Category Archives: Gene Medicine

SINGAPORE - The Republic is embarking on a mission to map the DNA and the complete set of genes of 100,000 Singaporeans - to unravel clues that will further the population's health and well-being.

By scrutinising genes and dissecting biological processes at the molecular level, doctors and researchers can pinpoint the underlying causes of cancer and chronic ailments, and identify new ways to prevent or slow down disease progression.

This would help to pave the way for new drugs, faster diagnostics and targeted treatments through precision medicine, said Professor Patrick Tan, executive director of Precision Health Research, Singapore (Precise), the entity that will helm the population study.

Over the next three years, the whole genomes of 100,000 healthy Singaporeans - between the ages of 30 and 80 - will be sequenced and analysed to create one of Singapore's largest research data sets.

Malay and Indian participants will comprise 40 per cent of the 100,000, and the rest will be Chinese.

The project, called SG100K, will end up as one of Asia's leading reference genome databases since Singapore's ethnic diversity captures more than 80 per cent of Asia's diversity.

On Thursday (May 26), Precise and genomics technology leader Illumina inked a partnership agreement to kick-start the SG100K effort.

The whole genome sequences will amount to 20 petabytes of data - which is equivalent to about 200,000 high-definition movies. The human brain can store around 2.5 petabytes of memory.

To date, the project has enrolled 70,000 participants from existing cohort studies, and is working towards recruiting the remaining 30,000 participants at a rate of 300 participants per week, said Deputy Prime Minister Heng Swee Keat, who attended the signing ceremony and toured Illumina's premises in Woodlands on Thursday.

Mr Heng added that Precise has in place robust safeguards to protect the privacy and security of data collected from people.

For one, Illumina has invested heavily in safeguarding its software, and researchers need approval to access the anonymised data, said Prof Tan.

The remaining 30,000 participants will be recruited mainly through referrals from the healthcare clusters, and from those who sign up for The Health For Life In Singapore population cohort study, led by Lee Kong Chian School of Medicine.

Under the SG100K project, participants' blood samples will be sent for whole genome sequencing.

The genomic data will also be linked to other physical health measurements taken from the participants. This means that participants have to undergo a whole day of tests, which includemeasuring blood sugar and cholesterol levels, a fitness test using trackers, and answer a questionnaire on their lifestyle.

Read the rest here:
Project SG100K: DNA of 100000 Singaporeans to be mapped to identify new ways to prevent diseases - The Straits Times

image:Mitochondria being engulfed by autophagosome view more

Credit: Jlio C. B. Ferreira/USP

A study by Brazilian scientists points to the probable biological mechanism that modulates the accumulation of mutant mitochondrial DNA in cells during aging, especially in the liver. The mechanism is called autophagy, a process of cellular cleansing that destroys organelles, including mitochondria, and recycles their components. The findings resolve a paradigm in the field and raise a hypothesis for the emergence of diseases associated with mitochondrial DNA, paving the way for the development of therapies.

The study was supported by FAPESP and is reported in an articlepublishedin the journalAutophagy.

Mitochondria are responsible for cellular respiration, processing substrates to supply energy to the organism. They have their own genome inherited solely from the mother, unlike the DNA found in cell nuclei, which is inherited from both parents. Mitochondrial DNA is also subject to mutations, some of which can lead to diseases. According to estimates, disorders caused by mitochondrial DNA mutations affect at least one in every 5,000 people worldwide. One of these disorders is MELAS syndrome (Mitochondrial Encephalopathy, Lactic Acidosis, and Stroke-like episodes), which can cause seizures, pain, altered consciousness and focal neurological deficits, among other symptoms.

Besides the disorders due directly to mitochondrial DNA mutations, there are others to which mutations contribute at least partly, saidMarcos Chiaratti, last author of the article. These include diabetes, Parkinsons and Alzheimers, he added, indicating that the study could pave the way for treatments involving autophagy in the context of different diseases.

Chiaratti is a professor in the Department of Genetics and Evolution at the Federal University of So Carlos (UFSCar) in So Paulo state. The other authors of the article are researchers affiliated with UFSCar and the University of So Paulo (USP) in Brazil, and with Cedars-Sinai Medical Center and Stanford University in the United States. The study was led by Chiaratti and supported by FAPESP via seven projects (16/07868-4,17/05899-2,17/04372-0,18/20028-0,20/15412-6,19/25049-9, and12/50231-6).

Aging

Research has shown that mitochondrial DNA mutations build up as part of aging. One of the oldest questions in biomedicine is why this is so, Chiaratti said. Theories that blame mitochondrial DNA replication or cell proliferation have been ruled out by previous studies. Given the importance of autophagy in the life cycle of cells and mitochondrial recycling, Chiaratti and colleagues decided to see if it played a role in mitochondrial DNA mutations. Autophagy is in vogue. The scientific literature on the topic is growing, he said. The 2016 Nobel Prize in Physiology or Medicine was awarded to Japanese cell biologist Yoshinori Ohsumi for his discoveries relating to autophagy mechanisms.

Methodology

Practically all humans have a small amount of mutant DNA. The coexistence of mutant and wild-type (non-mutant) mitochondrial DNA in a cell is called heteroplasmy. In this study, Chiaratti et al. used mice with heteroplasmy at 30%: a mutant strain known as NZB/BINJ (or NZB) accounted for 30% of their mitochondrial genome, while the rest was C57BL/6N (or BL6), considered normal.

The researchers cross-bred these mice with others that lacked the geneatg7specifically in the liver. Without this gene, the requisite machinery to make autophagy possible simply disappears. We spent almost a year just on breeding, Chiaratti said.

After this period, they had two groups of mice: a control group with NZB mitochondrial DNA and the geneatg7; and a group with NZB but without the geneatg7in liver cells, so that autophagy in the liver was impossible (liver-specificatg7knockout).

Experiments were conducted to see if autophagy affected the buildup of mutant mitochondrial DNA over time. Ten days after birth, similar levels of NZB were found in the tissue of both groups of mice, but after 21 days NZB began to accumulate in the livers of the control animals, while the knockout group had the same proportion as before. After 100 days, when mice are considered adults, the controls had even higher levels of NZB while the knockouts had practically the same levels as when they were born, making the difference between the two groups even more evident and suggesting that autophagy plays a role in the age-related buildup of mutant mitochondrial DNA.

The researchers then set out to validate this finding by conducting an experiment with mice that lacked the genePrkn, which is responsible for a specific stage of autophagy. InPrknknockout mice, autophagy occurred but was defective. We again compared these mice with the controls, Chiaratti said. Although thePrknknockout mice did eventually accumulate NZB mitochondrial DNA, the buildup was smaller than for the control group. This reinforced the theory that autophagy is involved since manipulation of another gene associated with it led to changes in the data.

The researchers also found that autophagy mostly cleared BL6 mitochondrial DNA in controls, which presumably explained the higher levels of NZB. On exploring this possibility further, they discovered that the presence of NZB enhanced mitochondrial respiratory capacity in liver cells. We, therefore, postulated that autophagy might help eliminate less functional mitochondria while conserving the fittest, which of course would be positive, Chiaratti said.

Another stage of the study entailed isolation of fibroblasts, a type of connective tissue cell, from the mice, and the in vitro observation that NZB mitochondrial DNA levels in them fell over time. However, when drugs that stimulated autophagy were applied, the decline was reversed. This was further evidence of the importance of autophagy to mitochondrial DNA regulation, Chiaratti said.

Next steps

While NZB was considered mutant in the mice involved in these experiments, the results show that it is not detrimental to the liver. On the contrary, it enhances mitochondrial respiratory capacity in liver cells, as already noted. Now we need to investigate the phenomenon with different mitochondrial DNAs that are known to be pathological, Chiaratti said.

If autophagy also proves relevant in this context, the researchers can begin to test treatments that stimulate the cell cleansing mechanism against diseases affected by the functioning of mitochondria. Other possibilities for future research, Chiaratti added, will include the role of autophagy in mitochondrial DNA accumulation in tissue elsewhere in the body apart from the liver and isolated fibroblasts.

###

About So Paulo Research Foundation (FAPESP)

The So Paulo Research Foundation (FAPESP) is a public institution with the mission of supporting scientific research in all fields of knowledge by awarding scholarships, fellowships and grants to investigators linked with higher education and research institutions in the State of So Paulo, Brazil. FAPESP is aware that the very best research can only be done by working with the best researchers internationally. Therefore, it has established partnerships with funding agencies, higher education, private companies, and research organizations in other countries known for the quality of their research and has been encouraging scientists funded by its grants to further develop their international collaboration. You can learn more about FAPESP atwww.fapesp.br/enand visit FAPESP news agency atwww.agencia.fapesp.br/ento keep updated with the latest scientific breakthroughs FAPESP helps achieve through its many programs, awards and research centers. You may also subscribe to FAPESP news agency athttp://agencia.fapesp.br/subscribe.

Autophagy deficiency abolishes liver mitochondrial DNA segregation

27-Feb-2022

Here is the original post:
Study reveals potential target for treatment of diseases associated with mitochondrial DNA mutations - EurekAlert

NEW YORK, May 25, 2022 /PRNewswire/ --Elevation Oncology, Inc. (Nasdaq: ELEV), a clinical stage biopharmaceutical company focused on the development of precision medicines for patients with genomically defined cancers, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to seribantumab for the tumor-agnostic treatment of advanced solid tumors that harbor NRG1 gene fusions. Seribantumab is currently being evaluated in the ongoing Phase 2 CRESTONE study, for which initial data will be presented in an oral presentation at the upcomingAmerican Society of Clinical Oncology (ASCO) 2022 Annual Meeting on Tuesday, June 7, 2022.

"There are currently no approved therapies that specifically target NRG1 fusions, and therefore, receipt of Fast Track designation in a tumor-agnostic setting is a significant step in addressing this unmet need," said Shawn M. Leland, PharmD, RPh, Founder and Chief Executive Officer of Elevation Oncology. "NRG1 fusions are a type of genomic alteration that causes unregulated cell growth and proliferation in a variety of solid tumors, and we look forward to working closely with the FDA as we continue exploring the potential of seribantumab to improve outcomes for patients whose tumor harbors this unique oncogenic driver."

Fast Track is an FDA process designed to facilitate the development and expedite the review of potential therapies that seek to treat serious conditions and fill an unmet medical need. A drug candidate that receives Fast Track designation is afforded greater access to the FDA for the purpose of expediting the drug's development, review and potential approval. Additionally, the designation allows for eligibility for Accelerated Approval and Priority Review, if relevant criteria are met, as well as a Rolling Review, which means a drug company can submit completed sections of its New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be submitted for review.

About Seribantumab and NRG1 Gene Fusions

Seribantumab is a fully human IgG2 monoclonal antibody that binds to human epidermal growth factor receptor 3 (HER3). HER3 is traditionally activated through binding of its primary ligand, neuregulin-1 (NRG1). The NRG1 gene fusion is a rare genomic alteration that combines NRG1 with another partner protein to create chimeric NRG1 "fusion proteins". The NRG1 fusion protein is often also able to activate the HER3 pathway, leading to unregulated cell growth and proliferation. Importantly, NRG1 gene fusions are predominantly mutually exclusive with other known genomic driver mutations and are considered a unique oncogenic driver event associated with tumor cell survival.

NRG1 fusions have been identified in a variety of solid tumors, including lung, pancreatic, gallbladder, breast, ovarian, colorectal, neuroendocrine, cholangiocarcinomas, and sarcomas. In preclinical experiments, seribantumab prevented the activation of HER3 signaling in cells that harbor an NRG1 gene fusion and destabilized the entire ERBB family signaling pathway including the activation of HER2, EGFR, and HER4. In addition to extensive nonclinical characterization and testing, seribantumab has been administered to over 800 patients across twelve Phase 1 and 2 studies, both as a monotherapy and in combination with various anti-cancer therapies. Seribantumab was granted Fast Track designation from the FDA for the tumor-agnostic treatment of patients whose solid tumors harbor NRG1 fusions and is currently being evaluated in the Phase 2 CRESTONE study for patients with solid tumors of any origin that have an NRG1 fusion.

About the Phase 2 CRESTONE Study

CRESTONE (Clinical Study of Response to Seribantumab in Tumors with Neuregulin-1 (NRG1) Fusions; NCT04383210) is a Phase 2 tumor-agnostic study evaluating seribantumab in patients with solid tumors that harbor an NRG1 fusion and have progressed after at least one prior line of standard therapy. The primary objective of the study is to describe the anti-tumor activity and safety of seribantumab as a monotherapy specifically in patients whose solid tumor is uniquely driven by an NRG1 gene fusion. CRESTONE offers a clinical trial opportunity for patients with advanced solid tumors who have not responded or are no longer responding to treatment. Patients are encouraged to talk to their doctor about genomic testing of their tumor. CRESTONE is open and enrolling today in the United States, Australia, and Canada. For more information visit http://www.NRG1fusion.com.

About Elevation Oncology, Inc.

Elevation Oncology is founded on the belief that every patient living with cancer deserves to know what is driving the growth of their disease and have access to therapeutics that can stop it. We aim to make genomic tests actionable by selectively developing drugs to inhibit the specific alterations that have been identified as drivers of tumor growth. Together with our peers, we work towards a future in which each tumor's unique genomic test result can be matched with a purpose-built precision medicine to enable an individualized treatment plan for each patient. Our lead candidate, seribantumab, is intended to inhibit tumor growth driven by NRG1 fusions and is currently being evaluated in the Phase 2 CRESTONE study for patients with solid tumors of any origin that have an NRG1 gene fusion. Details on CRESTONE are available at http://www.NRG1fusion.com. For more information visit http://www.ElevationOncology.com.

Forward Looking Statements:

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to, anticipated preclinical and clinical development activities, expected timing of announcements of clinical results, potential benefits of seribantumaband the company's other future product candidates, potential opportunities to expand the company's product candidate pipeline, potential market opportunities for seribantumaband the company's other future product candidates, the ability of seribantumaband the company's other future product candidates to treat their targeted indications, and our expectations about our cash runway. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. These forward-looking statements may be accompanied by such words as "aim," "anticipate," "believe," "could," "estimate," "expect," "forecast," "goal," "intend," "may," "might," "plan," "potential," "possible," "will," "would," and other words and terms of similar meaning. Although the company believes that the expectations reflected in such forward-looking statements are reasonable, the company cannot guarantee future events, results, actions, levels of activity, performance or achievements, and the timing and results of biotechnology development and potential regulatory approval is inherently uncertain. Forward-looking statements are subject to risks and uncertainties that may cause the company's actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the company's ability to advance its product candidates, the timing and results of preclinical studies and clinical trials, approvals and commercialization of product candidates, the receipt and timing of potential regulatory designations, the impact of the COVID-19 pandemic on the Company's business, the Company's ability to fund development activities and achieve development goals, the Company's ability to protect intellectual property, the Company's ability to establish and maintain collaborations with third parties and other risks and uncertainties described under the heading "Risk Factors" in documents the company files from time to time with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.

Elevation Oncology Investor and Media ContactCandice Masse, 978-879-7273Senior Director, Corporate Communications & Investor Relations[emailprotected]

SOURCE Elevation Oncology

More here:
Elevation Oncology Announces FDA Fast Track Designation Granted to Seribantumab for the Tumor-Agnostic Treatment of Solid Tumors Harboring NRG1 Gene...

INOVIO's DNA medicines immunotherapy in combination with Libtayo elicits vaccine-associated immune responses when administered with RT/TMZ to newly diagnosed GBM patients

INO-5401 + INO-9012 + Libtayoelicits cancer antigen-specific T cells

55% of MGMT methylated subjects remain alive at a median of 32.5 months

Dr. David Reardon, Principal Investigator, to present on June 6, 2022 at ASCO

PLYMOUTH MEETING, Pa., May 27, 2022 /PRNewswire/ -- Inovio (NASDAQ: INO) announced results from the company's novel Phase 1/2 trial of INO-5401 and INO-9012 in combination with PD-1 inhibitor Libtayo (cemiplimab) in the treatment of newly diagnosed glioblastoma (GBM), including encouraging median overall survival (OS) data from fifty-two subjects. Median OS duration in unmethylated MGMT (Cohort A) was 17.9 months. Median OS data in MGMT Methylated patients (Cohort B) are being presented for the first time, at a median of 32.5 months, which compares favorably to historical comparisons (23.2-25 months).

Overall, INO-5401 + INO-9012 is demonstrated to be tolerable and immunogenic when administered with Libtayo and RT/TMZ (radiation and temozolomide) to newly diagnosed GBM patients. Notably, INO-5401 elicited antigen-specific T cells that may infiltrate GBM tumors. The data from this study was selected to be presented in an oral presentation by Dr. David Reardon on Monday, June 6, 2022, at the 2022 American Society of Clinical Oncology (ASCO) at the McCormick Place Convention Center in Chicago, Illinois.

Presentation Details: June 6, 2022, 12:42 12:54 p.m. CDTPresenting Author: David A. ReardonCentral Nervous System Tumors Session

Abstract #2004: Intramuscular (IM) INO-5401 + INO-9012 with electroporation (EP) in combination with cemiplimab (REGN2810) in newly diagnosed glioblastoma

Fifty-two subjects were enrolled: 32 in Cohort A; 20 in Cohort B (35% women; median age 60 years [range 19-78 years]). The adverse event profile was consistent with known single-agent (INO-5401, INO-9012, EP or Libtayo) events; most events were Grade 2 and no related events were Grade 4. Median OS durations in Cohorts A and B were 17.9 months (95% CI 14.5-19.8) and 32.5 months (95% CI 18.4-not reached), respectively. Flow cytometry revealed activated, antigen specific CD4+CD69+PD1+ and CD8+CD69+PD1+ T cells, the latter with lytic potential as defined by presence of perforin and granzyme A. Both subsets exhibited HR<1.0 and p<0.05 when accounting for a 0.1% T cell frequency change, translating to a 23% and 28% reduced risk of death at 18 months, respectively.

A post-hoc exploratory analysis showed that gene expression levels of INO-5401 antigens and immune cell markers from pre-treatment tumor tissues were similar between alive and deceased groups; however, the alive group displayed significant differential expression of genes regulating apoptosis, proliferation, and immune responses. Post-treatment tumor tissue displayed altered gene expression for immune-related markers versus pre-treatment tissue, including markers of T cell infiltration, activation, and lytic potential.

Dr. David Reardon, Clinical Director, Center for Neuro-Oncology of Dana-Farber Cancer Institute and coordinating principal investigator of the study said, "GBM remains one of the most aggressive and hard-to-treat cancers. The fact that we have seen this novel combination trial of a T cell generating DNA medicine combined with a PD-1 checkpoint benefit a large percent of trial participants past 32 months is very encouraging. These latest results and continued development are welcoming as it continues to improve upon a standard of care which was defined 17 years ago and remains sub-optimal for our patients with GBM."

Dr. Jeffrey Skolnik, INOVIO's Senior Vice President, Clinical Development, said, "We, along with our collaborative partner Regeneron, remain encouraged with the progress to date from this novel combination therapy study. As concluded in the abstract, INO-5401 + INO-9012 has an acceptable risk/benefit profile and elicits robust immune responses that may correlate with a potentially enhanced survival when administered with Libtayo and RT/TMZ to newly diagnosed GBM patients. Our goal is to build upon INO-5401's ability to elicit antigen-specific T cells that can infiltrate GBM tumors and complement the clinically-active profile of Libtayo to a potentially larger study in the future."

INO-5401, INO-9012, Libtayo, and the combination of these products have not been approved or evaluated by any Regulatory Authority worldwide for the treatment of newly diagnosed GBM.

Study Design

The trial was designed to evaluate safety, immunogenicity and efficacy of INO-5401 and INO-9012 in combination with Libtayo, with radiation and chemotherapy, in subjects with newly diagnosed glioblastoma (GBM). This is a Phase 1/2, open-label, multi-center trial conducted in 52 evaluable patients with GBM. There are two cohorts in this trial. Cohort A includes 32 participants with a tumor with an unmethylated O6-methylguanine-deoxyribonucleic acid (DNA) methyltransferase (MGMT) promoter. Cohort B includes 20 participants with a tumor with a MGMT methylated promoter. Both cohorts received INO-5401 and INO-9012 and Libtayo at the same doses and on the same dosing schedule, and both cohorts received radiation and TMZ. For more information of the clinical study, see http://www.clinicaltrials.gov, identifier NCT03491683.

About INO-5401 and INO-9012

INO-5401 encodes for INOVIO's SynCon antigens for hTERT, WT1, and PSMA, and has the potential to be a powerful cancer immunotherapy in combination with checkpoint inhibitors. The National Cancer Institute previously highlighted hTERT, WT1, and PSMA among a list of important cancer antigens, designating them as high priorities for cancer immunotherapy development. These three antigens were reported to be over-expressed, and often mutated, in a variety of human cancers including glioblastoma, and targeting these antigens may prove efficacious in the treatment of patients with cancer. INO-9012 encodes for IL-12, which is a T cell immune activator.

About Glioblastoma (GBM)

GBM is the most common and aggressive type of brain cancer and remains a devastating disease for both patients and caregivers. Its prognosis is extremely poor, with very few new therapies approved over the last 10 years. The median overall survival for patients receiving standard of care therapy is approximately 15 to 22 months and the median progression-free survival is approximately 7-10 months. In the U.S., the estimated annual incidence of GBM is 11,362 cases or 3.21 cases per 100,000 persons and the median age at diagnosis is 65 years.

About INOVIOINOVIO is a biotechnology company focused on developing and commercializing DNA medicines to help protect people from infectious diseases and help treat people with cancer and HPV-associated diseases. Our DNA medicines are delivered using our proprietary smart device to produce a robust and tolerable immune response against targeted pathogens and cancers.

Partners and collaborators include Advaccine, ApolloBio Corporation, AstraZeneca, The Bill & Melinda Gates Foundation, Coalition for Epidemic Preparedness Innovations, Defense Advanced Research Projects Agency/Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense/Department of Defense, HIV Vaccines Trial Network, International Vaccine Institute, Kaneka Eurogentec, Medical CBRN Defense Consortium, National Cancer Institute, National Institutes of Health, National Institute of Allergy and Infectious Diseases, Ology Bioservices, the Parker Institute for Cancer Immunotherapy, Plumbline Life Sciences, Regeneron, Richter-Helm BioLogics, Thermo Fisher Scientific, University of Pennsylvania, Walter Reed Army Institute of Research, and The Wistar Institute. For more information, visit http://www.inovio.com.

CONTACTS:

Media: Jeff Richardson, 267-440-4211, jrichardson@inovio.comInvestors: Ben Matone, 484-362-0076, ben.matone@inovio.com

This press release contains certain forward-looking statements relating to our business, including our plans to develop DNA medicines, our expectations regarding our research and development programs, including the planned initiation and conduct of preclinical studies and clinical trials and the availability and timing of data from those studies and trials, and our ability to successfully manufacture and produce large quantities of our product candidates if they receive regulatory approval. Actual events or results may differ from the expectations set forth herein as a result of a number of factors, including uncertainties inherent in pre-clinical studies, clinical trials, product development programs and commercialization activities and outcomes, our ability to secure sufficient manufacturing capacity to mass produce our product candidates, the availability of funding to support continuing research and studies in an effort to prove safety and efficacy of electroporation technology as a delivery mechanism or develop viable DNA medicines, our ability to support our pipeline of DNA medicine products, the ability of our collaborators to attain development and commercial milestones for products we license and product sales that will enable us to receive future payments and royalties, the adequacy of our capital resources, the availability or potential availability of alternative therapies or treatments for the conditions targeted by us or our collaborators, including alternatives that may be more efficacious or cost effective than any therapy or treatment that we and our collaborators hope to develop, issues involving product liability, issues involving patents and whether they or licenses to them will provide us with meaningful protection from others using the covered technologies, whether such proprietary rights are enforceable or defensible or infringe or allegedly infringe on rights of others or can withstand claims of invalidity and whether we can finance or devote other significant resources that may be necessary to prosecute, protect or defend them, the level of corporate expenditures, assessments of our technology by potential corporate or other partners or collaborators, capital market conditions, the impact of government healthcare proposals and other factors set forth in our Annual Report on Form 10-K for the year ended December 31, 2021, our Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 and other filings we make from time to time with the Securities and Exchange Commission. There can be no assurance that any product candidate in our pipeline will be successfully developed, manufactured or commercialized, that final results of clinical trials will be supportive of regulatory approvals required to market products, or that any of the forward-looking information provided herein will be proven accurate. Forward-looking statements speak only as of the date of this release, and we undertake no obligation to update or revise these statements, except as may be required by law.

View original content:https://www.prnewswire.com/news-releases/inovio-announces-survival-results-for-ino-5401--ino-9012-in-combination-with-libtayo-cemiplimab-in-patients-with-newly-diagnosed-gbm-at-asco-annual-meeting-2022-301556446.html

SOURCE INOVIO Pharmaceuticals, Inc.

More:
INOVIO Announces Survival Results for INO-5401 + INO-9012 in Combination with Libtayo (cemiplimab) in Patients with Newly Diagnosed GBM at ASCO Annual...

DUBLIN, May 27, 2022 /PRNewswire/ -- The "Single-use Technologies for Biopharmaceuticals: Global Markets" report has been added to ResearchAndMarkets.com's offering.

The report summarizes the market, including a market snapshot and company profiles of key players in the sole-use technologies market. It provides a comprehensive market breakdown with in-depth information about each segment. The overview section of the report describes market trends and dynamics, including drivers, limitations, challenges, and opportunities for the market.

Furthermore, it provides information about market development and future trends useful for organizations, including distributors and exporters. It analyzes critical market players' revenue, product portfolio and recent activities. It further includes strategies adopted by emerging market players with strategic recommendations for new market entrants. Outright information is provided in the report, consisting of historical and current market size, including the market's future potential.

The report will also help inform market players and new entrants about the production and export of goods and services to original equipment manufacturers. The market is segmented based on technology, components, applications, and end user. A geographical market analysis is provided for all the major segments. The report offers a country-level analysis of the market to understand major components better.

Report Includes

Single-use bioreactor technology has gained considerable importance in biotechnology manufacturing over the years. Several single-use options are available. Scalability is the biggest limitation. The industry's willingness to use single-use bioreactors is influenced by production parameters, product value and development time. It takes more time to complete comparative studies with conventional stainless-steel bioreactors as the rate of implementation is lower than that of acceptance, thus making single-use technology highly desirable in the biopharmaceutical industry.

However, more clarity and understanding regarding the regulatory requirements for single-use bioreactor technology are needed. For example, U. S. FDA regulations for the Cord Blood Registry (CBR) do not explicitly mention single-use bioreactor technology, even though a large number of Investigational New Drug (IND) programs have been approved by the FDA using such systems.

The major factors influencing the growth of the market include increasing demand for personalized medicine, extensive ongoing development efforts, a strong product portfolio, and large application areas for single-use systems. Additionally, lower cost and reduction in the time necessary in the biomanufacturing process when using single-use technology are further driving the growth of the market.

The drug development rate has increased rapidly with the increasing demand for personalized medicines. This has, in turn, increased the demand for single-use technology to avoid the risk of contamination.

A strong product portfolio is further fueling the growth of the market during the forecast period. There are several companies that are offering single-use technologies, such as Thermo Fisher Scientific Inc. , Danaher Corp. , Sartorius AG, General Electric Co. , and PendoTECH LLC. PendoTECH LLC is focused on the development of pressure sensors used to measure static and dynamic pressure of gases and liquids in biopharmaceutical processes.

It also provides a wide range of single-use products such as single-use rotary flowmeters, single-use ultrasonic flowmeters and a compact low-flow ultrasonic flow meter with are usable fluid path.

Key Topics Covered:

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Market and Technology Background

Chapter 4 Biologics Process Development Pathway and Application of Single-Use Technologies

Chapter 5 Covid-19 Impact on Market

Chapter 6 Market Breakdown by Technology

Chapter 7 Market Breakdown by Single-Use Component

Chapter 8 Market Breakdown by Application

Chapter 9 Market Breakdown by End-user

Chapter 10 Market Breakdown by Region

Chapter 11 Patent Review

Chapter 12 Competitive Analysis and Market Opportunities

Chapter 13 Company Profiles

Chapter 14 Appendix: Abbreviations

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

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716

SOURCE Research and Markets

See the article here:
Outlook on the Single-use Technologies for Biopharmaceuticals Global Market to 2026 - Increasing Demand for Personalized Medicine is Driving Growth -...

When Dr. Sharl Azar first entered medicine he noticed something disturbing almost immediately.

I watched as a young man whose overwhelming pain still managed to betray his stoicism, was told by the physician who was caring for him in the (emergency department) that hed received all of the Dilaudid he was going to get for today and that he should go get his fix somewhere else, he said.

Azar, medical director at Massachusetts General Hospitals Comprehensive Sickle Cell Disease Treatment Center, has worked since to combat suffering and stigma caused by those who suffer from that disease, and, on Tuesday, he was asked to speak at the opening of a new, state-of-the-art cell and genetic therapy center in the citys Seaport neighborhood where that work will continue.

Vertex is allowing our center to bring our patients the world-class care that they have long been denied, he said.

Azar spoke as Vertex Pharmaceuticals, a Boston-based biotechnology company, officially opened their new corporate headquarters along Northern Avenue, where the drug developer will work hand in hand with hospitals like MGH to continue genetic research into the causes and hopefully the cure for diseases like sickle cell.

Called the Jeffrey Leiden Center for Cell and Genetic Therapies, the over 250,000-square-foot facility research and development facility includes 453 workspaces and 21,000 square feet of manufacturing space.

According to Leiden, Vertexs executive chairman, the facility represents the future in the fight against serious chronic disease.

I hope you can imagine a day when lifesaving cell and genetic therapies for many serious diseases sickle cell, diabetes, muscular dystrophy are being discovered, developed, manufactured and brought to patients by Vertex scientists and physicians working in this buildingI promise you that day is not very far away, Leiden said.

Gov. Charlie Baker was due to speak at the event but canceled his public appearances for the day after falling ill. Baker has tested negative for COVID-19, a spokesperson told the Herald.

Lt. Gov. Karyn Polito, who serves on the states STEM Advisory Council with Leiden, stepped in to open the facility on Bakers behalf.

This is a very proud moment, she said. Not just for the Leiden family and the Vertex team, but certainly for the city of Boston and for the commonwealth of Massachusetts.

Dr. Reshma Kewalramani, Vertex CEO, also announced the company would further expand into the Seaport with another research facility just across the street. She said the company will use the spaces to help solve serious medical problems.

Its going to happen. It may not happen tomorrow, but its going to happen in our lifetime, she said.

Continued here:
Bostons Seaport gets new cell and gene therapy research center, more to come - Boston Herald

NEW YORK, May 17, 2022 /PRNewswire/ -- SCRIPT BIOSCIENCES INC, a biotechnology company developing curative therapies using a novel gene editing platform (CasPlus), and NYU Grossman School of Medicine, presented a poster titled "Enhancement of Predictable and Template-free Gene Editing by the Association of CAS with DNA Polymerase" at the 25th annual American Society of Gene & Cell Therapy meeting in Washington DC (May 16-19, 2022). Highlights of the data include:

Dr. Chengzu Long, Principal Investigator and Assistant Professor, Division of Cardiology and The Helen and Martin Kimmel Center for Stem Cell Biology, NYU Grossman School of Medicine, said, "CRISPR/Cas9-mediated on-target DNA damage is an underappreciated risk factor for safe application of genome editing tools. While numerous approaches have already been developed to reduce well-known off-target effects of CRISPR-mediated editing, the CasPlus system prevents CRISPR-mediated collateral on-target large deletions and thus offers the promise of safer therapeutic gene editing in humans."

Anil Namboodiripad, Ph.D., CEO of Script Biosciences said, "We are encouraged by this data and it is a step forward in our mission to develop lasting cures for patients suffering from serious diseases. In vivo translational studies in humanized mouse models are ongoing. We plan to advance our lead program in DMD towards IND-enabling studies, while also expanding our pipeline to other gene targets".

About Script BiosciencesScript Biosciences (www.scriptbiosciences.com) is an early stage biotechnology company committed to developing safe, efficient, and durable cures for genetic diseases with few or no treatment options using our proprietary CasPlus genome editing platform (CasPlus). CasPlus corrects mutations by predictable and efficient insertion of base pairs. The superior precision of CasPlus paves the way towards safer genome editing in humans. The technology was developed at New York University with a worldwide license granted to Script.

SOURCE Script Biosciences Inc

More:
The CasPlus Gene Editing Platform Technology can Correct Mutations Associated with Duchenne's Muscular Dystrophy and Cystic Fibrosis with High...

PALO ALTO, Calif., May 17, 2022 /PRNewswire/ --Genascence Corporation ("Genascence"), a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, today announced that additional safety data from the Phase 1 clinical trial of GNSC-001 for the treatment of osteoarthritis (OA), including 12-month follow-up on all subjects, demonstrated that it was safe and well tolerated. These data will be delivered in a poster presentation today at theAmerican Society of Gene& Cell Therapy's(ASGCT) 25th Annual Meeting being held virtually and in-person May 16-19, 2022, in Washington, D.C.

GNSC-001 is the company's lead program in OA. GNSC-001 is a genetic medicine a recombinant adeno-associated vector (AAV) carrying a coding sequence for interleukin-1 receptor antagonist (IL-1Ra), a potent inhibitor of interleukin-1 (IL-1) signaling. IL-1 is considered one of the key mediators involved in the pathogenesis of OA, causing inflammation as well as cartilage destruction. GNSC-001 is designed to offer long-term, sustained inhibition of IL-1 following a single injection into the affected joint.

"Osteoarthritis is incapacitating, causing years of pain and disability for people living with the disease. Further, patients have limited treatment options, and nothing is currently available that is able to slow down progression of OA," said Thomas Chalberg, Ph.D., founder and CEO of Genascence. "We are excited by these findings as they demonstrate the initial safety of GNSC-001 and provide encouraging data to pursue GNSC-001 as a novel treatment for OA patients. We look forward to advancing the clinical program for GNSC-001 so that we can deliver transformative results for patients suffering from this disabling disease."

Title:A Phase I Trial of Osteoarthritis Gene Therapy (NCT02790723)Date:May 17, 2022 5:30-6:30 PM ETSession: Gene and Cell Therapy Trials in ProgressAbstract Number: 799Location: Walter E. Washington Convention Center, Hall DPresenter: Christopher H. Evans, Ph.D.

In this investigator-sponsored Phase 1 single-arm, open-label, dose-escalation clinical trial of GNSC-001, a total of nine subjects with knee OA were enrolled and monitored for one year. Three subjects were treated in each of three cohorts, receiving either 1x1011 vg, 1x1012 vg, or 1x1013 vg GNSC-001 delivered by intra-articular injection. The primary endpoint is safety and tolerability. Additional measures include levels of circulating viral genomes, immune response to the vector, blood and urine analysis, and physical examination. Although the study was not powered for efficacy and had no control group, patients reported pain via VAS (0-10) and pain and function via WOMAC. Knee joints were imaged by X-ray and MRI upon study entry and after one year.

Results showed that intra-articular injection of GNSC-001 produced no severe adverse events; blood chemistries and hematologies remained normal during the 12-month follow-up period with no evidence of neutropenia. There were no vector-related adverse events in eight of the nine subjects; one subject experienced a mild/moderate knee effusion following injection which resolved with ice and rest. Clinical trial participants developed various degrees of anti-AAV neutralizing antibodies after injection of GNSC-001, as seen in preclinical studies. Small amounts of viral genomes were found in peripheral blood, beginning one day after injection and clearing within four weeks. Injection of GNSC-001 was associated with increased concentrations of IL-1Ra in synovial fluid, which remained elevated after 12 months of follow up. Pain and function scores improved following injection of GNSC-001.

"These additional data from the Phase 1 trial of GNSC-001 in patients with osteoarthritis showed that it safe and well tolerated including after one year," said Dr. Evans. "These results are encouraging as we believe this therapy has the potential to reduce structural disease progression in osteoarthritis patients."

The study was supported by funding from the U.S. Department of Defense Peer Reviewed Medical Research Program (PRMRP). More information is available at https://clinicaltrials.gov/ct2/show/NCT02790723.

Abstracts can be accessed via the conference website at annualmeeting.asgct.org.

About Osteoarthritis (OA) of the Knee

Osteoarthritis (OA), or degenerative joint disease, is the leading cause of disability. It is characterized by destruction of cartilage and structural changes in bone within the joint, which contribute to pain and loss of joint function. Osteoarthritis affects more than 30 million Americans and is increasing as a result of the aging population and increasing prevalence of obesity. Osteoarthritis represents a major economic burden, owing to direct medical costs and loss of productivity. Each year, millions of patients are treated for knee OA with NSAIDs, opioids, and steroid injections into the knee to manage their knee pain. There are no currently available therapies known to alter or slow down OA progression.

About Genascence Corporation

Genascence, a clinical-stage biotechnology company revolutionizing the treatment of prevalent musculoskeletal diseases with gene therapy, is developing life-changing treatments for highly prevalent conditions affecting millions of people. The company was founded in 2017 with technology licensed from three leading U.S. research institutions: Mayo Clinic, University of Florida, and NYU Langone Health. Headquartered in Palo Alto, California, Genascence's founders and leadership team have deep experience in the design, development, and manufacturing of successful gene therapies and biological medicines. For more information, please visit http://www.genascence.com.

SOURCE Genascence

Read the original post:
Genascence Announces Data From Phase 1 Clinical Trial on GNSC-001, Company's Lead Program in Osteoarthritis, Presented at American Society of Gene...

DiNAQOR DiNAMIQS offers comprehensive range of biomanufacturing services,GMP-compatible process development, quality control and analytical development

ZURICH-SCHLIEREN, Switzerland, May 16, 2022 /PRNewswire/ --DiNAQOR announced today the launch of DiNAQOR DiNAMIQS, a biomanufacturing subsidiary, that will accelerate development timelines and reduce costs and risk for genetic medicine companies bringing new treatments to market.

DiNAQOR DiNAMIQS is based in the company's state-of-the-art, 1,200-square-meter (13,000 square feet) manufacturing facility in Bio-Technopark Schlieren-Zrich, the leading center for biotechnology companies in Central Europe. DiNAQOR's Chief Technology Officer, Eduard Ayuso, will serve as the CEO of DiNAQOR DiNAMIQS.

"We look forward to partnering with the world's leading gene therapy companies," said Johannes Holzmeister, M.D., Chairman and CEO of DiNAQOR."There are always challenges in the development process, and DiNAQOR DiNAMIQS is ideally suited to meet and overcome them. Manufacturing viral vectors at meaningful bioreactor scale and using GMP-compatible processes for preclinical studies will improve quality and safety while accelerating development timelines for genetic medicines."

DiNAQOR DiNAMIQS provides a comprehensive range of manufacturing services, process development, quality control and analytics solutions to support and accelerate gene therapy projects.

The DiNAMIQS platform employs innovative upstream and downstream processes and provides high-quality recombinant adeno-associated viral (AAV) vector manufacturing suitable for both in vitro and in vivo R&D applications up to 50L scale. Aligned with GMP regulations, the manufacturing protocol provides researchers with high quality vectors and minimal changes as projects progress toward clinical applications. DiNAMIQS is currently building a state-of-the-art GMP-compliant 2,400-square-meter (26,000 square feet) facility that can produce viral vectors at 500L scale.

DiNAQOR DiNAMIQS is also pioneering customizable, GMP-compatible process development that accelerates research and development efforts and is guided by a diagnostic procedure to determine relevant bioprocessing solutions. DiNAMIQS' process development expertise includes biomass expansion in bioreactor, large-scale transfection, harvest and clarification, ultrafiltration/diafiltration, affinity chromatography capture, ion exchange chromatography, preparative ultracentrifugation, desalting, dynamic dialysis, formulation, sterile filtration, automation assisted fill and finish.

Genetic medicinecompanies partnering with DiNAQOR DiNAMIQS will also use analytics that yield critical insights on viral vector potency, identity, and purity. DiNAMIQS in-house capabilities include digital PCR-based methods for titer quantification, ELISA, purity analyses, TCID50 infectivity assays and testing for bacterial endotoxins.

"Our state-of-the-art facility and stellar viral vector manufacturing team are prepared to help gene therapy developers bring their therapies efficiently to the clinic. I intend to bring my learnings from 20 years' experience in the field to our partners and provide them with high quality vectors. DiNAMIQS will shorten the time to market by closing the gaps between research grade vector supply, process development and GMP manufacturing," said Eduard Ayuso, CEO of DiNAQOR DiNAMIQS.

"Additionally, many promising gene therapy programs slow down when the costs associated with scaling up their manufacturing begin to mount. Our biomanufacturing expertise will enable these projects to move forward in a cost-effective way -- and do so quickly."

To learn more about DiNAQOR DiNAMIQS, visit http://www.dinamiqs.com.

About DiNAQORDiNAQOR is a life sciences platform company that is pioneering proprietary human-based tissue drug development and technology to enable organ-specific delivery of gene therapies and other therapeutics. The company is headquartered in Zurich-Schlieren, Switzerland, with additional presence in London, England; Hamburg, Germany; and Laguna Hills, California. For more information visit http://www.dinaqor.com.

Contact:KWM CommunicationsKellie Walsh914-315-6072[emailprotected]

SOURCE DiNAQOR

See the article here:
DiNAQOR Opens DiNAMIQS Subsidiary to Partner with Gene Therapy Companies Bringing New Treatments to Patients - PR Newswire

WASHINGTON, DC The covid virus killed some 460,000 Americanslast year, heart disease killednearly 700,000people in the U.S. in the same period.The fact is that heart attacks are the number one cause of death in America and throughout the world.So, the announcement that scientists are testing a way to prevent heart attacks with a single injection may sound like a life-saving game-changer, particularly for seniors over the age of 65 who, according to the National Institute on Aging, are much more likely than younger people to suffer aheart attack.

Verve Therapeutics describes itself as a biotechnology company created with a singular focus: to protect the world from heart disease.Over the next three years the company says it will be testing a radical gene therapy to permanently prevent the buildup of bad cholesterol with that single injection.Cardiologist, geneticist and CEO of Verve Therapeutics, Dr. Sekar Kathiresan, says The therapy will be relevant, we think, to any adult at risk of a heart attack.We want this not only for people who have heart attacks at a young age because of a genetic disorder, but for garden variety heart attacks as well.

Kathiresan resigned positions at Harvard Medical Schooland the Center for Genomic Medicine at Massachusetts general hospital to create Verve Technologies.We really think we can turn the tide against coronary disease by moving from a chronic care model to [eradication with] a one-time treatment, he toldThe Guardian.Currently, blood thinners, statins to lower cholesterol levels and high blood pressure medications are the standard treatments for individuals with a risk of heart disease.But seniors in particular can be overwhelmed by the treatments and may stop complying with their doctors orders.Or they may simply forget to take their meds.

Phase one of field tests of Verves procedure showed promise, according to aBloomberg report.Monkeys were treated and within six months their cholesterol levels were reduced by 59%.It will be months before human testing will begin and years before Verve might have sufficient proof that the process is safe and that it works in order to seek government approval of its use.

Ultimately, says Bloomberg, the company will face plenty of hurdles in its attempt to treat the masses.Its one of the firsttestsof using Crispr [a gene editing tool] to edit DNAinside the human body, and patients and doctors could be wary of making a permanent change without knowing much about long-term safety, saysElizabeth McNally, director of the Center for Genetic Medicine at the Northwestern University Feinberg School of Medicine.McNally also pointed out that the reluctance to take Covid-19 vaccines suggests some people may be loath to alter their DNA.

And then there is the matter of cost.Effective cholesterol medications are available for as little as nine dollars a month while Verves therapy will cost from $50,000 to $200,000 per patient, according to Bloomberg.

###

See the original post here:
Will we be able to check cholesterol using gene editing technology or will it be too dangerous and costly to even try? - L'Observateur - L'Observateur