Monthly Archives: July 2021

Kriya Therapeutics cofounder and CEO Shankar Ramaswamy.

Over the past several years, breakthroughs in gene therapy have led to treatments for rare diseases that were deadly just a decade ago. Take Zolgensmain 2019, it was the first gene therapy approved by the FDA to treat spinal muscular atrophy, a rare genetic disease that affects the mobility of infants and children. But gene therapies have historically had two drawbacks: They are only used for rare diseases, and they carry a hefty price tag (treatment with Zolgensma costs $2.1 million).

Kriya Therapeutics is trying to overcome these obstacles by creating gene therapies for the massesand manufacturing them at a lower cost. On Wednesday, the startup announced that it had raised a $100 million Series B funding round to get it closer to this goal. The round was led by investors from Patient Square Capital and also involved investors from QVT, Dexcel Pharma, Foresite Capital, Bluebird Ventures, Transhuman Capital, Narya Capital, Amplo and the Juvenile Diabetes Research Foundation T1D Fund.

We think this is going to be an extraordinarily important therapeutic class that will revolutionize the treatment of many diseases, says Jim Momtazee, managing partner at Patient Square Capital.

The Silicon Valley-based company was founded in late 2019 by three pharmaceutical industry alums, including a former cofounder of Spark Therapeutics and the former president of United Therapeutics Corp. Shankar Ramaswamy, Kriyas CEO, was part of the foundational team at Roivant Sciences. The new round brings the companys total funding to $180 million; the company declined to reveal its valuation.

Kriyas main focus is its uniquely designed Adeno-associated virusesviruses that are harmless when they enter the body, but deliver instructions to cells that then pump out genes that are missing in some people with genetic diseases. Though the company still plans to develop treatments for rare diseases, what sets it apart is its focus on more common genetic diseases, like some forms of diabetes and obesity. So far, gene therapy has been really constrained in many respects from achieving its full potential, Ramaswamy says. We are believers in gene therapy being applied to rare diseases as well as prevalent diseases.

Ramaswamys goal is to build a company that can go from genetic target discovery to manufacturing and then full commercialization of new therapies, unlike a typical biotech startup that might partner with a large pharmaceutical company for the later stages of development (Ramaswamy says the company will be open to partnerships, but can bring a drug to commercialization on its own). Once the company discovers and develops new gene therapies, its 51,000-square-foot manufacturing facility in North Carolina can produce Adeno-associated viruses at scale to deliver the genes to patients in need. Ramaswamy says that capability will bring down the cost, with savings passed along to patients. I think the innovations that were delivering will make gene therapies much more affordable and accessible to patients, he says. We are very committed to not being a burden on the healthcare system.

The companys current pipeline of products are all preclinical, though Ramaswamy says that they plan to submit Investigational New Drug applications to the FDA for several products in late 2022 and early 2023. So far the company is developing gene therapies for type 1 diabetes, solid tumors and two eye conditions: geographic atrophy and uveitis. In the U.S., more than 3 million people combined have at least one of these conditions, meaning Kriya has a huge pool of potential customers. By comparison, there are fewer than 25,000 children and adults with spinal muscular atrophy in the country.

Ramaswamy says that the new capital will go toward continuing the companys explosive growthit now has 80 full-time employeesas well as refining its vector delivery platforms and manufacturing capabilities. In the future, the money will allow the company to continue to develop new gene therapies for diseases both common and rare. Were taking a very new approach, which is to think more broadly, Ramaswamy says.

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This Company Raised $100 Million To Bring Gene Therapy To The Masses - Forbes

How did you begin working on the development and ideation of a bionic heart?

I was approached by The Science Museum of Minnesota to design and develop experiences for an upcoming exhibition based around transhumanism, the process of humans merging with technology. One of these projects was to create a 3D hologram animation that presents a series of futuristic bionic organs using the peppers ghost concept which presents the hologram. What I found particularly interesting, and challenging was conceptualising a bionic heart through digital design. I was tasked with designing something that was both visually compelling and yet believable, the design concept had to be to be physiologically so that if it was used to design the physical product it could be implanted into someone, connecting the tubing to their arteries.

The goal of this exhibition, which runs parallel with my own goals, is to introduce the public to the world of human enhancement in an exciting and informative way, and what is more engaging than a holographic image of futuristic implants? There is plenty of science fiction that considers bionic bodies, but they tend to paint a dystopian world that makes us fear technological progress rather than welcome it; I believe its essential we paint an exciting picture of the future in which the capabilities of humans are vastly expanded, and I feel this exhibition portrays that perfectly.

What will designing in AR look like ten years away?

Whilst the fundamental process of designing is unlikely to change, the tools we use during the process certainly will. Augmented reality (AR) is one of the most interesting and exciting tools that can be used for this. As computers continue to reduce in size and increase in power we will see AR devices like the Microsoft Hololens reduce in size from bulky headsets to glasses to contact lenses and eventually brain implants. All aspects of the design process from research to prototyping will become faster, more streamlined and more connected, with areas of design most affected being concepting/prototyping and collaboration. We will be able to design, prototype, package and release our creations on one single platform, just as we often do with computers now. Our freedom to design where and when will be improved, despite your location; and our ability to collaborate will greatly increase as you'll be able to sync with collaborators anywhere in the world and instantly feel like you are in the same room as them. Discussing changes to your car design that's represented digitally in front of you, quickly making tweaks to the cars surfacing or perhaps the paint finish. At Seymourpowell we are already utilizing this technology, for example, when we were building the interior of Virgin Galactic's spaceship, I could be in my home in VR taking in feedback from a 3D avatar representation of my colleagues as we analyzed the inside of the ship. This allowed me to test and identify issues far more closely and talk to top designers around the UK.

What role does extended reality (XR) play in the world of transhumanism?

XR will play a very prominent role in the world of transhumanism, perhaps one of the most important roles. It's worth asking what reality is at this point. Reality in its simplest form is the sum or aggregate of all that is real or existent within a system. Our experience of reality is largely defined by our senses. If our sensory organs and brains can be adjusted, then so can our reality. In decades to come we may be able to experience things we cant yet comprehend. Neuroscientist David Eagleman is already exploring sensory substitution, creating a vest that converts audio data to vibrations, allowing users to feel sounds. His findings show that after some time users who have lost their hearing can start to understand what others are saying through these vibrations. As AR becomes more integrated into our lives, the more we will rely on extended reality technologies, just as we rely so heavily on our mobile phones now. Elon Musk argues that our attachment to mobile phones already makes us a form of early cyborg, imagine trying to go about your daily life without using a mobile phone. Whilst some voice understandable concern about having technologies so closely connected to our bodies, there are huge benefits, especially in the medical world. We will develop a much closer understanding and level of control of our own bodies and XR will be our primary way of interfacing with this.

What is the future of neural implants and how is the design process defining this?

A neural implant is a piece of technology implanted into the brain. Currently they're in the very early stages, however, many neuroscientists and tech leaders are working on prototypes and testing. It's likely the first brain implants will be used for medical purposes like repairing eyesight or reversing effects of other neural based diseases. The technology will inevitably move into the world of brain enhancement, for example a brain computer interface (BCI), which does exactly what youd think, connects your brain directly to a computer. Once we step into the world of altering and enhancing our brains, we really begin to consider the reality of turning ourselves into super humans, science fiction no more! Imagine a world in which brain enhanced humans can learn languages overnight or perhaps communicate telepathically. It will eventually become as easy as closing your eyes and plugging into the virtual world. As with all technologies and inventions, neural implants are driven and developed by the design process. It's the designers job to plan and direct the development of these technologies and ensure the best possible outcome. As with any design project, the prototyping phase is critical in testing and understanding which paths to take, and to help avoid any possible detrimental outcomes.

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Five minutes with Rafe Johnson - 2021 - Articles - Transform magazine

Biotech Startup Kriya Therapeutics has raised $100 million in a Series B funding round to boost its efforts to revolutionize gene therapy for severe diseases like diabetes and morbid obesity.

The Durham-based startup, which also has offices in Palo Alto, had already raised $80.5 million last year. This brings the total funding received by the company to over $180.5 million, as the seed investment received from Transhuman capital was not disclosed.

The Series B funding round was led by Patient Square Capital and counted with the participation of new and existing investors such as Transhuman Capital, QVT Financial, Dexcel Pharma, Narya Capital, JDRF T1D Fund, and more.

The proceeds will be used to further develop the startups core technology platforms, as well as expand its therapeutic pipeline and current programs for metabolic disease, ophthalmology, and oncology.

Shankar Ramaswamy, M.D., Co-Founder and Chief Executive Officer of Kriya Therapeutics, referred to the role the startup will play in the increasingly relevant gene therapy field by stating,

In recent years we have seen the promise of gene therapy become a reality for the treatment of a number of devastating diseases.

However, the field has been constrained by critical limitations in manufacturing technology, vector design capabilities, and cost.

Kriya was formed with the mission of revolutionizing how gene therapies are designed, developed and produced by fully integrating advanced manufacturing technologies, computational tools and development capabilities within a single company.

Jim Momtazee, Managing Partner of Patient Square Capital, will join Kriyas Board of Directors, which will provide the startup with over 31 years of experience in the health industry, being part of important institutions. He referred to the startup he will help direct by stating:

We believe that gene therapy will have a transformative impact on medicine over time, and companies that are able to integrate platform capabilities delivering better treatments, lower cost and broader applications of the technology are going to drive that innovation

The biotech startup is part of an industry that has seen a spike in interest from investors as a result of the failures revealed by the Covid19 pandemic and other health crises in recent years.

The team expects the technology they are developing will prove successful to improve disease-fighting efforts around the globe by providing a more effective and cheaper alternative to traditional therapeutic approaches.

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Kriya Therapeutics Lands $100 Million to Revolutionize Gene Therapy - Grit Daily

RESEARCH TRIANGLE PARK Kriya Therapeuticsis poised to revolutionize gene therapies for highly serious diseases like diabetes and severe obesity after landing a whopping $100 million in capital.

Thats on top of $80.5 million raised last year.

The biotech startup, with headquarters in Durham and Palo Alto, secured the Series B financing from Patient Square Capital.

Existing institutional investors also participated in this round, including QVT, Dexcel Pharma, Foresite Capital, Bluebird Ventures, Transhuman Capital, Narya Capital, Amplo and JDRF T1D Fund. New investors included Woodline Partners LP, CAM Capital, Hongkou, Alumni Ventures and others.

The company said proceeds from the financing would be used to further develop Kriyas core technology platforms, expand its therapeutic pipeline and advance its current programs in metabolic disease, ophthalmology and oncology.

Gene therapy startup in Triangle lands $100 million in new funding

Meanwhile, Jim Momtazee, managing partner of Patient Square Capital, will join Kriyas board of directors.

In recent years, we have seen the promise of gene therapy become a reality for the treatment of a number of devastating diseases, said Shankar Ramaswamy, M.D., Kriya co-founder and chief executive officer.

However, the field has been constrained by critical limitations in manufacturing technology, vector design capabilities and cost. Kriya was formed with the mission of revolutionizing how gene therapies are designed, developed and produced by fully integrating advanced manufacturing technologies, computational tools and development capabilities within a single company.

Founded in 2019, Kriya is the brainchild of Ramaswamy, former chief business officer for Axovant Gene Therapies; Fraser Wright, co-founder of Sparks Therapeutics; and Roger Jeffs, the former United Therapeutics CEO who has deep rootsinNorth Carolina.

At present, the company is developing its SIRVE (System for Intelligent Rational Vector Engineering) platform for de novo vector design, sequence modification and data analysis.

It is also developing STRIPE (System to Realize Improved Production Efficiency), a proprietary high-efficiency manufacturing platform integrating advances in cell line technology and upstream and downstream process to achieve exponential reductions in production costs at scale.

STRIPE is being developed at Kriyas 51,000-square-foot manufacturing facility in Research Triangle Park.

The companys full cGMP manufacturing infrastructure is expected to be online this year.

(C) N.C. Biotech Center

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Durham startup's $100M fundraiser could help lead to revolution in gene therapies - WRAL Tech Wire

Many governments are increasingly approaching artificial intelligence with an almost religious zeal. By 2018 at least 22 countries around the world, and also the EU, had launched grand national strategies for making AI part of their business development, while many more had announced ethical frameworks for how it should be allowed to develop. The EU documents more than 290 AI policy initiatives in individual EU member states between 2016 and 2020.

The latest is Ireland, which has just announced its national AI strategy, AI Here for Good. It aims to become an international leader in using AI to benefit our economy and society, through a people-centred, ethical approach to its development, adoption and use.

This is to be obtained via eight policy commandments, including increasing trust in and understanding of AI by using an AI ambassador - a veritable AI high priest to spread the message around the country. Another aspect is to promote AI adoption by Irish businesses and the government within a special moral and ethical framework. There are several shortcomings in this strategy, which it shares with similar efforts by other countries (leaving aside more obviously bad AI strategies, such as that underlying Chinas surveillance state).

Such strategies uncritically share the hype and hysteria surrounding AI. A typical example would be the chief executive of Googles owner Alphabet, Sundar Pichai, claiming in 2016 that AI is one of the most important things humanity is working on. It is more profound than, I dunno, electricity or fire.

He would say this, as his companys business model critically depends on AI, and on people trusting the technology. Irelands strategy goes precisely along with such hype by repeating the claim that AI could double Irish economic growth by 2035. It doesnt detail whose growth, or how.

The strategy lauds various useful existing AI-based apps which, for instance, improve cycling infrastructure in Dublin, provide Irish language tools, save energy, and comfort dementia suffers but it is hard to see how more of these could double economic growth.

Most notably, AI is central to a few digital platform firms such as Google, Apple, Facebook, Amazon and Alibaba - GAFAA for short. They enjoy winner-takes-most benefits due to the fact that current AI requires large amounts of data. As more people use your platform, the profitability of the data grows exponentially.

This has given a huge first-mover advantage to those companies that got it right, turning them into monopolists and gatekeepers. Digital platform firms disrupt existing businesses by out-competing them every time - a good example being how Google, basically an online search-engine, disrupted the advertisement-driven business model of newspapers, or how Apple is selling more watches than the centuries-old Swiss watch industry.

These platforms also depress the start-up of new firms, for instance by buying up all potential new competitors. This stifles innovation.

And increasingly, entrepreneurs have to compete on these platforms for example, Amazon Marketplace. They can be at the mercy of abuses such as fake product reviews by competitors; rulings on such issues by the gatekeepers that are unpredictable and opaque; and sudden algorithm changes that can affect their business by making them, for example, less visible to potential customers. Then there is the phenomenon of digital subsistence entrepreneurs online sellers who barely earn a living wage.

This radically different (anti-) competition landscape sometimes labelled platform capitalism has caused regulators and antitrust authorities substantial headaches. The EU recently adopted proposals for a Digital Markets Act (DMA) and a Digital Services Act (DSA), which try to rein in the actual and potential abuses on large AI-based digital platforms.

If AI and automation had been a force to reckon with, we would have seen skyrocketing labour-productivity growth and rising unemployment. Instead, we see stagnating productivity growth for example, the UKs is the lowest in 200 years - and some of the lowest unemployment rates in western economies in decades.

Irelands AI strategy ignores the above problems with platform capitalism. The name Google appears only once in the entire document, and Amazon and Facebook not at all. There is no reference to digital platforms, platform capitalism, the DMA, DSA or the EUs many antitrust actions against Google. The omission is like Hamlet without the Prince.

Irelands AI strategy should have specified how and when AI will achieve the economic benefits it mentions and who will reap them. It also should have offered a vision of how to make sure that the nation does not suffer from the GAFAAs or become a mere agent of them.

The strategy also assumes that a lack of trust in AI is due to people not understanding the technology well enough. So voil, teaching people data science and having an AI ambassador, like a modern-day prophet, is the answer. One may expect precisely the opposite outcome: the better people understand AI, the less they will trust it.

This would actually be desirable, of course. In the US, where understanding of AI is fairly advanced, adoption rates of AI are in fact meagre. A recent US Census Bureau survey of more than 800,000 US firms found that only 2.9% were using machine learning as recently as 2018. A 2020 survey by the European Commission also pointed to very low adoption levels.

Many other surveys confirm the low adoption rate of AI. Firms do not adopt it, not because they dont trust it, but because it makes little business sense. It is too expensive, usually with paltry returns, and comes with an exorbitant environmental price tag and all that before you factor in the domination of the incumbents.

Irelands AI - Here for Good, like many similar national strategies, seems to believe in miracles, for instance that various circles can be squared. These include enabling access to large volumes of relevant data for all firms while protecting everyones privacy, and turning the country into a powerhouse for training sizeable deep-learning models and massive data centres while cutting CO emissions. It admits no trade-offs.

The implied message is that Ireland can pluck wonderful fruits from a thicket of thorns, just so long as it trusts in AI and adheres to its particular ethical commandments. Transhumanists, GAFAA, and other winners-takes-all in the digital economy will approve wholeheartedly.

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Artificial intelligence: governments see huge business potential, but ignore the downsides - The Conversation UK

CONTENT WARNING: This story contains details that might be triggering to some readers.

Six words could hold the key to guiding someone out of the irrational, impulsive mental state that puts them at dangerously high risk of taking their own life.

People considering suicide generally lose their ability to rationalise, and see the act as a short sharp solution to their pain, clinical psychologist and clinical neuropsychologist Dr Roy Sugarman told news.com.au.

To help pull them away from such a state, asking one specific question could be critical, particularly if the person has expressed feeling as though nothing they do makes a difference.

Asking someone, what is important to you now? could be crucial to their survival, Dr Sugarman said.

Its saying, whatever is going to make a difference, where you feel that what you do does make a difference, Ill do it with you and Ill help you, he said.

The biggest driver is going to be this avoidance of thinking nothing I do makes a difference so I might as well avoid the pain by creating a very short sharp solution to the pain.

People in the grips of such a dire mental state have got there most likely as a result of a collapse in their values and meaningful aspects of their life, Dr Sugarman said.

So when youre asking the question, its in the here and now, and finding out who or what is critical to them at that moment in time, he said.

What to do after asking the question

After getting an answer, Dr Sugarman recommended staying with the person and beginning the process of helping them problem solve, step by step.

Make it manageable and give them a sense that what they do now makes a difference, he said.

Stay with them and help them plan. Help them solve problems.

If we can get people to not look at the big picture, and instead look at things that make a big difference like smaller micro goals, you start to get some movement and progress.

Dr Sugarman stressed that while posing the question may help, it was more important that a warm relationship was maintained over an extended period of time.

Its like anything else, to avoid a terrible event, youve got to maintain your relationship with a person. Its not about weaving some amazing web of speech. Your best chance of keeping someone alive is a warm relationship, he said.

The thing about suicide is that you cant use a rational speech to help a person who has got there by being irrational. The inspiring speech is not going to address the fact that at this stage, there is cognitive depletion. Theyre not going to respond to rationalisation.

Phone app to prevent suicide

Dr Sugarman is the co-founder of suicide prevention app Be A Looper developed alongside Transhuman Inc CEO Amanda Johnstone.

The app, released in 2017, allows users to rate areas of their mental health daily and share their score with four other people they trust.

Ms Johnstone worked closely with RUOK? in the development of the app, and used the same framework, called ALEC, in digitising is at an efficient and practical support tool.

The concept of Be A Looper was largely an extension of Ms Johnstones previous efforts to support her friends. For years she set alarms on their phones and requested they send her an emoji indicating their mental health every day at 4pm.

Ms Johnstone, who was named CEO magazines 2020 Start-up Executive of the Year, independently supported dozens of people for about 12 years before adapting her idea into an app.

I knew that system worked really well and that people would do it, she told news.com.au.

Were all on our phones so often with Instagram and everything, if theres one swipe each day that will make us more connected, it can save lives.

Countless users have contacted Ms Johnstone sharing instances of how an alert from the app which notifies others if someone records a low rating has saved theirs or a loved ones life.

Dr Sugarman said it was no surprise the app had been so effective in preventing suicide, given it harnessed already familiar concepts of swiping and using the 1-10 rating system.

It works because you establish as a routine the sharing of how you feel, so if you are a person who needs to use it, it gets you into the habit of expressing yourself as a number to four other people, he said.

Its very simple, concrete and non verbal, and you will be able to express how you feel when your logic has escaped you and youre a bundle of hot mess an emotions.

The app can be downloaded for free via the Apple app store.

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The critical question you need to ask someone if they express a desire to take their own life - NEWS.com.au