Category Archives: Biotechnology

SAN FRANCISCO BAY AREA, Calif., Sept. 7, 2022 /PRNewswire/ -- Rondo Therapeutics, a privately-held biotechnology company advancing next-generation immuno-oncology platforms, announces the addition of Carolyn Bertozzi, Ph.D., to its Scientific Advisory Board. Dr. Bertozzi is a Professor of Chemistry and Director of the Sarafan ChEM-H institute at Stanford Universityand is a world leader in the field of glycobiology, generating new insights regarding disease-associated changes in cell surface glycosylation patterns. Jessica Stark, Ph.D., an American Cancer Society postdoctoral fellow in the Bertozzi Research Group, is also joining Rondo as a scientific consultant. Dr. Stark has pioneered new approaches in understanding the role of glycobiology in the functioning of the innate immune system.

Dr. Bertozzi has been recognized with many honors and awards for both her research and teaching accomplishments. She is an elected member of the National Academy of Sciences, the American Academy of Arts and Sciences, and the German Academy of Sciences Leopoldina. Recent awards of note include the Welch Award in Chemistry (2022); The Dickson Prize in Medicine, University of Pittsburgh (2022); Dr H.P. Heineken Prize for Biochemistry and Biophysics (2022); Wolf Prize (2022); AAAS Lifetime Mentor Award (2022). Dr. Bertozzi is also an accomplished entrepreneur, having founded several biotech companies including Palleon Pharma and Lycia Therapeutics.

"We are excited and privileged to be working with both Carolyn and Jessica in an exciting new area of biology," said Nathan Trinklein, Ph.D., co-founder and President of Rondo Therapeutics. "By combining their deep biological knowledge with Rondo's unique ability to rapidly develop bispecific antibodies, we are aiming to create a new class of biologics with a unique mechanism of action."

"We are thrilled to be working with the Rondo team to take on glycobiology as an emerging frontier for immunotherapy," said Jessica Stark, Ph.D., American Cancer Society postdoctoral fellow at Stanford University. "Through development of a novel class of bispecifics, we hope to make a difference for patients in multiple solid tumor indications."

Dr. Bertozzi is joining a group of highly distinguished scientists and clinicians on Rondo's Scientific Advisory Board: James Ford, M.D., Professor of Medicine, Stanford University; Alessandra Cesano, CMO, ESSA Pharmaceuticals; and Leonard Presta, Ph.D., renowned Protein Engineer.

About Rondo Therapeutics

Rondo Therapeutics is a biopharmaceutical company exploring new frontiers in cancer therapy. Rondo is committed to advancing the field of immuno-oncology, with a focus on treating solid tumors that fail to respond to current therapies. Our solution is to create a new class of bispecific antibodies that safely engage the immune system to initiate and sustain a robust anti-tumor response and overcome the suppressive tumor microenvironment.

Learn more at: http://www.rondotx.com.

SOURCE Rondo Therapeutics

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Carolyn Bertozzi, Professor and Director of the Sarafan ChEM-H institute at Stanford University, Joins the Rondo Therapeutics Scientific Advisory...

Nearly half (44%) of top 50 biopharma companies are now involved in RNA work sparking new demand for tools purpose built for the full spectrum of RNA R&D

SAN FRANCISCO, Sept. 7, 2022 /PRNewswire/ -- Benchling, the R&D Cloud powering the biotechnology industry, today launched the industry's first full set of capabilities for designing, modeling, and studying both natural and chemically modified RNA in a single solution. As the biopharmaceutical industry increases its focus on developing RNA therapeutics, Benchling's new RNA solution is the first purpose-built offering on the market to help R&D organizations accelerate the delivery of drug candidates. Featuring an RNA-aware registry with visualization tools for oligo and mRNA design and the only tool with support for chemical modification of mRNA sequences, RNA biologists and chemists can now collaborate more efficiently, standardize their work across teams, and develop drug candidates faster.

Following the successful deployment of RNA technology in the Pfizer-BioNTech and Moderna Covid-19 vaccines, biopharma companies have quickly shifted their pipelines to uncover therapeutic opportunities with RNA and mRNA. RNA therapeutics include a diverse group of therapeutic molecules that range from mRNA vaccines to antisense oligos, RNA-based gene therapy, and guide RNA-based CRISPR complexes. The unique function of these molecules, combined with the ability to adjust key properties through chemical modification or conjugation, has opened up novel ways of treating and preventing diseases. Nearly half (44%) of top 50 global biopharma companies are involved in RNA work through their own pipelines, M&A, or strategic alliances, based on GlobalData research1. Venture capital has also invested $3 billion into RNA-based therapies in 2020 alone, according to Nature.

While RNA therapeutics hold tremendous potential to impact human health, the R&D tools to create, test, and develop these molecules have been severely limited. Traditional chemistry tools cannot model the intricacies of RNA biology, while biology tools ignore the chemistry involved with RNA modifications altogether. As a result, there has not been a single software solution to date that addresses the combined biological and chemical nature of RNA therapeutics. Scientists have had to contend with manual, low-throughput, and disconnected software to manage their R&D work. Meanwhile, 82% of the pipeline of disclosed RNA and nucleotide therapeutics is in the research or preclinical stage of development2.

"We're at a tipping point for RNA therapeutics, having figured out tough challenges like durability and stability, the industry is now looking for the right tools to accelerate RNA R&D," said Ashu Singhal, co-founder and president of Benchling. "Covid vaccines were really just the beginning - as we put more tools in the hands of scientists to help support their RNA therapeutics R&D, this field will really take off. With Benchling's new RNA capabilities, scientists can now design, develop, and test a new mRNA vaccine or RNA cancer treatment from start to finish on a single platform."

"RNA has nearly unlimited potential to cure a wide range of diseases. Benchling's full set of purpose-built tools for RNA therapy development is going to enable us to realize this potential at an even greater pace than before," said Francois Vigneault, co-founder and CEO of Shape Therapeutics, an RNA technology company advancing programmable medicine and a longtime Benchling customer. "We are excited to see Benchling's continued investment in RNA innovation, and appreciate their commitment to helping companies like ShapeTX accelerate next-generation biopharma solutions."

Benchling's complete RNA solution is available today, and scientists working in RNA and mRNA therapeutics, RNA-based gene therapy, and gene editing will benefit from:

Along with the general availability launch of the RNA full solution, Benchling is making additional new product features generally available, including:

Benchling's mission is to unlock the power of biotechnology by creating modern software for modern science that empowers R&D teams to focus more time on science (not their software)! To learn more about Benchling and these new capabilities, please visit the company's What's New page.

About BenchlingBenchling is the pioneer of the R&D Cloud, software that unlocks the power of biotechnology. More than 200,000 scientists at over 1,000 companies and 7,500 academic and research institutions globally have adopted the Benchling R&D Cloud to make breakthrough discoveries and bring the next generation of medicines, food, and materials to market faster. The Benchling R&D Cloud helps these organizations modernize their scientific processes and accelerate collaboration so they can convert the complexity of biology into world-changing results. For more, please visit Benchling.comor follow us on Twitter at @Benchling.

1GlobalData by Benchling, August 20222GlobalData by Benchling, August 2022

Contact: [emailprotected]

SOURCE Benchling

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Benchling Launches the First Complete Solution for RNA R&D to Accelerate mRNA and RNA Therapeutics Development - PR Newswire

NANJING, China, Sept. 7, 2022 /PRNewswire/ -- InxMed Co., Ltd, a clinical-stage biotechnology company dedicates to developing innovative therapies targeting drug resistance and metastasis for hard-to-treat solid tumors, today announced the dosing of the first patient in its Phase 2 pivotal studyevaluating IN10018, a highly potent and selective oral inhibitor of focal adhesion kinase (FAK), in combination with pegylated liposomal doxorubicin (PLD) in patients with platinum-resistant recurrent ovarian cancer (PROC).

The Phase 2 pivotal study is a randomized, double-blind, placebo-controlled trial investigating the efficacy and safety of IN10018 in combination with PLD in patients with PROC. A total of 168 PROC patients will be enrolled and randomized in a 2: 1 ratio to receive IN10018 in combination with PLD or placebo of IN10018 in combination with PLD treatment in a double-blind manner.

Progression-free survival (PFS) as assessed by blinded independent central review (BICR) will be used as primary efficacy endpoint and overall survival (OS) will be used as key secondary efficacy endpoint. Objective remission rate (ORR), overall disease control rate (DCR), duration of remission (DOR), etc. as assessed by BICR and investigator will be other secondary efficacy endpoints.

InxMed previously reported results from its Phase Ib study of IN10018 in combination with PLD on ASCO 2022, demonstrated promising antitumor efficacy and a well-controlled safety profile in the treatment of patients with PROC.

The Phase 2 pivotal study was initiated inAugust 2022and is expected to be completed in Q1 2024. The company also has completed a Type B Meeting discussion with U.S. Food and Drug Administration (FDA) and planned to submit US IND for a Phase 2 pivotal studyevaluating IN10018 in combination with PLD for PROC in US by September this year.

InxMed set up a global clinical development program for IN10018. Clinical trials currently underway in the US, China and Australia are designed for platinum-resistant recurrent ovarian cancer, NRAS mutant metastatic melanoma, triple-negative breast cancer, head and neck cancer, pancreatic cancer, and other solid tumors that are still lacking effective treatment.IN10018 received fast track designation from the US FDA in August 2021, and breakthrough designation from China National Medical Products Administration(NMPA) in April 2022 for the treatment of patients with platinum-resistant ovarian cancer.

About InxMed

InxMed is a clinical-stage biotech company established in the end of 2018. The company dedicates on developing innovative therapies targeting stroma microenvironment and solid tumor resistance and metastasis, especially new drug development on anti PD-1/PD-L1 treatment drug resistance. InxMed committees to building an efficient engine for clinical translational science and proof of concept platform driven by in-depth understanding of disease biology and being a China based biotech company with global impact. We have built translational medicine and clinical development team across Shanghai, Beijing, Nanjing, United States, Canada and Australia. We have built a highly differentiated pipeline and established partnership with various multinational pharmaceutical companies including Merck, Roche and Boehringer Ingelheim.

SOURCE InxMed Co., Ltd

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InxMed Announces First Patient Dosed in Phase 2 Pivotal Study of IN10018 In Patients with Platinum-Resistant Recurrent Ovarian Cancer - PR Newswire

With significant investments being made into biotechnology and research and development (R&D), life science organizations are becoming increasingly profitable targets for cybercriminals. Tremendous strides are being made in terms of scientific discoveries and companies must also keep pace by managing security risks and protecting scientific data.

One cybersecurity report found that ransomware attacks a form of malware that locks users out of their devices or files until a ransom is paid increased by 485% in 2020 compared to 2019, likely influenced by the COVID-19 pandemic. Additionally, another report found that the average total cost of a data breach in the pharmaceutical industry was $5.06 million.

In light of these rising cybersecurity risks and the threats they pose, Technology Networks spoke to Zach Powers, chief information security officer at Benchling, an R&D cloud platform for the biotechnology industry. We discuss why the biotechnology industry is being targeted by cybercriminals, the importance of data security and how the industry can mitigate these threats.

Sarah Whelan (SW): Can you explain what the Benchling R&D Cloud is, and how it is designed to advance scientific research and development? How can this benefit small academic laboratories through to large-scale biotechnology companies?

Zach Powers (ZP): Benchling was started with a vision of making research and development what its meant to be a collaborative process to turn ideas into scientific progress. In the past few years with the pandemic, this vision has felt more urgent and important than ever. Now, more than 200,000 scientists use Benchlings R&D Cloud as a central source of truth for biotech R&D to centralize data, improve collaboration and access insights, ultimately accelerating the path to discovery.

Looking to examples of how our R&D Cloud facilitates progress in the scientific community we helped Syngenta go from data silos to data as an advantage, now with a data infrastructure that serves 90 locations across different languages, regulations and time zones in their mission to build crops that require fewer inputs while producing great outputs. Using Benchling, Syngenta reported a 72% improvement in sharing data across geos and a large team.

SW: What considerations need to be made in terms of data security for these types of cloud-based platforms?

ZP: Biotech organizations generate revenue based on intellectual property, and if compromised, a great deal of revenue stands to be lost. These organizations are also highly regulated due to the potential human impact of their products and complying with regulations can make or break the organizations ability to compete.

Both of these factors mean that for a cloud-based platform like Benchling, maintaining industry-leading security, privacy and compliance standards for biotech customers is paramount. Enterprise software as a service (SaaS) companies have a responsibility to develop cloud software and infrastructure securely. To do this, they use automated vulnerability management, routine penetration testing, asset management, configuration management, threat detection and response engineering, etc. The end result is that many cloud software products undergo more security scrutiny, on a more frequent basis, than on-premises technologies do. Not all cloud products are the same when it comes to security, but it is becoming increasingly common for enterprise SaaS companies to approach security in this way. When evaluating cloud platforms, customers should evaluate how much an enterprise SaaS company invests in security on an ongoing basis; is there an economy of scale on security that the customer can benefit from?

SW: How important is data security and governance to the industry, and how has this changed over the years as new discoveries are made and biotechnology becomes a more lucrative target for cybercriminals?

ZP: In recent years, threat actors have become more advanced and are highly funded, educated and organized businesses. Whats more, the most dangerous threat actors are being employed by adversaries of the USA and European Union. These organizations are in business to make profits, and many even have revenue targets. They aim to gain illegal access to some of the worlds most sensitive intellectual property for financial gain.

Pharmaceutical companies are now routinely targeted and attacked by these advanced threat actors, and in 2021 almost all (98%) of pharmaceutical companies experienced at least one security intrusion. In fact, over 20% of businesses have lost business-critical data or intellectual property in the last year alone.

It is clear that robust data security and governance are more important than ever, especially as the biotechnology industry continues to increase in value with the influx of valuable data it generates.

SW: What lessons do you think life sciences and biotechnology institutions can take from other industries regarding managing security risks?

ZP: Managing security risks appropriately today requires engineering, automation, real-time analytics, threat intelligence, significant tooling etc. It also takes a strategy of applying security throughout an organization, with multiple layers of defense, points of detection and built-in response options. This level of investment can seem daunting, but against adversaries who are well funded and are singularly focused on their targets, doing less only makes it easier for a threat actor to accomplish their goals. In the security industry, we often talk about the cost to the attacker and how appropriately investing in security can raise the cost sufficiently to either deter an attacker or slow down their attacks sufficiently for detection mechanisms to trigger and response plans to be executed. Threat actors consider the cost to carry out an attack; it is a business after all. Biotech institutions have the ability to influence that cost model.

When evaluating whether to invest in security at this level, many life sciences and biotech institutions have sticker shock as the cost of security is rising rapidly year over year. The advice I give biotech institutions is to look at how many other industries have taken advantage of the economies of scale that mature cloud computing companies can offer on security, resiliency, disaster response and more. If a biotech institution is not ready to invest materially in security themselves, building out the type of world-class security program and capabilities necessary to protect data today, then they can still get secure outcomes by moving their data and workflows into cloud platforms that have invested materially in security. More times than not, mature cloud platforms have invested orders of magnitude more in security than their customers do and continue to on an ongoing basis. No security strategy is perfect, but a strategy that takes advantage of the economies of scale on security that mature cloud platforms provide tends to fare far better than not.

There is another fundamental benefit to approaching security in this way. The adoption of a cloud-first strategy can significantly increase a biotech institutions data liquidity. Cloud architectures excel at enabling data to be found, to be accessed by those who need it, be interoperable between disparate systems and to be reusable. These are known as the FAIR data principles. It is a key focus for biotech institutions today, which have struggled with data residing in disparate, on-premises silos for years.

We can again draw lessons from many other industries, looking at how they evolved and profited from greater data liquidity. For example, enterprise SaaS, banking and healthcare each came to view cloud computing and more modern security as keys to unlocking data liquidity, supporting rapid growth and unparalleled innovation. If data liquidity is the destination, then the easiest road to take is via cloud computing and data platforms. Cloud computing and data platforms bring consistency in data modeling, easily allow for programmatic interfaces, allow for easier governance and security assurance and allow people to find, access and use data readily.

SW: What changes do you think are needed in the future to ensure data security as science advances? What are the biggest challenges that need to be addressed?

ZP: One of the biggest challenges I see is a distrust in cloud technology, which is, unfortunately, a more common sentiment in biotech, particularly in Europe. A lot of biotech institutions are still adhering to a security strategy from the late 1990s, using on-premises technology and essentially using firewalls as the first and only line of defense. More times than not, maintaining an on-premises strategy exposes you to more risk because 100% of the security responsibility and resourcing is on you. Most companies that distrust cloud computing are actually less secure than the cloud providers they distrust.

There are many myths about whether or not cloud computing is secure and its important to separate fact from fiction. When we look at breach statistics, nothing in the data says that on-premises technologies are more secure. But beyond taking a data-driven approach to making security decisions, the most important lens I can offer to change attitudes around the security of cloud computing is that of economies of scale. Companies that adopt cloud and enterprise SaaS take advantage of economies of scale on security that modern software companies provide. Enterprise SaaS companies have a responsibility for security, and they have security capabilities and teams beyond what most companies can afford.

Its the same with Benchling, security is an integral part of the product were offering to our customers. To this aim, we invest far more in security than most customers can afford to, and we have an abundance of expertise. Benchling embeds security engineering into our software development lifecycle and cloud infrastructure operations. Vulnerability testing happens daily, all code checked into production undergoes security testing and any security issues found are fixed within industry-leading service level agreements.

Biotech institutions can get a more secure outcome by taking advantage of cloud software and platforms. We take care of the hard stuff in security so that our customers can focus on advancing science and delivering humanity-impacting products.

Zach Powers was speaking to Sarah Whelan, Science Writer for Technology Networks.

Link:
Tackling Cybersecurity Threats in the Biotechnology Industry - Technology Networks

ISLAMABAD Three-day Pakistan-Turkey-Kazakhstan Youth Forum on Biotechnology will be arranged in the month of September with an emphasis on increasing the share of Muslim countries in the biotechnology global market.

The forum, to be held from September 13-15, is being sponsored by COMSTECH-the OIC Standing Committee for Scientific and Technological Cooperation, Islamic Organization for Food Security (IOFS) and Islamic Cooperation Youth Forum (ICYF), an official of COMSTECH told APP.

The academics, students and researchers from the OIC countries are likely to participate in the forum.

The global biotechnology market is anticipated to reach a market value of US$ 775 billion by 2024 with an annual growth rate of 7.7%.

Increased spending on biotechnology research and development, favourable initiatives by governments, global food security and the increasing need for emerging technologies are some of the factors influencing this growth.

Unfortunately, the share of Muslim countries in this global market of biotechnology is very small and needs to be increased.

This proposed tripartite youth forum (Pakistan-Turkey-Kazakhstan) will help promote excellence and competence in the field of agricultural biotechnology among Muslim countries. The theme of the forum Agriculture Biotechnology aims at providing the best knowledge and resources to young participants to advance their research goals, the official informed.

The youth forum will stimulate the desire to collaborate and change the world of agricultural biotechnology and innovation by promoting state-of-the-art practices in biotechnology research and promoting evidence-based practices. The event will include informative talks from young as well as experienced scientists from the three countries.

The event will also include keynote lectures, plenary sessions, oral and poster presentations, discussions and other educational and social events that stimulate several networking opportunities among the young participants of Pakistan, Turkey and Kazakhstan.

The official highlighted that the forum aims to facilitate interactions within the young research community to discuss the latest developments in this rapidly advancing field and find ways to respond to the increasing demands of professionals and communities across the world.

This youth forum is planned to be a hybrid event depending on the preference of the participants in their submitted registration forms.

The topics to be covered during the forum include Genome editing and new breeding technologies, large-scale genomics and genomic selection in crop and livestock breeding, speed breeding for rapid genetic gain, variants of Cas proteins and their potential applications and high throughput phenomics.

See more here:
Pak-Turk-Kazakh Youth Forum on Biotech to be held in Sept - The Nation

Citizens vague sense of unease about genetically modified (GM) crops and genome-edited foods will never be alleviated. Why?The main reason for this is likely to be the structure of reporting that emphasizes citizens and the behavioral principles of reporters.However, with the advent of the Internet, which allows anyone to send information, this structure is beginning to shake.

I would like to give an example of an article that leaves an impression of anxiety.An article in the Mainichi Shimbun dated April 20, 2019, headlinedStop genome-edited puffer fish. Miyazu hometown tax return gift Co-op Natural Kyoto Requests City.

Follow the latest news and policy debates on agricultural biotech and biomedicine? Subscribe to our newsletter.

It would be good to report the movements of the citizens movement itself.The problem is inside.In the article,

The safety of genome-edited food has not been confirmed

What kind of impact will it have if we continue to eat genome-edited food? Insufficient verification

I feel uncomfortable with Miyazus wonderful seafood and genome-edited 22nd century blowfish'

No one in the world knows whether genome-edited food is safe. Moreover, there is no labeling obligation. This is the current situation, and as a parent with a child, I have no worries.

In short, the article is filled with only the voices of the opposition.

This is an excerpt. Read the original post here

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Viewpoint: GMO and gene edited agricultural solutions will never gain traction if journalists continue to carry water for anti-biotechnology...

DUBLIN--(BUSINESS WIRE)--The "Global Cleanroom Technologies Market with COVID-19 Impact, by Product (Fan Filter Units, HVAC, vacuum systems, disinfectants), Construction (Drywall, Hardwall, Softwall), End User (Pharmaceutical, Biotechnology, Medical device manufacturers) - Forecast to 2026" report has been added to ResearchAndMarkets.com's offering.

The cleanroom technologies market is projected to reach USD 9.6 billion by 2026 from USD 7.1 billion in 2021, at a CAGR of 6.2% during the forecast period. The growth of this market is majorly driven by the stringent regulatory framework, growth of the biologics sector, rising demand for sterilized pharmaceutical formulations, increasing demand for medical devices, and technological advancements in cleanroom technology.

Also, the increasing demand in developing economies and the growing focus on energy-efficient cleanrooms are expected to offer significant opportunities for market growth in the coming years. However, the high operational cost associated with the cleanrooms is expected to restrain market growth to a certain extent.

Based on product, the consumables segment holds the largest market share during the forecast period

Based on product, the cleanroom technologies market is segmented into consumables, equipment, and controls. The consumables segment accounted for the larger market share in 2020. The high and growing number of pharmaceutical, biotech, and medical device companies facilitating the use of disposable protective clothing has resulted in the increased adoption of the consumables in the cleanroom technologies market. Also, the large number of R&D activities in the healthcare industry is resulting in a stable demand for cleanroom consumables among end users.

Based on construction type, the hardwall cleanrooms segment is expected to register the highest CAGR during the forecast period

Based on construction type, the cleanroom technologies market is segmented into standard/drywall cleanrooms, hardwall cleanrooms, softwall cleanrooms, and pass-through cabinets. The hardwall cleanrooms segment is expected to witness the highest growth during the forecast period. This is mainly due to the higher demand for hardwall cleanrooms, as they are more design-flexible than standard and softwall cleanrooms, quick and easy to install, freestanding for easy portability, and easy to expand or reconfigure.

The pharmaceutical industry end-user segment accounted for the largest share of the cleanroom technologies market in 2020

Based on end user, the cleanroom technologies market is segmented into the pharmaceutical industry, biotechnology industry, medical device manufacturers, hospitals, and other end users. The pharmaceutical industry end-user segment accounted for the largest share of the market in 2020. This is attributed to the industrial growth in this sector, the robust pipeline of injectable formulations, and rising focus on ensuring the quality of healthcare products.

Asia Pacific is expected to grow at the highest CAGR in the cleanroom technologies market during the forecast period

The cleanroom technologies market in the Asia Pacific is estimated to grow at the highest CAGR during the forecast period. This is due to its favorable government regulations, increasing healthcare expenditure, and the growing base of pharma companies in the country, all of which are driving the adoption of cleanroom solutions in the Asia Pacific.

Market Dynamics

Market Drivers

Market Restraints

Market Challenges

Industry Trends

Key Topics Covered:

1 Introduction

2 Research Methodology

3 Executive Summary

4 Premium Insights

5 Market Overview

6 Cleanroom Technologies Market, by Product

7 Cleanroom Technologies Market, by Construction Type

8 Cleanroom Technologies Market, by End-user

9 Cleanroom Technologies Market, by Region

10 Competitive Landscape

11 Company Profiles

12 Appendix

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/qfd326

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Global Cleanroom Technologies Market Report 2022: Stringent Regulatory Framework in the Pharmaceutical and Biotechnology Industries is One of the Key...

AUSTIN, Texas, Aug. 18, 2022 /PRNewswire/ -- Aeglea BioTherapeutics, Inc. (NASDAQ:AGLE), a clinical-stage biotechnology company developing a new generation of human enzyme therapeutics to benefit people with rare metabolic diseases, today announced that a Marketing Authorization Application (MAA) for pegzilarginase for the treatment of Arginase 1 Deficiency (ARG1-D) has been submitted to and successfully validated by the European Medicines Agency (EMA). The MAA was submitted by Immedica Pharma AB, Aeglea's commercialization partner in Europe and the Middle East.

Pegzilarginase is a novel, recombinant human arginase 1 enzyme that in clinical trials has been shown to normalize the elevated levels of the amino acid arginine in patients with ARG1-D, a rare, progressive disease characterized by high levels of arginine. People living with ARG1-D experience severe spasticity-related mobility limitations, seizures, developmental delay, intellectual disability, and early mortality.

"For progressive diseases like ARG1-D, every day that passes without an approved therapy increases the disease burden and worsens the outcome for patients and their families. If approved, we believe pegzilarginase has the potential to benefit these families," said Anthony G. Quinn, M.B., Ch.B., Ph.D., president and chief executive officer of Aeglea. "We are thrilled with the progress being made with the validation of the MAA and look forward to continuing to work withand support our partner Immedica throughout the EMA review process."

Dr. Quinn continued, "I would like to thank all the patients, families, investigators, staff and advocates who participated in our clinical trials and helped in the advancement of pegzilarginase, potentially the first health authority-approved treatment to address the underlying driver of ARG1-D, elevated arginine levels."

Immedica's MAA submission includes data from multiple clinical studies in ARG1-D, including the double-blind, placebo-controlled PEACE Phase 3 study and its ongoing long-term extension, a Phase 1/2 clinical trial and an open-label extensionstudy. Results from these trials demonstrate that pegzilarginase is able to rapidly and sustainably lower arginine levels and showed improvements in measures of mobility. In the PEACE study, most treatment-emergent adverse events were mild or moderate in severity and there were no discontinuations due to treatment-emergent adverse events. The EMA has granted pegzilarginase Orphan Drug Designation.

About Pegzilarginase in Arginase 1 Deficiency

Pegzilarginase is a novel recombinant human enzyme engineered to degrade the amino acid arginine and has been shown to rapidly and sustainably lower levels of the amino acid arginine in plasma. Aeglea is developing pegzilarginase for the treatment of people with Arginase 1 Deficiency (ARG1-D), a rare debilitating and progressive disease characterized by the accumulation of arginine. ARG1-D presents in early childhood and patients experience spasticity, seizures, developmental delay, intellectual disability and early mortality.

The PEACE Phase 3 clinical trial met its primary endpoint with a 76.7% reduction in mean plasma arginine compared to placebo. Additionally, 90.5% of pegzilarginase treated patients achieved normal plasma arginine levels. The arginine lowering was accompanied by a positive trend in Gross Motor Function Measure Part E, a measure of patient mobility. Aeglea's Phase 1/2 and Phase 2 Open-Label Extension (OLE) data for pegzilarginase in patients with ARG1-D demonstrated clinical improvements and sustained lowering of plasma arginine. Pegzilarginase has received multiple regulatory designations, including Rare Pediatric Disease, Breakthrough Therapy, Fast Track and Orphan Drug designations from the U.S. Food and Drug Administration as well as Orphan Drug Designation from the European Medicines Agency.

About Aeglea BioTherapeutics

Aeglea BioTherapeutics is a clinical-stage biotechnology company redefining the potential of human enzyme therapeutics to benefit people with rare metabolic diseases with limited treatment options. Pegzilarginase achieved the primary endpoint of arginine reduction in the PEACE Phase 3 clinical trial and has received both Rare Pediatric Disease and Breakthrough Therapy Designations. Aeglea also has an ongoing Phase 1/2 clinical trial of AGLE-177 for the treatment of Homocystinuria. AGLE-177 has been granted Rare Pediatric Disease Designation. Aeglea has an active discovery platform focused on engineering small changes in human enzymes to have a big impact on the lives of patients and their families. For more information, please visit http://aeglea.com.

Safe Harbor / Forward Looking Statements

This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, statements we make regarding our ability to obtain regulatory approval for, and commercialize, pegzilarginase, recognize milestone and royalty payments from our agreement with Immedica, the timing and success of our clinical trials and related data, the timing and expectations for regulatory submissions and approvals, including the MAA for pegzilarginase in Europe, timing and results of meetings with regulators, the timing of announcements and updates relating to our clinical trials and related data, our ability to enroll patients into our clinical trials, the expected impact of the COVID-19 pandemic on our operations and clinical trials, success in our collaborations, the length of time that we believe our existing cash resources will fund operations, the potential addressable markets of our product candidates and the potential therapeutic benefits and economic value of our lead product candidate or other product candidates. Further information on potential risk factors that could affect our business and its financial results are detailed in our most recent Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 filed with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

SOURCE Aeglea BioTherapeutics, Inc.

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Aeglea BioTherapeutics Announces European Medicines Agency Validation of Marketing Authorization Application for Pegzilarginase for the Treatment of...

Colossal, a Texas-based biotechnology 'de-extinction' company, will partner with University of Melbourne to attempt to recreate the thylacine by using gene-editing technology

A file image of the Tasmanian tiger. Imag courtesy: Wikimedia Commons/ Report of the Smithsonian Institution/Smithsonian Institution archives

Scientists in the US and Australia have announced a plan to bring back the thylacine, also known as the Tasmanian tiger, which went extinct around ninety years ago.

This is the second announced animal de-extinction project from Colossal, which uses breakthrough gene-editing technologies for a new wave of wildlife and ecosystem conservation.

The company previously announced its plans to de-extinct the woolly mammoth and restore the keystone species to the Arctic Tundra last September.

Colossal, the Texas-based biotechnology de-extinction company, will partner with University of Melbourne to attempt to recreate the thylacine.

Lets take a look at what you need to know:

What were Tasmanian tigers?

The Tasmanian tiger was a slim, striped keystone species native to Australia, including Tasmania and New Guinea.

Dog-like in appearance and with stripes across its back, it was extensively hunted after European colonisation.

As per The Guardian, the thylacine was Australias only marsupial apex predator.

It had roamed the Earth for millions of years before humans hunted it to extinction.

As per the Scientific American, European colonizers in Tasmania in the 1800s and early 1900s wrongly blamed Tasmanian tigers for killing their sheep and chickens.

The settlers slaughtered thylacines by the thousands, exchanging the animals skins for a government bounty.

As per CNET, the last Tasmanian tiger in the wild was killed on 13 May, 1930.

The last known Tasmanian tiger, Benjamin, died in captivity at the Hobart Zoo in 1936.

Whats the plan to bring them back?

The plan is to use gene-editing technology.

As per CNET, the task begins with decoding thylacine DNA.

Thankfully, this was accomplished in 2017 and researchers now have the "recipe" or "blueprint" needed to engineer it.

The next step is to extract cells from a close living relative from a fat-tailed dunnart, a mouse-like marsupial that could fit in the palm of your hand for example of which they have the DNA blueprint.

Thepremiseis to identify all the differences between the dunnart DNA and the thylacine DNA. This is an active area of research requiring a ton of computing power and bioinformatics.

But suppose they can pinpoint those differences; they will then take cells from the dunnart and, using the gene-editing tool CRISPR, build a thylacine cell.

How will Australia benefit?

The company claims bringing back the thylacine will not only return the iconic species to the world, but has the potential to re-balance the Tasmanian and broader Australian ecosystems, which have suffered biodiversity loss and ecosystem degradation since the loss of the predator earlier this century.

What do experts say?

But others remain unconvinced.

Mammal expert Kris Helgen of the Australian Museum, who worked on sequencing the thylacines mitochondrial genome in 2009, told Scientific American altering the dunnarts DNA to truly resemble a thylacines will be an impossible feat and that the two species are separated by as much as 40 million years of evolution.

The idea that science could restore the thylacine is just so lovely it captures the imagination, says Helgen.

But the thylacine is extinct in Australia and in Tasmania, and theres no way to bring it back. He added that some species are gone forever due to their uniqueness.

A few million dollars [are] not going to give us an escape hatch from extinction, he added.

Hugh Possingham, a conservation biologist at the University of Queensland, told CNET: "If funding de-extinction reduces investment in saving the species we have, then it doesn't make sense," he says. "If funding de-extinction does not compromise conservation funding, then it is an intriguing but high-risk activity."

Some were even more blunt.

Its better to spend the money on the living than the dead, lead author Joseph Bennett of Carleton University in Ontario told Science.

With inputs from agencies

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How scientists are planning to revive the Tasmanian tiger that's been extinct since 1936 - Firstpost

While it may seem valuations are likely to stop falling at this level, this is not necessarily the case in biotech. Typically, these early-stage companies are yet to have a product approved, and they subsequently rely heavily on external backing to fund the development of new treatments.

This makes these sorts of companies particularly risky prospects for investors in the biotech space. However, among companies whose values have dropped 70-80% from their peak levels in the spring of 2021 are some gems, with compelling science and sound underlying business fundamentals.

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Given the darkening macroeconomic outlook, identifying these diamonds in the rough requires an in-depth understanding and analysis of the funding outlook for early-stage biotech businesses.

Banks generally do not lend to development stage biotech companies, as the nature of their business is deemed too risky and clinical trials are far from cheap. The majority of funds are raised through private series rounds, IPOs, and future secondary rounds, as this type of equity funding is cheaper and has fewer strings attached. It also generally means companies can retain control of their assets.

In recent years, it was relatively easy for early-stage biotech companies to raise money.

The IPO market was booming, and many firms took the opportunity to raise excess capital while the market was hot. This was a sensible approach, as things can turn very rapidly and funding can dry up fast, as has been proven in recent months.

Investor appetite for biotech IPOs has now dried up, and many biotech companies fear not being able to raise equity finance, or at best, with stock prices at current levels, being forced to raise money at low valuations, thereby diluting current shareholders.

Companies with limited access to equity finance are faced with a tough decision. It is considered unethical to halt a clinical trial, but some companies choose to stop or delay pre-trial clinical programmes to preserve capital. If there are doubts on the efficacy of their lead asset, they may start to explore potentially riskier second tier assets, burning yet more cash.

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Companies may also seek to partner their assets or regional rights with larger biotech or pharmaceutical companies. While this means losing a proportion of the potential value, it provides them with cash up front and extends their runway, enabling them to continue developing treatments. Others may sell out in full, as these situations are a magnet for larger companies seeking to pick up distressed assets at knock down valuations.

Companies resorting to option financing such as convertible notes or at-the-money instruments tend to be in a fairly desperate financing position, as these are generally considered the most expensive and least attractive sources of finance.

With early stage funding options dwindling, investors in the biotech space may be tempted to avoid riskier development stage businesses and opt instead for larger-cap stocks with profitable products already on the market.

However, this could mean foregoing potentially significant returns. The earlier stage biotech firms boast the most exciting new cutting-edge therapies with the potential to form the next generation of medicine.

Investing in companies trading at an EV at or below zero takes great discipline, experience, and cautiousness. One of the key metrics to evaluate is a company's cash burn rate', which refers to the future cost of programmes the business is committed to, versus the time it will take the firm to reach a clinical trial read out.

This stage is critical for early-stage biotech players, as reporting positive drug trial results boosts the chances of a successful capital raise at a higher valuation. Avoiding companies that will need refinancing within the next two years is a good rule of thumb to follow.

There are other key factors to consider. The management team's quality and experience are also vital, as investors must be satisfied the team can withstand tough market conditions and present an attractive proposition to potential funders.

Additionally, the ownership of the company is important. If a company is supported by professional investors, known for their skillsets and deep pockets', risk is considerably mitigated.

Even if a company is sufficiently well financed and managed, the underlying science behind its innovations must be of top quality. Our approach with these higher-risk assets is to build a small toe in the water' position to start with, and add to it incrementally as the company proves itself and our relationship with the management team deepens.

Marek Poszepczynski and Ailsa Craig are co-investment managers of the International Biotechnology Trust

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How to identify biotech diamonds in the rough - Investment Week