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Our genes know kindness is the best medicine – Brainerd Dispatch

Posted: September 23, 2020 at 10:59 pm

Editor's note: Life can be stressful, and many of us have a hard time administering self-care. The current world situation ripe with conflicts, shortages and a pandemic makes things even harder. Dr. Amit Sood, formerly of Mayo Clinic and now head of the Global Center for Resiliency and Wellbeing, is the author of books including "The Mayo Clinic Handbook for Happiness" and "SMART with Dr. Sood, and creator of the mobile app Zizo: Your Resilience Pal. Now, he is writing a weekly column answering readers' questions on these topics. See the tagline to learn how to send him your questions, and he will answer in future columns.

Dear friend,

Of the hundreds of ice-creams I have eaten so far, the only one I remember is the ice-cream I never ate.

That was in 1993. My wife Richa and I were sitting outside a shop in the sweltering New Delhi. We had just bought an ice cream to cool down. From the corner of my eye, I saw a little boy who could use a few calories. I walked up to him and offered my cone that he gladly took. I have savored the gleam I saw in his eyes at least a dozen times since then.

In a very interesting study, researchers looked at the genetic fingerprint of the two types of happiness hedonistic (self-centric) and eudaimonic (other-centric). People who were hedonistically happy had a higher inflammatory and lower anti-viral gene expression. It was just the opposite for the eudaimonic ones. With many illnesses caused by inflammation, you can see why this is so important for our health.

My take on this research is that our genes and the immune system know what is right for us and society. In the current times when a healthy immune system is extremely important for us, promoting kindness is imperative.

Kindness, research shows, pays three times over. Your kind words and actions enhance your health and wellbeing, help others, and the memory of kind actions by itself enhances your wellbeing. A very simple way to enhance your self-worth and happiness today is to count your previous kind actions.

I suggest take out a pen and paper and write the three most selfless things you have done in your life. If you feel up to it, share your experience with someone. Just counting previous acts of kindness can enhance your self-worth and bring happiness to others (you guessed it right witnessing or hearing about others kind actions also increases happiness).

In kindness,


Dr. Amit Sood answers your questions about stress, resilience, happiness, relationships, and related topics in his column. Email

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Our genes know kindness is the best medicine - Brainerd Dispatch

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Researchers explore naturally occurring viral vectors: ‘Our study will change the trajectory of AAV-based gene therapies’ –

Posted: September 23, 2020 at 10:59 pm

Culture-adapted AAV2 is a viral vector which is used to deliver gene therapy to the liver. However, clinical trials targeting diseases of the liver have had an 'unexpectedly low success rate' using the vector, according to researchers: who have now found that naturally occurring AAVs may be more effective.

The prototypical AAV2 discovered more than 50 years ago provides the serotype on which the field of AAV vectorology and gene therapy is based. The researchers from Australias Childrens Medical Research Institute (CMRI) say the discovery 'will shake the foundations of the field of AAV-based gene therapeutics and will mark the beginning of a new era not only for biomedical research, but most importantly, for millions of patients affected by genetic disorders'.

One area of interest in gene therapy is using AAVs to target the liver, which is involved in genetic disorders such as haemophilia and various enzyme deficiencies.

AAV2 is a viral vector used to deliver gene therapy to the liver, carrying therapeutic DNA to target cells in the body. It binds to a receptor on the target cell. However, the researchers found that while AAV2 binds to the attachment receptors - heparan sulfate proteoglycans (HSPCs) - it does so too tightly.

This means that the vector can get trapped on other cells in the body and not the target liver cells. This reduces the number of vectors that deliver their therapeutic cargo to the liver, diminishing therapeutic efficacy.

The teams of Dr Leszek Lisowski, Head of the Translational Vectorology Research Unit, and Prof Ian Alexander, Head of the Gene Therapy Research Unit, then turned to naturally occurring vectors isolated from liver samples. They found that these which use an as of yet unknown receptor are much more successful at delivering therapies to the liver.

CMRI researchers are now able to make vectors in the lab that use this better receptor, instead of HSPGs, potentially making the next generation of gene therapy targeting the liver 'vastly more successful'.

Theorizing that manufacturing methods could be playing a role, the researcherscompared traditional AAV vectors grown in culture with naturally occurring vectors that they isolated from liver samples. They observed that the cultured vectors rapidly mutated as they replicated in the lab: with these changes making the vectors bind more tightly to molecules called HSPGs on the surface of liver cells, but also impeding their ability to infect humanized liver cells in mice.

In contrast, the naturally occurring vectors infected liver cells more efficiently and bound less tightly to HSPGs, although these effects disappeared when the scientists grew the natural vectors in culture over time.

This really challenges a basic concept in our field that binding strongly to HSPG was essential for AAVs' entry into human cells and suggests that vectors targeting the other receptor used by natural AAVs, of human liver origin, are likely to be more effective for clinical gene therapy applications, said Dr Lisowski. The prototypical AAV2, discovered over 50yrs ago, is the serotype on which the entire field of AAV vectorology and gene therapy is based.

Our study sheds new light and challenges our previous understanding and corrects misconceptions about how the vector binds to the cells.

Researchers at the CMRI can now start to improve on the use of vectors to help children with liver conditions. A better vector can increase safety and improve efficiency, while the increased therapeutic efficacy will mean lower doses are needed and thus reduce the cost of treatment.

The insights on adeno-associated virus receptor binding can potentially be extended to other tissues beyond the liver, add researchers. This makes this a very impactful study which will change the trajectory of AAV-based gene therapies.

Adeno-associated viruses (AAVs) were discovered in the 1960s. The vectorization of AAV2, a human isolate, in 1984 set in motion the development the use of the viral vector in gene therapy.

The liver is a key target for developing more efficient AAV vector delivery, given its direct involvement in a number of genetic and acquired diseases.

Source: Science Translational Medicine, September 9, 2020.DOI: 10.1126/scitranslmed.aba3312

Title: Restoring the natural tropism of AAV2 vectors for human liver

Authors: M. Cabanes-Creus; C.V. Hallwirth; A. Westhaus; B.H. Ng; S.H.Y. Liao; E. Zhu; R.G. Navarro; G. Baltazar; M. Drouyer; S. Scott; G.J. Logan; S.L. Ginn; I.E. Alexander; L. Lisowski at University of Sydney in Westmead, NSW, Australia; C.V. Hallwirth; S. Scott; G.J. Logan; S.L. Ginn; I.E. Alexander at Sydney Children's Hospitals Network in Westmead, NSW, Australia; A. Westhaus; G. Santilli; A.J. Thrasher at University College London in London, UK.

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Researchers explore naturally occurring viral vectors: 'Our study will change the trajectory of AAV-based gene therapies' -

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CPhI Annual Report 2020 Part 1: 2025 Will See Cell and Gene Therapy Capacity Shortages in the USA and 10+ mAb Approvals in China – Healthcare…

Posted: September 23, 2020 at 10:59 pm

October 5-16, 2020. Report on Cell and Gene Therapy to be discussed.

CPhI Annual Reportlaunched ahead of the first CPhI Festival of Pharma(5-16 October, 2020), the worlds largest digital pharma Expopredicts dramatic growth of new mAb production in China, capacity shortages for cell and gene therapies in the USA, and the widespread global adoption of single-use technologies, but only limited continuous bioprocessing.

Three CPhI experts from BioPlan AssociatesVicky Qing XIA, Leo Cai Yang and Eric Langerexplore the rapidly changing global biologics markets, with special reference to the implications for contract outsourcing and Chinas continued emergence as a hub for both bio innovation and contract services.

Remarkably, China is predicted to continue its rapid bio growth rates, with more than 10 new mAbs predicted to be launched per year in the country by 2025. In fact, the total market size will quadruple by 2025, reaching 120bn RMB, and rising further to 190bn RMB by 2030.

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As most early-stage biotech in China lack manufacturing facilities, the need for contract manufacturing services is rising quickly, and has been accelerated further by the 2016 MAH reforms,commented Vicky XIA, senior project manager at BioPlan Associates.

According to the CPhI report, bioprocessing outsourcing in China is currently highly stratified with four tiers and just one domestic company intieroneWuXi Biologicsand a number of international CDMOs including BI, Lonza, and Merck. However, by 2025 it is anticipated that as many as five more domestic CDMOs may have reached tier one status, with FDA and EU facility approvals. Significantly, pending regulatory changes for contract vaccine production will likely spur further rapid growth amongst the existing tier one CDMOs.

WuXi Biologics in 2019 realized 35.3% of its total revenue of USD 0.57 billion from China, which is ~USD 0.2 billion and would translate to ~35% of the total biopharma outsourcing service market in China1. However, despite sizable growth in revenue, its overall share of the market will now fall, as the mAb market expands rapidly,says XIA.

In terms of global manufacturing, Single Use Systems (SUS) are now the leader at both pre-clinical and clinical stages, with nearly 85% now involving a substantial SUS component. Yet whilst its usage continues to grow, continuous bioprocessing is not anticipated to be in mainstream usage by 2025.

Eric Langer,president and managing partnerof BioPlan Associates says: Because preclinical and clinical pipeline products require flexible manufacturing, SUS lends itself to these scales, but many of these will fail as they progress through the pipeline.This means that while more commercial-scale biologics are going to be made in SUS platforms, or hybrid systems, over the next 2+ years, stainless platforms are, and will remain, critical to bioprocessing as well.

The report also suggested that in the United States and Europe there is likely to be a cell and gene therapy capacity crunch by 2025, with CDMOs investing in this area already expanding to try and meet the pipelines demand. However, capacity alone is not the biggest challenge as there is a shortage of bothspecialized platforms, and trained personnel to operate them. Significantly, for some facilities in these emerging areas the equipment required for expansion and up-scaling may not yet exist, nor are regulatory authorities fully aligned with issues around patient treatmentswhich could slow approvals.

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Tara Dougal, head of content at CPhI Festival of Pharma, commented:This first part of the CPhI Annual Report arrives at a very poignant moment as the CPhI Festival of Pharma launches next month. The cell and gene therapy space is hugely fast-moving and we will run a session on the potential for M&As as both manufacturers and CDMOs try to buy-in expertise rather than build. More generally, our report also points to sustained bio growth in China, almost universal adoption of single use technologies, with continuous processing growing but from a far lower base. In fact, we have invited the Jefferson Institute for Bioprocessing to expand on this theme, as they will explore both current strategies and look ahead at future approaches for bio manufacturing. Its one of the reasons we are so excited about the CPhI Festival of Pharma, as it provides avital platform to meet and exchange ideasat a time when the industry is changing quickly.

To download a copy of the first part of the CPhI Annual Report, please visitGlobal Pharma Insightsor register for early bird discount (ending September 20th) at theCPhI Festival of Pharmathe largest ever digital gathering of professionals with thousands of attendees from dozens of countries. Eric Langer will be part of the cell and gene therapy M&A session (Wednesday 7th, October), with theJefferson Institute for Bioprocessing session on Thursday 15th, October.

CPhI Annual Report expert summaries:

Eric Langer, President and Managing Partner, BioPlan Associates

Industry trends and opportunities in biopharma

Vicky XIAandLeo Yang,BioPlan Associates

Future trends and opportunities in China

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CPhI Annual Report 2020 Part 1: 2025 Will See Cell and Gene Therapy Capacity Shortages in the USA and 10+ mAb Approvals in China - Healthcare...

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Muscular Dystrophy Association Awards 15 Grants Totaling More Than $4 Million for Neuromuscular Disease Research – PRNewswire

Posted: September 23, 2020 at 10:59 pm

NEW YORK, Sept. 23, 2020 /PRNewswire/ --The Muscular Dystrophy Association (MDA) announced today the awarding of 15 new MDA grants totaling more than$4 million toward research focused on a variety of neuromuscular diseases (NMDs), including Duchenne muscular dystrophy (DMD), Charcot-Marie-Tooth disease (CMT), Becker's muscular dystrophy (BMD), spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), myotonic dystrophy type 1 (DM1) and facioscapulohumeral muscular dystrophy (FSHD). This round of grant funding reinforces MDA's unwavering commitmentin the face of declining income due to the COVID-19 pandemicto the progress of neuromuscular disease research and builds on the more than$1 billionMDA has already invested in research to uncover new treatments and cures for NMDs since its inception. Some grants will go into effect this year, while others will be awarded in 2021.

"We continue to fund the most innovative research that will lead us to cures for a range of neuromuscular diseases," saysSharon Hesterlee, PhD, executive vice president and chief research officer for Muscular Dystrophy Association. "We have already seen our investment pay off with the first effective neuromuscular disease therapies, and these grantees are pushing the envelope even further in diseases once thought incurable."

Dr. Hesterlee added, "Although COVID led the cancellation of MDA's spring review session, we are pleased to announce the funding of these projects, which were reviewed in 2019."

The newly funded projects will aim to advance research discoveries and new therapy development in multiple areas. The awarded grants will fund studies to further advance our understanding of genetic causes of and risk factors for NMDs, investigate new approaches to developing gene therapies and other innovative potential treatments, including stopping disease progression and improving genetic testing technologies.

For a complete list of individual awards for this grant cycle, visit MDA's website and explore theGrants at a Glancesection. Highlights from thegrant awards for this grant cycleinclude:

ALS grants will be announced separately later this month, as will grants being given jointly by MDA and other organizations.

About the Muscular Dystrophy AssociationFor 70 years, the Muscular Dystrophy Association (MDA) has been committed to transforming the lives of people living with muscular dystrophy, ALS, and related neuromuscular diseases. We do this throughinnovations in scienceandinnovations in care. As the largest source of funding for neuromuscular disease research outside of the federal government, MDA has committed more than $1 billion since our inception to accelerate the discovery of therapies and cures.Research we have supportedis directly linked to life-changing therapies across multiple neuromuscular diseases.MDA's MOVRis the first and only data hub that aggregates clinical, genetic, and patient-reported data for multiple neuromuscular diseases to improve health outcomes and accelerate drug development. MDA supports thelargest network of multidisciplinary clinicsproviding best in class care at more than 150 of the nation's top medical institutions. OurResource Centerserves the community with one-on-one specialized support, and we offer educational conferences, events, and materials for families and healthcare providers. Each year thousands of children and young adults learn vital life skills and gain independence atsummer campand through recreational programs, at no cost to families.During the COVID-19 pandemic, MDA continues to produce virtual events and programming to support our community when in-person events and activities are not possible. MDA's COVID-19 guidelines and virtual events are posted For more information,

SOURCE Muscular Dystrophy Association

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Muscular Dystrophy Association Awards 15 Grants Totaling More Than $4 Million for Neuromuscular Disease Research - PRNewswire

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University of Sydney to advance COVID 19 DNA vaccine to human trials – News – The University of Sydney

Posted: September 23, 2020 at 10:59 pm

This phase 1/1b, multi-centre, observer-blinded, dose-ranging, randomized, placebo-controlled trial will assess the safety, reactogenicity and immunogenicity of different doses of the vaccine given to healthy participants aged 18 to 75 years old in three states (NSW, SA and WA) across Australia.

As a phase 1 trial, the key goal is to examine the safety of two doses of the vaccine given one month apart. If the trial is successful, then a phase 2 trial will be undertaken in a larger number of participants.

Through this network of key experts and partners, this collaborative approach promises to accelerate the development and access to this COVID-19 candidate vaccine in Australia.

The Vax4COVID specialised expertise focuses on rapid planning, recruitment, ethics and genetically modified organism (GMO) approvals, and execution of SARS-CoV-2 vaccine trials in healthy individuals and/or at-risk populations, including healthcare workers and older adults, with the capacity to pre-recruit cohorts of interested individuals.

This project embodies the important role the University of Sydneys health and medical researchers play in rising to the health challenges of our time. In what has been a difficult year, it is the collaborations between our leading scientists and industry, such as this, that will provide the solutions and pave the way forward, said Professor Duncan Ivison, Deputy Vice-Chancellor (Research) at the University of Sydney.

Mr Laurent Dapremont, Chief Executive Officer of Technovalia said: We are very pleased and proud to be able to contribute to the development of an effective solution to the crisis we have been living for many months. This is a unique collaborative effort with outstanding partners and clinicians, and most importantly, plans are being put in place with BioNet and LuinaBio to rapidly develop a vaccine againstCOVID-19 which can be produced in Australia.

Dr Pham Hong Thai, Chief Executive Officer of BioNet-Asia, said: We are glad to continue our collaboration with Technovalia as this is the second vaccine to be evaluated in Australia, first Pertagen and now this vaccine, two recombinant vaccines against highly contagious respiratory diseases, respectively pertussis (whooping cough) and COVID-19. We are also delighted to join A/Prof. Wood and his colleagues from Vax4COVID in the fight against the COVID-19 pandemic in Australia.

University of Sydney to advance COVID 19 DNA vaccine to human trials - News - The University of Sydney

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Libra Therapeutics Launches with $29 Million Series A to Develop Novel Therapeutics for Neurodegenerative Diseases – BioSpace

Posted: September 23, 2020 at 10:59 pm

Sept. 23, 2020 12:00 UTC

Company appoints Isaac Veinbergs, Ph.D., an established leader with strong expertise in diseases of the central nervous system, as President and CEO

SAN DIEGO--(BUSINESS WIRE)-- Libra Therapeutics, Inc. launched today to develop novel drug candidates focused on restoring the cellular balance lost in neurodegenerative diseases. These include amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), which are both characterized by disequilibrium caused by the production and decreased clearance of neurotoxic proteins. In conjunction with the companys launch, Libra Therapeutics has secured $29 million in Series A financing co-led by Boehringer Ingelheim Venture Fund (BIVF), Epidarex Capital, and Sant. The round was joined by Yonjin Venture, Dolby Family Ventures, and Sixty Degree Capital.

Libra Therapeutics was founded on breakthrough science generated by Axxam S.p.A., a leading discovery research organization, which provides Libra Therapeutics novel and proprietary chemical matter, cutting edge and exclusive assays, and robust drug discovery expertise. The financing proceeds will be used primarily to build the team and advance a pipeline of novel small molecule drug candidates for the treatment of neurodegenerative diseases.

My career has been dedicated to CNS drug discovery. I have seen firsthand the devastation diseases like ALS, Alzheimers, and Parkinson's disease have on patients and their families, said Isaac Veinbergs, Ph.D., President and CEO of Libra Therapeutics. We are delighted to build on Axxams pioneering foundational research to accelerate the development of novel therapies with the potential to slow or prevent disease progression to deliver meaningful improvements in patient outcomes across a wide range of neurodegenerative diseases.

The most frequent genetic cause of ALS and FTD is the expansion of the C9orf72 gene. Multiple studies have proposed that C9orf72 regulates intracellular trafficking and autophagy in neuronal cells, and thus Libra Therapeutics aims to increase autophagy to counter the loss of C9orf72 function. The companys therapeutic platform is uniquely positioned to discover and develop novel small molecule drugs that can both increase autophagy to more rapidly clear toxic proteins and attenuate the production of neurotoxic proteins.

Libra Therapeutics takes a unique approach to tackling neurodegenerative diseases with two distinct but complementary approaches that target key pathways that drive neurodegeneration, said Martn Heidecker, Ph.D., Managing Director, BIVF USA. By modulating scientifically and genetically validated targets to decrease neurotoxic proteins, theres a clearly defined development path with translational and clinical biomarkers.

We are delighted to have played a key role in the creation and funding of Libra Therapeutics as well as co-leading the financing. The science behind the company is outstanding and has enabled the recruitment of a highly experienced executive and an exceptional international investor syndicate, said Henning Steinhagen, Ph.D., Venture Partner, Epidarex Capital. By bringing together these key components, Libra Therapeutics has a highly differentiated platform to generate novel therapies that aim to improve the lives of patients with neurodegenerative diseases.

About BIVF

Created in 2010, the Boehringer Ingelheim Venture Fund GmbH (BIVF) invests in groundbreaking therapeutics-focused biotechnology companies to drive innovation in biomedical research. BIVF is searching for significant enhancements in patient care through pioneering science and its clinical translation by building long-term relationships with scientists and entrepreneurs. BIVFs focus is to target unprecedented therapeutic concepts addressing high medical needs in immuno-oncology, regenerative medicine, infectious diseases and digital health. For more information, visit

About Epidarex Capital

Epidarex Capital is a transatlantic venture capital firm with a track record of building exceptional life science companies in emerging hubs in the US and UK. Epidarex experienced team of early stage investors partner with entrepreneurs and leading research institutions to transform world-class science into highly innovative products addressing major unmet needs in the global healthcare market. For more information, visit

About Sant

Founded in 2006, Sant is a specialized healthcare and life sciences investment firm with over a half-billion dollars in capital under management. The Firm invests in early-stage companies developing innovative new medical technologies, biotechnologies and digitally-enabled healthcare services. Since inception, the Firm has made nearly 40 portfolio investments, including Claret Medical (Boston Scientific), TVA Medical (Becton Dickinson), Millipede Medical (Boston Scientific), Molecular Templates (MTEM), AbVitro (Celgene) and Explorys (IBM Corp). Sant invests nationally and is headquartered in Austin, Texas, with additional offices in Houston. For more information, please visit

About Libra Therapeutics

Libra Therapeutics is a biotechnology company focused on developing novel disease-modifying therapeutics that can restore the cellular balance disrupted in neurodegenerative diseases. The company is advancing three distinct and novel preclinical small molecule programs designed to increase autophagy and decrease production of neurotoxic protein aggregates. Libra Therapeutics was founded to capitalize on proprietary technologies and therapeutic assets from Axxam, a leading discovery research company. The company is based in San Diego. To connect with the company, visit

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Libra Therapeutics Launches with $29 Million Series A to Develop Novel Therapeutics for Neurodegenerative Diseases - BioSpace

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