Monthly Archives: July 2021

MILAN, July 20, 2021 /PRNewswire/ --Panaks Partners, the leading Italian venture capital firm in the Life Sciences sector, announces the first closing of its 150 million ($180 million) Purple Fund, the firm's second fund.

Panaks' Purple Fund is currently the largest venture capital fund actively investing in Italian companies and the most significant fund dedicated wholly to the Life Sciences sector in Italy. The fund will invest in companies at the forefront of innovation, with a focus on Europe, and Italy in particular, which remains underserved in terms of Venture Capital funding.

The Purple Fund is the second venture capital fund dedicated to life sciences launched by Panaks Partners. Panaks' first fund, raised in 2016, supported companies in the medtech sector. To-date it has invested in 12 portfolio companies, which have collectively received almost 200 million in funding. Thanks to this financial support, these companies have already brought five innovative medical products to the market and have a further ten products in active clinical trials.

The Purple Fund has been backed by investors from the first fund as well as new investors. The Fund's two anchor investors are EIF and the Fund of Funds FoF VenturItaly managed by CDP Venture Capital SGR. The EIF investment is backed under both the InnovFin Equity initiative from the European Commission under Horizon 2020, the Framework Programme for Research and Innovation, as well as the pan-European Guarantee Fund (EGF).

These anchor investors have been joined by several Italian banking foundations and pension funds, as well as numerous Italian companies and family offices in the Life Sciences sector. These include Menarini, the Cogliati family (Elemaster Group), the Colombo family (SAPIO Group), the Rovati family (Rottapharm Biotech), the Petrone family (Petrone Group), the Re family (Digitec Group), the Bassani family (Movi Group) and others.

The Purple Fund will invest mainly in Series A funding rounds, as well as later stage opportunities. The majority of investments will be in companies developing innovative therapeutics and products in the fields of biotechnology, diagnostics, and medical devices.

The fund aims to support the growth of entrepreneurial companies who will reshape healthcare globally by addressing real medical needs, saving lives and providing a better quality of life for patients. By achieving these goals, the fund aims to generate value for both investors and for society as a whole.

"We are delighted with the successful first close of our new Purple Fund, and we would like to thank the high-quality investors who have trusted us. Over 500 innovative life science companies have already submitted funding requests to us in the first six months of 2021," said Fabrizio Landi, President of Panaks and a founding partner of the firm alongside Diana Saraceni and Alessio Beverina. "The fund will remain open for additional subscribers until the end of the year, with a new target of 180 million. By expanding into the biotech sector, we hope to contribute to the growth of companies active in the development of new therapies and vaccines," concluded Landi.

"Panaks has established a strong track record and solid international credibility since it was created a few years ago, also with the support of the CDP Group," commented Enrico Resmini, Chief Executive Officer of CDP Venture Capital SGR. "We are delighted to invest in Panaks' second fund, as it extends its activity into biotechnology, a sector where long-term planning and the availability of capital is essential to finance the R&D that is expected to lead to the innovative new therapies of tomorrow."

Alain Godard, Chief Executive of the European Investment Fund (EIF/FEI), added: "We are happy to once again support Panaks after our previous investment in its first fund. Panaks has managed to build a strong brand in Italy and beyond thanks to its expertise in identifying and investing in novel medtech opportunities. With the extension of its investment strategy into biotech and the resulting growth of the team, Panaks will be able to further support European Life Sciences companies, and particularly those in Italy, which have exceptional R&D but are strongly underserved in terms of Venture Capital funding. We are glad to be able to use both the InnovFin mandate from the European Commission and the direct backing of EU Member States under the European Guarantee Fund to further support this exciting market segment."

To support its expansion into the biotech sector, Panaks intends to recruit three new professionals with significant experience in drug discovery and development in the pharmaceutical industry to its existing team, which is currently made up of 11 professionals. Recently, Barbara Castellano has been promoted to the role of Partner, while the management team of the SGR has been strengthened with the arrival of a new CFO, Lorenzo Giordano, and a Financial Assistant, in the person of Andrea Steffanini.

Panaks Advisory Board has also been expanded and strengthened with the appointment of Biotech and Digital Health industry experts Fabio Pammolli, Professor of Economics, Finance, and Management Science at Politecnico di Milano, and Sergio Abrignani M.D. Ph.D. Full Professor at the National Institute of Molecular Genetics (INGM) in Milan.

About Panaks

Panaks Partners is a Milan-based venture capital firm that aims to improve the lives of people around the world by providing the most promising companies and teams with the financial and corporate support needed to build the next generation of companies bringing revolutionary technologies and products to the field of life sciences. Panaks was founded in 2015 by Fabrizio Landi, Alessio Beverina and Diana Saraceni.

http://www.Panaks.it

About CDP Venture Capital SGR Fondo Nazionale Innovazione

CDP Venture Capital is an asset management company (70% owned by CDP Equity and 30% owned by Invitalia) with over 1 billion euro of assets under management. It aims to make Venture Capital a strategic pillar to Italy's economic development and innovation, creating the conditions for a comprehensive and sustainable growth of the Venture Capital ecosystem. It operates through a series of funds that aim to support startups in all their life cycle stages, making both direct and indirect investments.

About EIF

TheEuropean Investment Fund(EIF) is part of the European Investment Bank Group. Its central mission is to support Europe's micro, small and medium-sized businesses (SMEs) by helping them to access finance. EIF designs and develops venture and growth capital, guarantees and microfinance instruments which specifically target this market segment. In this role, EIF fosters EU objectives in support of innovation, research and development, entrepreneurship, growth, and employment. The EIF investment is supported by InnovFin Equity, with the financial backing of the European Union under Horizon 2020, the Framework Programme for Research and Innovation (2014-2020). Through or alongside selected Venture Capital (VC), Business Angels (BA), Technology Transfer funds and funds-of-funds, the EU provides risk capital financing to enterprises, research organisations, universities in their proof-of-concept, pre-seed, seed, start-up and other early-stage phases allowing them to set up or reach their next stage of development.The EIF participation is also backed under the European Guarantee Fund (EGF), which was set up by the EIB Group with contributions from Italy and other EU Member States to shield companies suffering from the COVID-19 crisis. Using nearly EUR 25 billion in guarantees, the EGF allows the EIB and the EIF to quickly make loans, guarantees, asset-backed securities, equity and other financial instruments available to mostly small and medium-sized enterprises. The EGF is part of the European Union's recovery package aiming to provide a total of EUR 540 billion boost those parts of the EU economy that have been hit the worst.

Media Contacts:

Panaks Partners

news@panakes.it

MEDiSTRAVA Consulting

Sylvie Berrebi, David Dible

panakes@medistrava.com

Tel: +44 (0)20 7638 9571

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Panaks Partners Announces the First Closing Of Its New 'Purple' Global Biotech/ Medtech Fund at 150 Million ($180 Million) - WFMZ Allentown

Health & Welfare

Monday, 19 July 2021 Silvia Garca-Ballesteros, Ph.D. Beatriz Villanueva, Ph.D. Jess Fernndez, Ph.D. Juan Pablo Gutirrez, DVM Isabel Cervantes, DVM

Most selective breeding programs for shrimp focus on improving growth traits only, but as growth rate increases and production intensifies, other traits related to the quality and uniformity of the final product gain importance for both consumers and producers, including size uniformity.

Shrimp are graded and classified according to standards that are defined in high-quality marketing evaluations and are mainly determined by their physical characteristics and uniformity of size. In particular, shrimp are graded according to their size and count per unit of weight. Prices between size categories vary widely and a larger number of smaller shrimp per unit weight reduces their price, so increasing the consistency of size within a specific count range can improve profitability.

In addition, large variation in body size can cause competition among shrimp (dominance hierarchies), which negatively affects growth rate, mortality and feed efficiency, and increases the need for management practices such as size grading. Another indirect benefit of improving uniformity is the potential to improve resilience, which is defined as the ability of an animal to maintain performance in spite of environmental perturbations. For all these reasons, and given that weight is genetically highly correlated with size, uniformity of weight is a clear candidate trait to be included in shrimp breeding programs.

Weight uniformity depends on the sensitivity of an individual to macro- and micro-environmental factors. Macroenvironmental factors are measurable factors such as temperature, seasonality, diet and management, whereas microenvironmental factors are non-measurable animal-specific factors within a given macroenvironment. A necessary condition to increase weight uniformity is the existence of genetic variance for response to such microenvironmental factors, such that individuals with genotypes [complete set of genetic material] that make them less sensitive to environmental disturbances will have more homogeneous offspring and show less environmental within-family variance.

In aquaculture selective breeding programs, the breeding nucleus (in which selection is performed) is usually kept separate from the commercial population that is composed of individuals destined for sale in the market. In aquaculture, the macroenvironmental rearing conditions can differ greatly between the nucleus and the commercial population. Thus, if genotype-by-environment interactions exist, genetic improvement achieved in the nucleus may not be fully translated to the commercial population.

This article summarized and adapted from the original publication [Garca-Ballesteros, S. et al. 2021. Genetic parameters for uniformity of harvest weight in Pacific white shrimp (Litopenaeus vannamei). Genet Sel Evol 53, 26 (2021)] reports on a research study to estimate the genetic variance of body weight uniformity in a farmed population of Pacific white shrimp (Litopenaeus vannamei), and to investigate whether selecting for increased weight uniformity in the breeding nucleus leads to improvement of uniformity in the commercial population.

Influence of stressors on shrimp susceptibility to White Spot Disease, Part 1

The data used in this study were obtained from CAMANICA S.A., a Nicaragua-based company, carrying out a breeding program in shrimp with discrete generations and selection for shrimp body weight. Once animals reach the appropriate size, random samples of individuals are tagged, with half of them being individually tagged with eye-rings and assigned to the nucleus (N) population and the other half being tagged at the family level with elastomers and assigned to the commercial (C) population.

Within the nucleus, all families are reared in the same tank. However, in the commercial population, three to four ponds that are located in different geographical zones are used per generation, with each family equally represented in each pond. Environmental conditions differ greatly between the nucleus and the commercial populations. Thus, weight in the selection nucleus and weight in the commercial population are considered as two different traits.

The data used here are from three consecutive generations and 425 families. The total number of individuals with phenotypic records for body weight at harvest was 89,643, of which 51,346 belonged to the nucleus and 38,297 belonged to the commercial population. Harvest time was established by estimating the days required to reach an average weight of 15 grams in the nucleus. This time was set for both commercial (all ponds) and for the nucleus environments. However, for management reasons, recording the phenotypes of all shrimp can take a few days. Sex, year and pond were also recorded.

The resulting database had records for body weight on 51,346 shrimp from the selection nucleus and 38,297 shrimp from the commercial population. We used a double hierarchical generalized linear model [used in genetics studies to model quantitative traits (a measurable phenotype the observable characteristics or traits of an organism from genetic and environmental factors spread in magnitude in a population rather than none or all) with respect to molecular marker effects (molecules containing genetic information from a sample) to analyze weight uniformity in the two environments. Fixed effects included sex and year for the nucleus data and sex and year-pond combination for the commercial data. Environmental and additive genetic effects were included as random effects.

For detailed information on the study data, parameters evaluated, and analyses, refer to the original publication.

Although weight uniformity is a very relevant trait with the potential of being included in shrimp breeding programs, there is very little information on the existence of genetic variation for this trait. To our knowledge, ours is the first study that uses a double hierarchical generalized linear model to estimate genetic variance for body weight uniformity in shrimp and constitutes a first step to investigate the possibility of including this trait in the breeding goal. This is important since the weight uniformity evaluated here was individual sensitivity to microenvironmental disturbances.

Estimates of the additive genetic variance, heritability and genetic coefficient of residual variation for weight uniformity that were obtained for this L. vannamei population in the nucleus, in which selection takes place, were all different from 0, which indicates that genetic improvement for this trait is possible. In addition, the genetic correlation of weight uniformity between the nucleus and the commercial population was relatively high, which indicates that improvement obtained in the nucleus would be partially transmitted to the commercial population, with the economic benefits that this would entail.

Results showed that our estimates of the global heritability for body weight at harvest in N and C were within the range of those found in the literature for shrimp. More important is the fact that estimates of the additive genetic variance for uniformity of weight and for the residual heritability were also in the range of those described for shrimp, other aquaculture species and various terrestrial species. This indicates the existence of genetic variation in microenvironmental sensitivity among full-sib families [common parents], which implies that the phenotypes of offspring of different families will be differentially affected by the environment. Thus, our results show that the potential of genetic selection to improve weight uniformity in L. vannamei is similar to that for other species.

To evaluate the potential economic benefit of including weight uniformity in the breeding goal, correlations with other traits that are currently in the breeding goal, such as body weight, must be estimated. The ideal scenario would be the existence of a negative genetic correlation between weight and its variability because it would facilitate selection for higher weight and more uniformity. In aquaculture, estimates of the genetic correlation between weight and its variability vary largely in the literature. Our estimate was not significantly different from 0, which indicates that it may not be difficult to improve weight and weight uniformity simultaneously through a selection index. This would require the economic value for uniformity to be determined, which is unknown at this point.

It is very important that genetic improvements made in the nucleus are transferred to the commercial population that is composed of individuals for sale in the market. Thus, a high genetic correlation between the nucleus and commercial environments for traits that are selected for in the nucleus is desirable. This is not always the case because, although conditions are intended to be similar in the two environments, this is not usually feasible. Particularly in aquaculture species, some environmental factors are more important than others in affecting the re-ranking of individuals based on their estimated breeding values.

In our study, estimates of the genetic correlation between environments N and C were for weight and for weight uniformity were within the range reported for weight in other aquaculture species. Our estimate of the genetic correlation of weight between environments was lower than what has been reported for shrimp by some researchers, but within the range reported by others. Our study provides, for the first time, an estimate of the genetic correlation of weight uniformity between different environments for shrimp, and it is similar to what has been reported for trout.

Many studies have shown that the proportion of phenotypic variance [variability in phenotypes in a population, including height, weight, body shape and others] due to common environmental effects, although significantly different from 0 in shrimp and other aquaculture species, is of low magnitude. Some studies have suggested that common environmental effects are difficult to separate from family genetic effects. The few studies that have included common environmental effects for weight uniformity did not achieve significant estimates.

Our results show that genetic variability for the environmental variance of weight at harvest exists in shrimp, both in the selection nucleus and in the commercial population. The genetic variation for these traits (uniformity measured in the nucleus and in the commercial population) was large enough to conclude that response to selection could be obtained if these traits were included in the breeding program.

Including weight uniformity should not decrease weight since the genetic correlation between the two traits was not significantly different from zero. Further investigation is necessary to determine what is the best combination of these traits to reach the greatest economic benefit. Based on the genetic correlation of weight uniformity between the two environments estimated here, selection in the nucleus will be transmitted to the commercial population.

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Estimating the genetic variance of body weight uniformity in a farmed population of Pacific white shrimp Global Aquaculture Advocate -...

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The figure above summarizes the findings of the team in terms of genetic, epigenetic and stochastic differences among the EGFR-mutant lung cancer cells studied.

Leonard Harris, assistant professor of biomedical engineering, led a team of researchers from Vanderbilt Universitythat has shown how an in vitro model of tumor heterogeneity, or diversity, resolves three different sources of cell state variability in cancer cells.

The paper has been published in PLOS Biology, part of the Public Library of Science.

A heterogeneous tumor is a tumor that is made up of many different types of cancer cells. Often, the cells have different types of genetic mutations and co-exist within a tumor. The diversity of the tumor is what makes cancer difficult to treat.

"It's like the success of a diverse team," Harris explains. "A team made up of people from different backgrounds, ages, stages of their career, etc., are often better at tackling problems because the team members provide different perspectives."

In a tumor, different cells respond to drug treatments differently. Some cells are able to survive and regrow the tumor and spread, which is why Harris and his team continue to research the ways surviving cancer cells differ from the other tumor cells.

But genetic mutations are not the only way cancer cells can differ from each other. Cells that have the exact same DNA can exist in very different states. For example, your skin cells and your liver cells have exactly the same DNA but they function very differently; that is an example of epigenetic heterogeneity. Moreover, when a skin cell divides, it produces two skin cells. The cells do not inherit the skin cell state from the DNA; it has to come through some other means. It is this non-genetic form of inheritance that makes the process epigenetic.

Cancer cells also differ due to random fluctuations in molecule numbers inside each cell: molecules randomly interact with each other, degrade, are synthesized by the cell, secrete into and out of the cell, etc. This type of non-genetic heterogeneity is called stochastic variability and is not heritable, unlike epigenetic processes. It might not seem like a big deal, but researchers have shown that stochastic variability can have major effects.

The experimental and computational work reported in the paper was performed at Vanderbilt University in collaboration with Corey E. Hayford, Darren R. Tyson, C. Jack Robbins III, Peter L. Frick and Vito Quaranta and has motivated many additional research projects. It is now the foundation for Harris' U of A laboratory.

"Cancer is commonly referred to as a 'genetic disease', meaning it is caused by mutations in critical parts of the DNA that cause cells to grow out of control," Harris said. "This has led to decades of research on the genetics of cancer, which has resulted in significant advances, including the development of numerous therapeutic drugs that target so-called 'driver oncogenes.' While exceptionally effective in the short term, these targeted drugs fail almost universally, with patient tumors recurring within a few months to a few years. This has led many researchers to begin considering the role of non-genetic processes in the response of tumors to drugs."

Modeling and experimental techniques were used to distinguish the three different sources of variability among lung cancer cells: genetic, epigenetic and stochastic. As stated above, epigenetic and stochastic variabilities are different types of non-genetic variability. Epigenetically distinct cells look different, like the skin and liver cells from the example above, whereas stochastically distinct cells appear nearly identical but may act completely different.

"Distinguishing genetic from non-genetic, and epigenetic from stochastic, factors in drug response is crucial for developing new therapies that can kill tumor cells before they have a chance to acquire genetic resistance mutations," Harris said. "They all contribute to tumor drug response in different ways."

A framework for distinguishing genetic and non-genetic sources of heterogeneity in tumors has been proposed previously but is not yet widely accepted within the cancer research community because of a lack of strong experimental evidence. The team's paper provides strong support for this framework.

The analysis presented in the paper was applied specifically to EGFR-mutant non-small cell lung cancer. Harris' lab is currently applying these ideas to other cancer types as well, including small cell lung cancer, melanoma and bone-metastatic breast cancer.

"In my laboratory, we are working on building computational models of the molecular networks within cancer cells that give rise to the different epigenetic states, across which cells can transition to survive drug treatments," Harris said. "The long-term goal of my lab's research is to expand these models until they are of sufficient detail to act as virtual platforms for testing the effects of various drugs and identifying novel drug targets."

By constructing these so-called "digital twins," the hope is to one day use them to perform virtual drug screens on models built from samples of real patient tumors and then design personalized treatment options for those patients. This will require forming collaborations with bioinformaticians, experimentalists and clinicians here at U of A, the Winthrop P. Rockefeller Cancer Institute at the University of Arkansas for Medical Sciencesin Little Rock, and elsewhere. "Hopefully, the publication of this paper will help spark some of those collaborations," Harris said.

About the Public Library of Science: The Public Library of Science states on its website, "PLOS Biology empowers authors to publish the full arc of their research without compromising quality. Researchers can more fully and accurately represent their science and get credit for all their work."

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Research Shows Non-Genetic Tumor Diverseness Contributes to Treatment Failure in Cancer Patients - University of Arkansas Newswire

PITTSBURGH, July 16, 2021 - For the first time ever, researchers from the University of Pittsburgh School of Medicine discovered that phages--tiny viruses that attack bacteria--are key to initiating rapid bacterial evolution leading to the emergence of treatment-resistant "superbugs." The findings were published today in Science Advances.

The researchers showed that, contrary to a dominant theory in the field of evolutionary microbiology, the process of adaptation and diversification in bacterial colonies doesn't start from a homogenous clonal population. They were shocked to discover that the cause of much of the early adaptation wasn't random point mutations. Instead, they found that phages, which we normally think of as bacterial parasites, are what gave the winning strains the evolutionary advantage early on.

"Essentially, a parasite became a weapon," said senior author Vaughn Cooper, Ph.D., professor of microbiology and molecular genetics at Pitt. "Phages endowed the victors with the means of winning. What killed off more sensitive bugs gave the advantage to others."

When it comes to bacteria, a careful observer can track evolution in the span of a few days. Because of how quickly bacteria grow, it only takes days for bacterial strains to acquire new traits or develop resistance to antimicrobial drugs.

The researchers liken the way bacterial infections present in the clinic to a movie played from the middle. Just as late-arriving moviegoers struggle to mentally reconstruct events that led to a scene unfolding in front of their eyes, physicians are forced to make treatment decisions based on a static snapshot of when a patient presents at a hospital. And just like at a movie theater, there is no way to rewind the film and check if their guess about the plot or the origin of the infection was right or wrong.

The new study shows that bacterial and phage evolution often go hand in hand, especially in the early stages of bacterial infection. This is a multilayered process in which phages and bacteria are joined in a chaotic dance, constantly interacting and co-evolving.

When the scientists tracked changes in genetic sequences of six bacterial strains in a skin wound infection in pigs, they found that jumping of phages from one bacterial host to another was rampant--even clones that didn't gain an evolutionary advantage had phages incorporated in their genomes. Most clones had more than one phage integrated in their genetic material--often there were two, three or even four phages in one bug.

"It showed us just how much phages interact with one another and with new hosts," said Cooper. "Characterizing diversity in early bacterial infections can allow us to reconstruct history and retrace complex paths of evolution to a clinical advantage. And, with growing interest in using phages to treat highly resistant infections, we are learning how to harness their potency for good."

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Researchers surprised to find bacterial parasites behind rise of 'super bugs' - Science Codex

- Webinar on Wednesday, July 28 @ 10 a.m. ET -

ST. LOUIS, July 19, 2021--(BUSINESS WIRE)--M6P Therapeutics ("M6PT" or "the Company"), a privately held life sciences company developing next-generation recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs), today announced that it will host a key opinion leader (KOL) webinar on LSDs on Wednesday, July 28, 2021 at 10:00 a.m. ET.

The webinar will feature a fireside chat with KOLs Gregory Enns, M.D., Lucile Salter Packard Childrens Hospital Stanford School of Medicine, and Mark S. Sands, Ph.D., Departments of Medicine and Genetics at Washington University School of Medicine, who will discuss the current treatment landscape and unmet medical needs in LSDs, including Gaucher disease, Fabry disease, Pompe disease, mucopolysaccharidoses, and mucolipidoses. LSDs are a family of approximately 50 rare, genetic, and life-threatening diseases characterized by a deficiency in a specific lysosomal enzyme.

The event will also feature an update from the M6PT management team on its recombinant enzyme and gene therapy S1S3 bicistronic technology platform for the treatment of LSDs. The Company plans to initiate its first clinical program in 2022.

Dr. Enns, Dr. Sands, and M6PT management will also take questions from the audience.

To register for the webinar, please click here.

Dr. Enns is a Professor of Pediatrics and Genetics at the Lucile Salter Packard Childrens Hospital Stanford School of Medicine. He completed his medical education at the University of Glasgow (1990) in Scotland and completed his residency at the Children's Hospital Los Angeles Pediatric Residency in California. He then went on to complete his fellowship at the UCSF Medical Center in California. He is board certified in Clinical Genetics and Genomics. Dr. Enns research interests include novel means of diagnosing and treating mitochondrial disorders, with an emphasis on antioxidant therapy, lysosomal disorders, and newborn screening by tandem mass spectrometry. His current pursuits include the analysis of glutathione and antioxidant status in patients who have mitochondrial disorders and the development of new techniques for diagnosing and treating these conditions.

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Dr. Sands is a Professor in the Departments of Medicine and Genetics at Washington University School of Medicine in St. Louis. Dr. Sands received his Ph.D. in Molecular Pharmacology from the State University of New York at Stony Brook. He was a postdoctoral fellow at The Jackson Laboratory (Bar Harbor, ME) and at the University of Pennsylvania School of Veterinary Medicine before joining the faculty at Washington University School of Medicine. The goals of Dr. Sands laboratory are to better understand the underlying pathogenesis and developing effective therapies for inherited childhood diseases, specifically LSDs. A major focus of his group is to determine the safety and efficacy of adeno-associated viral gene transfer vectors for the treatment of both the central nervous system (CNS) and systemic manifestations of these diseases. In addition, his group has developed lentiviral-mediated hematopoietic stem cell-directed gene therapy approaches, as well as small molecule drugs, and more recently rational combinations of these approaches. The primary diseases that Dr. Sands studies are mucopolysaccharidosis type VII (MPS VII), Krabbe disease, and Infantile Neuronal Ceroid Lipofuscinosis.

About M6P Therapeutics

M6P Therapeutics is a privately held, venture-backed biotechnology company developing the next-generation of targeted recombinant enzyme and gene therapies for lysosomal storage disorders (LSDs). M6P Therapeutics proprietary S1S3 bicistronic platform has the unique ability to enhance phosphorylation of lysosomal enzymes for both recombinant enzyme and gene therapies, leading to improved biodistribution and cellular uptake of recombinant proteins and efficient cross-correction of gene therapy product. This can potentially lead to more efficacious treatments with lower therapy burden, as well as new therapies for currently untreated diseases. M6P Therapeutics team, proven in rare diseases drug development and commercialization, is dedicated to fulfilling the promise of recombinant enzyme and gene therapies by harnessing the power of protein phosphorylation using its S1S3 bicistronic platform. M6P Therapeutics mission is to translate advanced science into best-in-class therapies that address unmet needs within the LSD community. For more information, please visit: http://www.m6ptherapeutics.com.

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Contacts

Contact us to learn about partnering opportunities with M6P Therapeutics:

M6P Therapeutics: 314-236-9694info@m6ptherapeutics.com

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M6P Therapeutics to Host Key Opinion Leader Webinar on Lysosomal Storage Disorders - Yahoo Finance

Newswise In February 2020, a trio of bio-imaging experts were sitting amiably around a dinner table at a scientific conference in Washington, D.C., when the conversation shifted to what was then a worrying viral epidemic in China. Without foreseeing the global disaster to come, they wondered aloud how they might contribute.

Nearly a year and a half later, those three scientists and their many collaborators across three national laboratories have published a comprehensive study in Biophysical Journal that alongside other recent, complementary studies of coronavirus proteins and genetics represents the first step toward developing treatments for that viral infection, now seared into the global consciousness as COVID-19.

Their foundational work focused on the protein-based machine that enables the SARS-CoV-2 virus to hijack our own cells molecular machinery in order to replicate inside our bodies.

From structure to function to solutions

It has been remarked that all organisms are just a means for DNA to make copies of itself, and nowhere is this truer than in the case of a virus, said Greg Hura, a staff scientist at Lawrence Berkeley National Laboratory (Berkeley Lab) and one of the studys lead authors. A viruss singular task is to make copies of its genetic material unfortunately, at our expense.

Viruses and mammals, including humans, have been stuck in this battle for millions of years, he added, and over that time the viruses have evolved many tricks to get their genes copied inside us, while our bodies have evolved counter defenses. And although viruses often perform a long list of other activities, their ability to harm us with an infection really does come down to whether or not they can replicate their genetic material (either RNA or DNA, depending on the species) to make more viral particles, and use our cells to translate their genetic code into proteins.

The protein-based machine responsible for RNA replication and translation in coronaviruses and many other viruses is called the RNA transcription complex (RTC), and it is a truly formidable piece of biological weaponry.

To successfully duplicate viral RNA for new virus particles and produce the new particles many proteins, the RTC must: distinguish between viral and host RNA, recognize and pair RNA bases instead of highly similar DNA bases that are also abundant in human cells, convert their RNA into mRNA (to dupe human ribosomes into translating viral proteins), interface with copy error-checking molecules, and transcribe specific sections of viral RNA to amplify certain proteins over others depending on need while at all times trying to evade the host immune system that will recognize it as a foreign protein.

As astounding as this sounds, any newly evolved virus that is successful must have machines that are incredibly sophisticated to overcome mechanisms we have evolved, explained Hura, who heads the Structural Biology department in Berkeley Labs Molecular Biophysics and Integrated Bioimaging Division.

He and the other study leads - Andrzej Joachimiak of Argonne National Laboratory and Hugh M. ONeill at Oak Ridge National Laboratory specialize in revealing the atomic structure of proteins in order to understand how they work at the molecular level. So, the trio knew from the moment they first discussed COVID-19 at the dinner table that studying the RTC would be particularly challenging because multitasking protein machines like the RTC arent static or rigid, as molecular diagrams or ball-and-stick models might suggest. They're flexible and have associated molecules, called nonstructural and accessory proteins (Nsps), that exist in a multitude of rapidly rearranging forms depending on the task at hand akin to how a gear shifter on a bike quickly adapts the vehicle to changing terrain.

Each of these Nsp arrangements give insights into the proteins different activities, and they also expose different parts of the overall RTC surface, which can be examined to find places where potential drug molecules could bind and inhibit the entire machine.

So, following their serendipitous convergence in Washington, the trio hatched a plan to pool their knowledge and national lab resources in order to document the structure of as many RTC arrangements as possible, and identify how these forms interact with other viral and human molecules.

Science during shutdowns

The investigation hinged on combining data collected from many advanced imaging techniques, as no approach by itself can generate complete, atomic-level blueprints of infectious proteins in their natural states. They combined small-angle X-ray scattering (SAXS), X-ray crystallography, and small-angle neutron scattering (SANS) performed at Berkeley Labs Advanced Light Source, Argonnes Advanced Photon Source, and Oak Ridges High Flux Isotope Reactor and Spallation Neutron Source, respectively, on samples of biosynthetically produced RTC.

Despite the extraordinary hurdles of conducting science during shelter-in-place conditions, the collaboration was able to work continuously for more than 15 months, thanks to funding for research and facility operations support from the Department of Energys Office of Science National Virtual Biotechnology Laboratory (NVBL). During that time, the scientists collected detailed data on the RTCs key accessory proteins and their interactions with RNA. All of their findings were uploaded into the open-access Protein Data Bank prior to the journal articles publication.

Of the many structural findings that will help with drug design, one notable discovery is that assembly of the RTC subunits is incredibly precise. Drawing on a mechanical metaphor once more, the scientists compare the assembly process to putting together a spring-based machine. You cant put a spring in place when the rest of the machine is already in position, you must compress and place the spring at a specific step of assembly or the whole device is dysfunctional. Similarly, the RTC Nsps cant move into place in any random or chaotic order; they must follow a specific order of operations.

They also identified how one of the Nsps specifically recognizes the RNA molecules it acts upon, and how it cuts long strands of copied RNA into their correct lengths.

Having the vaccines is certainly huge. However, why are we satisfied with just this one avenue of defense? said Hura. Added Joachimiak: This was a survey study, and it has identified many directions we and others should pursue very deeply; to tackle this virus we will need multiple ways of blocking its proliferation.

Combining information from different structural techniques and computation will be key to achieving this goal, said ONeill.

Due to the similarity of RTC proteins across viral strains, the team believe that any drugs developed to block RTC activity could work for multiple viral infections in addition to all COVID-19 variants.

Reflecting back to the beginning of their research journey, the scientists marvel at the lucky timing of it all. When we started to talk, said Hura, we had no idea that this epidemic would soon become a pandemic that would change a generation.

This study was supported by the DOE Office of Science through the NVBL, a consortium of DOE national laboratories focused on the response to COVID-19, with funding provided by the Coronavirus CARES Act; and by the National Institutes of Health. The Advanced Light Source, Advanced Photon Source, High Flux Isotope Reactor, and Spallation Neutron Source are DOE Office of Science user facilities.

# # #

Founded in 1931 on the belief that the biggest scientific challenges are best addressed by teams, Lawrence Berkeley National Laboratory and its scientists have been recognized with 14 Nobel Prizes. Today, Berkeley Lab researchers develop sustainable energy and environmental solutions, create useful new materials, advance the frontiers of computing, and probe the mysteries of life, matter, and the universe. Scientists from around the world rely on the Labs facilities for their own discovery science. Berkeley Lab is a multiprogram national laboratory, managed by the University of California for the U.S. Department of Energy's Office of Science.

DOE's Office of Science is the single largest supporter of basic research in the physical sciences in the United States, and is working to address some of the most pressing challenges of our time. For more information, please visit energy.gov/science.

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Deconstructing the Infectious Machinery of SARS-CoV-2 - Newswise

PANAKS PARTNERS ANNOUNCES THE FIRST CLOSING OF ITS NEW PURPLE GLOBAL

BIOTECH/ MEDTECH FUND AT 150 MILLION ($180 MILLION)

Panaks will use the successful closing of its second Fund to extend its investment activity to biotech, while maintaining its ongoing activity in medtech, the focus of Panaks first fund

Panaks plans to invest the new fund in companies at the forefront of global innovation with the potential to transform patient care, with a focus on Europe, and Italy in particular

Panaks Purple Fund has been backed by the European Investment Fund (EIF), the Fund of Funds managed by CDP Venture Capital SGR, financial institutions and some of the main Italian companies operating in the Life Sciences sector

Milan (Italy), July 20, 2021 - Panaks Partners, the leading Italian venture capital firm in the Life Sciences sector, announces the first closing of its 150 million ($180 million) Purple Fund, the firms second fund.

Panaks Purple Fund is currently the largest venture capital fund actively investing in Italian companies and the most significant fund dedicated wholly to the Life Sciences sector in Italy. The fund will invest in companies at the forefront of innovation, with a focus on Europe, and Italy in particular, which remains underserved in terms of Venture Capital funding.

The Purple Fund is the second venture capital fund dedicated to life sciences launched by Panaks Partners. Panaks first fund, raised in 2016, supported companies in the medtech sector. To-date it has invested in 12 portfolio companies, which have collectively received almost 200 million in funding. Thanks to this financial support, these companies have already brought five innovative medical products to the market and have a further ten products in active clinical trials.

The Purple Fund has been backed by investors from the first fund as well as new investors. The Funds two anchor investors are EIF and the Fund of Funds FoF VenturItaly managed by CDP Venture Capital SGR. The EIF investment is backed under both the InnovFin Equity initiative from the European Commission under Horizon 2020, the Framework Programme for Research and Innovation, as well as the pan-European Guarantee Fund (EGF).

These anchor investors have been joined by several Italian banking foundations and pension funds, as well as numerous Italian companies and family offices in the Life Sciences sector. These include Menarini, the Cogliati family (Elemaster Group), the Colombo family (SAPIO Group), the Rovati family (Rottapharm Biotech), the Petrone family (Petrone Group), the Re family (Digitec Group), the Bassani family (Movi Group) and others.

The Purple Fund will invest mainly in Series A funding rounds, as well as later stage opportunities. The majority of investments will be in companies developing innovative therapeutics and products in the fields of biotechnology, diagnostics, and medical devices.

The fund aims to support the growth of entrepreneurial companies who will reshape healthcare globally by addressing real medical needs, saving lives and providing a better quality of life for patients. By achieving these goals, the fund aims to generate value for both investors and for society as a whole.

We are delighted with the successful first close of our new Purple Fund, and we would like to thank the high-quality investors who have trusted us. Over 500 innovative life science companies have already submitted funding requests to us in the first six months of 2021, said Fabrizio Landi, President of Panaks and a founding partner of the firm alongside Diana Saraceni and Alessio Beverina. The fund will remain open for additional subscribers until the end of the year, with a new target of 180 million. By expanding into the biotech sector, we hope to contribute to the growth of companies active in the development of new therapies and vaccines, concluded Landi.

Panaks has established a strong track record and solid international credibility since it was created a few years ago, also with the support of the CDP Group. commented Enrico Resmini, Chief Executive Officer of CDP Venture Capital SGR. We are delighted to invest in Panaks second fund, as it extends its activity into biotechnology, a sector where long-term planning and the availability of capital is essential to finance the R&D that is expected to lead to the innovative new therapies of tomorrow.

Alain Godard, Chief Executive of the European Investment Fund (EIF/FEI), added: We are happy to once again support Panaks after our previous investment in its first fund. Panaks has managed to build a strong brand in Italy and beyond thanks to its expertise in identifying and investing in novel medtech opportunities. With the extension of its investment strategy into biotech and the resulting growth of the team, Panaks will be able to further support European Life Sciences companies, and particularly those in Italy, which have exceptional R&D but are strongly underserved in terms of Venture Capital funding. We are glad to be able to use both the InnovFin mandate from the European Commission and the direct backing of EU Member States under the European Guarantee Fund to further support this exciting market segment.

To support its expansion into the biotech sector, Panaks intends to recruit three new professionals with significant experience in drug discovery and development in the pharmaceutical industry to its existing team, which is currently made up of 11 professionals. Recently Barbara Castellano has been promoted to the role of Partner, while the management team of the SGR has been strengthened with the arrival of a new CFO, Lorenzo Giordano, and a Financial Assistant, in the person of Andrea Steffanini.

Panaks Advisory Board has also been expanded and strengthened with the appointment of Biotech and Digital Health industry experts Fabio Pammolli, Professor of Economics, Finance, and Management Science at Politecnico di Milano, and Sergio Abrignani M.D. Ph.D. Full Professor at the National Institute of Molecular Genetics (INGM) in Milan.

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PANAKS PARTNERS ANNOUNCES THE FIRST CLOSING OF ITS NEW PURPLE GLOBAL BIOTECH/ MEDTECH FUND AT 150 MILLION ($180 MILLION) - PharmiWeb.com