio of pulmonary acceleration time to pulmonary ejection time (PAT/PET), blood pressure in the right ventricle, arterial oxygenation levels, the diameter of pulmonary arteriesand also the thickness of their walls.

More studies need to be completed before nanoparticles can be tested in human newborns with ACDMPV, such as safety tests and establishing whether recurrent treatments would be required.

Sun, F., et al. (2021) Nanoparticle Delivery of STAT3 Alleviates Pulmonary Hypertension in a Mouse Model of Alveolar Capillary Dysplasia. Circulation. doi.org/10.1161/CIRCULATIONAHA.121.053980.

Source: https://www.cincinnatichildrens.org/

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Nanoparticle-Based Therapy Could Treat Fatal Lung Disease in Newborns - AZoNano

DUBLIN--(BUSINESS WIRE)--The "Regenerative Medicine Market - Global Outlook and Forecast 2021-2026" report has been added to ResearchAndMarkets.com's offering.

The regenerative medicine market size to grow at a CAGR of around 34% during the period 2020-2026.

Increased R&D investments by pharmaceutical companies will drive the demand for regenerative medicines. Europe plays a significant role in supporting the development and authorization of these products for several genetic and rare disorders. Increased funding via several venture capitalists and governments, and private institutions contribute significantly to the global regenerative medicine market growth.

The increased prevalence of diseases such as cardiovascular diseases and diabetes can drive cell and gene therapy and tissue-engineered products. With the rise in thermal burns, occupational burn accidents, and chronic wounds, regenerative medicine products will experience steady growth. Novartis and Gilead Sciences are the key companies offering various therapies to treat cancer, genetic, and rare disorders.

The report considers the present scenario of the regenerative medicine market and its market dynamics for 2019-2026. It covers a detailed overview of several market growth enablers, restraints, and trends. The study covers both the demand and supply sides of the market. It also profiles and analyzes leading companies and several other prominent companies operating in the market.

REGENERATIVE MEDICINE MARKET SEGMENTATION

The regenerative medicine market research report includes a detailed segmentation by application, products, end-users, geography. Oncology constitutes the largest portion of the global regenerative medicine market share. The development of curative therapies by CAR-T and cell and gene therapies is widely popularized in the oncology therapeutic area. The increasing global prevalence rates and the increasing rates of different types of life-threatening cancers are the most important key factors that drive the oncology segment.

Consistent innovations in gene therapies due to the increased number of clinical trials and pipeline products are driving the growth prospects. Hence, the increased inflow of funding for the development of gene therapy is one of the driving factors for the sector growth. Cell therapy is the major revenue contributor. The increasing prevalence of diabetes and foot ulcers is the primary factor contributing to the growth of tissue-engineered products. The tissue-engineered product segment to grow at a CAGR of 8% by 2026.

Hospitals are likely to remain a dominant revenue contributor to the global regenerative medicine market. Around 50% of therapeutic surgeries performed in the US annually, including cardiovascular and musculoskeletal, occur in hospitals. Cancer care centers are likely to witness an incremental growth of approx. USD 10 billion by 2026.

As cancer is the second leading cause of death across the globe, which is responsible for approx. 10 million deaths annually, the scope of cancer centers is growing. Key vendors are focusing more on cancer care centers than hospitals to promote their products. As the cancer centers are being covered under reimbursement schemes, the growth of these facilities is likely to increase during the forecast period.

KEY QUESTIONS ANSWERED:

1. How big is the regenerative medicine market?

2. What are the critical applications of regenerative medicine products?

3. Who are the key players in the regenerative medicine market?

4. Which segment accounted for the largest regenerative medicine market share?

5. Which region holds the largest share in the global regenerative medicine market?

6. How has the COVID-19 pandemic affected the regenerative medicine industry?

Key Vendors

Other Prominent Vendors

Key Topics Covered:

1 Research Methodology

2 Research Objectives

3 Research Process

4 Scope & Coverage

5 Report Assumptions & Caveats

6 Market at a Glance

7 Introduction

8 Market Opportunities & Trends

8.1 Latest Advances In Tissue-Engineering Therapies

8.2 Robust Product Pipeline Of Regenerative Medicine Companies

8.3 Strategic Acquisitions By Regenerative Medicine Companies

9 Market Growth Enablers

9.1 Increase In Acute, Chronic, & Genetic Disorders

9.2 Increasing Demand For Car T-Cell Therapies

9.3 Faster Regulatory Approvals & Special Designations Of Rm Products

10 Market Restraints

10.1 Manufacturing, Operational, & Ethical Challenges

10.2 High Cost of Regenerative Therapies

10.3 Outbreak of The COVID-19 Pandemic

11 Market Landscape

11.1 Market Overview

11.2 Market Size & Forecast

11.3 Five Forces Analysis

12 Application

12.1 Market Snapshot & Growth Engine

12.2 Market Overview

12.3 Dermatology

12.4 Musculoskeletal

12.5 Oncology

12.6 Genetic Disorders

13 Product

13.1 Market Snapshot & Growth Engine

13.2 Market Overview

13.3 Gene Therapy

13.4 Cell Therapy

13.5 Tissue-Engineering

14 End-Users

14.1 Market Snapshot & Growth Engine

14.2 Market Overview

14.3 Hospitals

14.4 Cancer Care Centers

14.5 Wound Care Centers

14.6 Ambulatory Surgical Centers

For more information about this report visit https://www.researchandmarkets.com/r/cfc68n

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Regenerative Medicine Market - Global Outlook and Forecast 2021-2026 - Increasing Demand For Car T-Cell Therapies - ResearchAndMarkets.com - Business...

June 16, 2021

The Discovery Labs of King of Prussia now boasts another major research and development organization within its growing "Cellicon Valley." The innovation hub announced a multi-year lease with the University of Pennsylvania's Gene Therapy Program, a cutting-edge research and development organization, last month. It will focus its endeavors in Montgomery County on the development of medicine for rare and orphan diseases and treatments for contagious pathogens such as COVID-19.

Currently located in the University City neighborhood of Philadelphia, the Gene Therapy Program has outgrown its space at Penn's Translational Research Center. It's located in an area where space is at a premium. Rather than pay University City real estate's steep priceswhich have remained resilient amid the pandemicor commission new construction, Director James M. Wilson, M.D., Ph.D., has decided to expand outward. The space at the Discovery Lab, formerly occupied by pharmaceutical giant GSK, provides the necessary infrastructure and size to house an expanding and technologically advanced research and development (R&D) program,

"The past few years have been a pivotal time in the development of gene therapies, and our new space at Discovery Labs will propel us even further in charting the future of the field," Wilson said. "With additional state-of-the-art equipment and increased operational capabilities, we are well-positioned to enhance the design and execution of our innovative, transformative therapies."

Largely driven by the lack of research and drug development dedicated to patients with rare, inherited diseases, the Gene Therapy Program has been at the forefront of research into Adeno-Associated Virus (AAV) Vectors. AAVs are largely benign viruses that have proven modifiable by gene therapy. By reconstructing or repairing the genetic material within the viruses, scientists have been able to produce therapeutic effects in patients with rare, genetic diseases. Wilson and others at the Gene Therapy Program have also expanded their research to include similar approaches to pathogenic viruses such as COVID-19.

Moving to the Discovery Labs enhances the program's ability to conduct such advanced research. The new space includes two full floors for the Penn Vector Core, a major technological resource for investigators and therapy designers that advances the understanding of gene function, allowing for the development of new vector medicines. The location also provides ample space for every stage of drug R&D, from discovery research all the way through applications for clinical trials. All told, the multi-year lease includes over 150,000 square feet of lab space and two buildings on the Discovery Lab's campus.

With innovative R&D organizations such as Penn's Gene Therapy Program flocking to the Discovery Labs, King of Prussia will find itself in an enviable position post-pandemic. Penn joins Thomas Jefferson University Health, CHOP, the Centers for Breakthrough Medicine, GSK, and numerous other major healthcare companies and institutions at the Discovery Labs, positioning the company as the host for some of the most innovative and groundbreaking medical research in the United States.

Led by founder and chairman Brian O'Neill, the Discovery Labs have sought to leverage its more than one million square feet of lab space in conjunction with the amenities of their immediate location and proximity to Philadelphia's "Cellicon Valley," to attract some of the largest and most impactful healthcare companies and organizations to King of Prussia. So far, it appears to be working.

"Dr. Wilson and his incredible team at Penn have helped establish Philadelphia as a leader in gene therapies, and we are deeply honored to have the opportunity to work with him and his incredible team of scientists through the expansion of their world-class research and development labs," O'Neill said. "To welcome Penn's Gene Therapy Program as the anchor tenant for our planned life sciences cluster here in King of Prussia is strong validation for the incredible life science ecosystem that is emerging in Greater Philadelphia."

It seems as if, for the foreseeable future, King of Prussia will stand as one of the brighter extensions of the Delaware Valley's expanding "Cellicon Valley," where medical innovation, scientific research, and treatment development are in the midst of an ongoing renaissance. The advent and growth of the Discovery Labs underscore the increased demand for top-flight R&D real estate, and offers promising returns for both the area and the greater medical community through its latest partnership with the University of Pennsylvania.

The Delaware Valley Journal provides unbiased, local reporting for the Philadelphia suburbs of Bucks, Chester, Delaware and Montgomery Counties. For more stories from the Delaware Valley Journal, visit DelawareValleyJournal.com

Link:
Penn's Gene Therapy Program Moves To Innovation Hub In King Of Prussia - Patch.com

LAS VEGAS, June 17, 2021 (GLOBE NEWSWIRE) -- Zhittya Genesis Medicine, Inc. (a private company) (Zhittya or the Company), is proud to announce that Mr. Daniel C. Montano, CEO, and founder of Zhittya Genesis Medicine Inc. has received the award of Biotech Entrepreneur of the Year from the Nevada Biotechnology & Health Science Consortium. We also congratulate Dr. Marc Kahn, Dean of the Kirk Kerkorian School of Medicine at the University of Nevada, Las Vegas (UNLV), for receiving the Medical Professional of the Year Award from the same institution.

The Nevada Biotechnology & Health Science Consortium (NevBio) was founded in 2007 to encourage the development and expansion of the Health Sciences and Biotechnology in Southern Nevada. NevBio is part of the national biotechnology association, the Biotechnology Innovation Organization (BIO) and the Council of Bioscience Associations. Thousands of people have attended the monthly presentations sponsored by NevBio over the last 14 years on topics such as immunotherapy, nuclear medicine, gene editing, sepsis, cancer, C-Diff, pain, biofilm, angiogenesis, clinical trials, patents, raising capital, and much more. NevBio has given legislative advice both in Carson City & Washington DC, on assisting the development of health sciences in Southern Nevada. Senator Harry Reid and the late Nevada Chancellor Jim Rogers have spoken at the award dinners.

Daniel C. Montano, CEO of Zhittya Genesis Medicine Inc., is a biotech pioneer leading the creation of a revolutionary biopharmaceutical treatment termed Therapeutic Angiogenesis and is the recipient of the Biotech Entrepreneur of the Year Award. Daniel Montano moved to Las Vegas in 2002 from California to advance biotechnology here. Therapeutic Angiogenesis has demonstrated in US FDA clinical trials that it can trigger the growth of new blood vessels, thereby treating heart disease, the number one cause of death in the world. In animal experiments, monkeys given experimental Parkinsons disease, then treated with Therapeutic Angiogenesis, reversed their disease progression. Heart disease, strokes, peripheral artery disease, Parkinsons disease, multiple sclerosis and Alzheimers disease are all diseases he is applying Therapeutic Angiogenesis to treat. If successful, Las Vegas could become the biotech center of the world for Regenerative Medicine through Therapeutic Angiogenesis.

Dr. Marc Kahn will be awarded the Medical Professional of the Year, as the Dean of the new Kirk Kerkorian School of Medicine. Dr. Kahns experience is in both medicine and teaching medical professionals the business of medicine. His introduction of a joint MD-MBA program was very well received. His leadership of the School of Medicine is critical to the long-term success of making Las Vegas a biomedical center.

The Las Vegas Valley is developing into a biomedical center, with three medical schools and biotech companies that have either started here or have moved here from California. Biotech and health science companies are moving to Las Vegas to take advantage of its low costs, world-class airport, and hospitality facilities. The Las Vegas Valleys population has grown to almost 2.8 million people and is still growing. The Awards Dinner will be on Thursday, July 15th at 6 pm at the Ahern Hotel in Las Vegas. Register at nevbio.org.

The Awards Dinner will be:

Contact Information: Daniel Montano, CEO Zhittya Genesis Medicine, Inc. Phone: (1) 702-790-9980 E-mail: dan@zhittyamedicine.com Website: zgm.care

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Mr. Daniel C. Montano, CEO, is recognized as Biotech Entrepreneur of the Year and Dr. Marc - GlobeNewswire

DALLAS--(BUSINESS WIRE)--Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced its participation in virtual fireside chats at the LifeSci Genetics Medicine Conference and LSX Biotech Growth CEO Forum.

Conferences Details:

Event:

LifeSci Genetics Medicine Conference

Date:

Tuesday, June 22, 2021

Time:

10:00 am ET

Format:

Fireside chat

Participants:

RA Session II, President, Founder and CEO

Dr. Suyash Prasad, Chief Medical Officer and Head of R&D

Kamran Alam, Chief Financial Officer

Event:

LSX Biotech Growth CEO Forum

Topic:

Too Much of a Good Thing Can be Wonderful: Optimizing Value From Broad Discovery Platforms Through Strategy and Strategic Partnering

Date:

Tuesday, June 22, 2021

Time:

1:10 pm ET

Format:

Fireside chat

Participants:

RA Session II, President, Founder and CEO

Webcasts for these conferences will be available in the Events & Media section of the Taysha corporate website at https://ir.tayshagtx.com/news-events/events-presentations. Archived versions of the webcasts will be available on the website for 60 days.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

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Taysha Gene Therapies to Participate in Upcoming Investor Healthcare Conference and CEO Forum - Business Wire

Scientists led by UNC School of Medicine researchers Silvia Kreda, PhD, and Rudolph Juliano, PhD, created an improved oligonucleotide therapy strategy with the potential for treating other pulmonary diseases, such as COPD and asthma.

CHAPEL HILL, NC UNC School of Medicine scientists led a collaboration of researchers to demonstrate a potentially powerful new strategy for treating cystic fibrosis (CF) and potentially a wide range of other diseases. It involves small, nucleic acid molecules called oligonucleotides that can correct some of the gene defects that underlie CF but are not addressed by existing modulator therapies. The researchers used a new delivery method that overcomes traditional obstacles of getting oligonucleotides into lung cells.

As the scientists reported in the journal Nucleic Acids Research, they demonstrated the striking effectiveness of their approach in cells derived from a CF patient and in mice.

With our oligonucleotide delivery platform, we were able to restore the activity of the protein that does not work normally in CF, and we saw a prolonged effect with just one modest dose, so were really excited about the potential of this strategy, said study senior author Silvia Kreda, PhD, an associate professor in the UNC Department of Medicine and the UNC Department Biochemistry & Biophysics, and a member of the Marsico Lung Institute at the UNC School of Medicine.

Kreda and her lab collaborated on the study with a team headed by Rudolph Juliano, PhD, Boshamer Distinguished Professor Emeritus in the UNC Department of Pharmacology, and co-founder and Chief Scientific Officer of the biotech startup Initos Pharmaceuticals.

About 30,000 people in the United States have CF, an inherited disorder in which gene mutations cause the functional absence of an important protein called CFTR. Absent CFTR, the mucus lining the lungs and upper airways becomes dehydrated and highly susceptible to bacterial infections, which occur frequently and lead to progressive lung damage.

Treatments for CF now include CFTR modulator drugs, which effectively restore partial CFTR function in many cases. However, CFTR modulators cannot help roughly ten percent of CF patients, often because the underlying gene defect is of the type known as a splicing defect.

CF and splicing defects

Splicing is a process that occurs when genes are copied out or transcribed into temporary strands of RNA. A complex of enzymes and other molecules then chops up the RNA strand and re-assembles them, typically after deleting certain unwanted segments. Splicing occurs for most human genes, and cells can re-assemble the RNA segments in different ways so different versions of a protein can be made from a single gene. However, defects in splicing can lead to many diseases including CF when CFTRs gene transcript is mis-spliced.

In principle, properly designed oligonucleotides can correct some kinds of splicing defects. In recent years the U.S. Food and Drug Administration has approved two splice switching oligonucleotide therapies for inherited muscular diseases.

In practice, though, getting oligonucleotides into cells, and to the locations within cells where they can correct RNA splicing defects, has been extremely challenging for some organs.

It has been especially difficult to get significant concentrations of oligonucleotides into the lungs to target pulmonary diseases, Kreda said.

Therapeutic oligonucleotides, when injected into the blood, have to run a long gauntlet of biological systems that are designed to keep the body safe from viruses and other unwanted molecules. Even when oligonucleotides get into cells, the most usually are trapped within vesicles called endosomes, and are sent back outside the cell or degraded by enzymes before they can ever do their work.

A new delivery strategy

The strategy developed by Kreda, Juliano, and their colleagues overcomes these obstacles by adding two new features to splice switching oligonucleotides: Firstly, the oligonucleotides are connected to short, protein-like molecules called peptides that are designed to help them to distribute in the body and get into cells. Secondly, there is a separate treatment with small molecules called OECs, developed by Juliano and Initos, which help the therapeutic oligonucleotides escape their entrapment within endosomes.

The researchers demonstrated this combined approach in cultured airway cells from a human CF patient with a common splicing-defect mutation.

Adding it just once to these cells, at a relatively low concentration, essentially corrected CFTR to a normal level of functioning, with no evidence of toxicity to the cells, Kreda said.

The results were much better with than without OECs, and improved with OEC dose.

There is no mouse model for splicing-defect CF, but the researchers successfully tested their general approach using a different oligonucleotide in a mouse model of a splicing defect affecting a reporter gene. In these experiments, the researchers observed that the correction of the splicing defect in the mouse lungs lasted for at least three weeks after a single treatment hinting that patients taking such therapies might need only sporadic dosing.

The researchers now plan further preclinical studies of their potential CF treatment in preparation for possible clinical trials.

Yan Dang, Catharina van Heusden, Veronica Nickerson, Felicity Chung, Yang Wang, Nancy Quinney, Martina Gentzsch, and Scott Randell were other contributors to this study from the Marsico Lung Institute; Ryszard Kole a co-author from the UNC Department of Pharmacology.

The Cystic Fibrosis Foundation and the National Institutes of Health supported this work.

Media contact: Mark Derewicz, UNC School of Medicine, 919-923-0959

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Scientists Demonstrate Promising New Approach for Treating Cystic Fibrosis | Newsroom - UNC Health and UNC School of Medicine

The aim of the present study was to investigate mutation status of the cKit and PDGFRA genes in patients with a gastrointestinal stromal tumor (GIST).In total, 96 patients with a GIST were included in the study, in which polymerase chain reaction amplification and gene sequencing were used to detect the sequences of exons 9, 11, 12, 13, 14, 17, and 18 in KIT and exons 12, 14, and 18 in PDGFRA.KIT mutations were detected in 65 cases (67.71%), of which 81.54% (53/65) were located on exon 11, 12.31% (8/65) were located on exon 9, 4.61% (3/65) were located on exon 17, which included a concomitant mutation of exon 9 and 11, and 4.08% (2/65) were located on exon 13, which included a concomitant mutation on exon 11. The most common mutation in exon 11 was deletion, which accounted for 77.36% (41/53) of the cases, followed by a point mutation observed in 22.64% (12/53) of the cases. Among the 31 GIST cases without a KIT mutation, a mutation in PDGFRA was detected in 5 cases (5.21%, 5/96; 16.13%, 5/31). With respect to gender, age, tumor max diameter, tumor position, and mitotic index, there were no significant differences between KIT/PDGFRA mutations and non-mutations.GIST mainly occurs in the stomach, and the cytological morphology is mainly spindle cells, and the mutations mainly occur in KIT genes. We need a large sample size to analyze the regularity of GIST gene mutations in Hakka population and understand the independent prognostic correlation of all KIT/PDGFRA genotypes.

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Mutations of the and gene in gastrointestinal stromal tumors among hakka population of Southern China. - Physician's Weekly

SEOUL, Korea, June 14, 2021 (GLOBE NEWSWIRE) -- On June 14, GENECAST (CEO, SeungChan Baek), a specialist in liquid biopsy-based cancer diagnostics, announced that it will begin a clinical study for the early diagnosis of non-small cell lung cancer (NSCLC). The team will be led by Professor Jhingook Kim in the Lung and Esophageal Surgery Division within the Department of Thoracic Surgery at Samsung Medical Center.

This study evaluates whether the results of liquid biopsy-based EGFR testing with GENECAST's ADPS technology can represent the results of EGFR testing on tumor tissue DNA. This study is especially significant for identifying the clinical effects of liquid biopsies on early cancer detection, given that it involves early-stage cancer patients at the 1B to 3A clinical stages.

To date, the clinical effects of liquid biopsies applied to lung cancer patients have mostly been researched with a focus on lung cancer that has been systemically spread through blood. In other words, lung cancer containing a portion of multiple tumor cells in the blood. On other hand, little research has been done about the power to detect EGFR gene mutations before surgery in patients with lung cancer that is resectable due to the absence of lesions spread through blood. This is because carrying out this research is considerably difficult.

In recent years, active discussions have globally been under way over the possibility of researching the pre-surgical administration of EGFR inhibitors for resectable early-stage lung cancer. This is because the pre-surgical administration of EGFR inhibitors is expected to not only enable surgery by blocking the spread of tumors in early stages and reducing the size of tumors, but also increase the actual survival rate of patients. In addition, it is easier to administrate drugs before surgery. Using EGFR inhibitors should be preceded by the diagnosis of EGFR gene mutations, but traditional biopsies often involve high risks or are impossible due to the nature of sites of lung cancer.

If the clinical effects of liquid biopsies with ADPS technology prove that this diagnostic method can detect early-stage NSCLC, then it is expected that this will lead to more active research efforts on the administration of EGFR inhibitors in these early-stage patients.

GENECAST's chief technical officer (CTO) Byungchul Lee ph.D commented, "With the recent FDA approval of Osimertinib as an adjuvant therapy for early-stage NSCLC, the early diagnosis of this cancer using liquid biopsies is receiving attention again. If liquid biopsies can diagnose not only metastatic lung cancer, but also operatable early-stage lung cancer, then they are likely to improve the survival rates of NSCLC patients."

Professor Jhingook Kim, a specialist in lung cancer in the Department of Thoracic Surgery at Samsung Medical Center, expressed his expectations for this study: "The study is very significant in terms of identifying the effects of liquid biopsies on early-stage patients considering resective surgery for a complete recovery, not patients with systemic metastases. Depending on the study results, follow-up studies will likely be conducted on various topics such as the use of liquid biopsies in the early diagnosis of cancer.

# Overview of GENECAST

GENECAST is a specialist in liquid biopsy-based cancer diagnostics that analyze cancer genes. This company has achieved 0.01% of actual detection sensitivity and the highest sensitivity of 0.0001% through its own original technology called the Allele-Discriminating Priming System (ADPS). ADPS is a qPCR-based technology that provides simpler and faster testing than other liquid biopsy-based diagnostic methods. It enables the analysis of cancer genes in Stage 1 cancer patients by realizing the highest detection sensitivity available today. GENECAST is operating a range of businesses including precision medicine, LDT commercialization, and companion diagnostics based on ADPS technology, and is dedicated to the development of new technologies that can realize early diagnostics according to the type of cancer. More information can be found at http://www.igenecast.com.

Contact:

Soobin ShinGENECAST Press OfficeTel: +82-2-2157-3151E-mail: comm@genecast.co.kr

A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/96d91465-1699-41a3-89f2-6be43bf58df5

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GENECAST begins clinical research on early diagnosis of lung cancer with Samsung Medical Center - GlobeNewswire

The two entities are looking to expand the potential applications of mRNA therapeutics to tackle a range of diseases.

The delivery of mRNA effectively and safely into the cell is one of the biggest challenges for expanding the use of mRNA therapeutics to promising fields such as cancer immunotherapy, protein replacement and gene editing.

The German company said a polymer-based delivery system was developed at the US university and complements its own technology platform for mRNA delivery. Known as ChargeAlteringReleasableTransporters (CART), the system was developed by Professor Robert Waymouth, Professor Paul Wender and Professor Ronald Levy.

Starting this month, Evonik and Stanford scientists will begin a three-year sponsored research collaboration to develop CART, which Evonik will license and commercialize.

Through this project we look forward to enabling the next generation of mRNA-based medicine, said Dr Thomas Riermeier, head of Evoniks Healthcare business line.

Evonik said its team will work together with Stanford University scientists to scale up the synthesis and formulation, and further develop its innovative technology for organ selective delivery based on a non-animal-derived, synthetic degradable polymer.

Evonik said it is aiming to make this technology GMP quality and available for use in clinical-stage developments and, ultimately, on a commercial scale.

The move is intended to expand Evoniks portfolio as a system solutions partner for advanced drug delivery.

Evonik said it recognized the potential of gene-based therapeutic approaches early on, making a targeted investment in this space with the acquisition of Transferra Nanosciences in 2016, a Vancouver-based lab with a strong focus on parenteral drug formulation development using lipid nanoparticles and liposomes.

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Evonik collaborating with Stanford University on 'next generation' of mRNA-based medicine - BioPharma-Reporter.com