Scribe Therapeutics change the genes responsible for causing diseases

Imagine being able to change the genes responsible for causing diseases. For Scribe Therapeutics, a gene-editing company that develops genetic medicines, this is no longer a dream but a reality. Scribe Therapeutics is one of several companies approaching genetic medicines through Crispr, the now-famous “molecular scissors” employed to cut and edit DNA. But the company is taking a new approach to leveraging Crispr technology. Instead of relying on wild-type or naturally occurring Crispr molecules such as Cas9, Scribe Therapeutics have built their own, highly-specialized varieties.

Founded by Jennifer Doudna, Benjamin Oakes, Brett Staahl, and David Savage, Scribe Therapeutics is creating an advanced platform for Crispr-based genetic medicine.

Crispr is changing how we think about treating diseases,” says co-founder, President, and CEO of Scribe Therapeutics, Benjamin Oakes. “When I finished my undergraduate degree, I shadowed doctors and realized we had no way to treat the underlying causes of diseases. This changed my career path to creating Crispr-based tools that can actually treat the underlying causes.”

Scribe Therapeutics has collaborated with Biogen to create Crispr-based genetic medicines for diseases such as amyotrophic lateral sclerosis (ALS). The company is also studying how to use adeno-associated virus (AAV) vectors to deliver Crispr components to the nervous system, eyes, and muscles. AAV vectors can deliver DNA to specific target cells for therapeutic uses.

Today, Scribe Therapeutics announced a $100 million Series B funding round that will help the company grow and expand. One of the key ways it stands out from other synthetic biology and gene-editing companies is through its approach to doing science. Other companies sometimes create tools without thinking about the problems they can solve, but Scribe Therapeutics is different. Instead of building technology in need of a solution, Scribe Therapeutics finds the problem first and creates the technology to fix it.

We face challenges head-on and continue to inspire people to try the hard things. You have to encourage fearlessness in science. If your experiment failed today, it doesn’t mean you’re a failure. You have to keep trying,” says Oakes.

Scribe Therapeutics‘ “Crispr by designplatform has custom-engineered millions of novel molecules specifically designed for therapeutic uses within the human body. For example, its X-editing (XE) technology is an engineered molecule that offers greater specificity, activity, and deliverability when used therapeutically.

Source: https://www.forbes.com/

How To Make Robots More Effective On The Future Battlefield

In an effort to make robots more effective and versatile teammates for Soldiers in combat, Army researchers are on a mission to understand the value of the molecular living functionality of muscle, and the fundamental mechanics that would need to be replicated in order to artificially achieve the capabilities arising from the proteins responsible for muscle contraction.

Bionanomotors, like myosins that move along actin networks, are responsible for most methods of motion in all life forms. Thus, the development of artificial nanomotors could be game-changing in the field of robotics research.

Researchers from the U.S. Army Combat Capabilities Development Command‘s Army Research Laboratory ‘(CCDC ARL) have been looking to identify a design that would allow the artificial nanomotor to take advantage of Brownian motion, the property of particles to agitatedly move simply because they are warm.

The CCDC ARL researchers believe understanding and developing these fundamental mechanics are a necessary foundational step toward making informed decisions on the viability of new directions in robotics involving the blending of synthetic biology, robotics, and dynamics and controls engineering.

Army researchers are on a mission to understand the value of the molecular ‘living’ functionality of muscle, and the fundamental mechanics that would need to be replicated in order to artificially achieve the capabilities arising from the proteins responsible for muscle contraction

By controlling the stiffness of different geometrical features of a simple lever-arm design, we found that we could use Brownian motion to make the nanomotor more capable of reaching desirable positions for creating linear motion,” said Dean Culver, a researcher in CCDC ARL’s Vehicle Technology Directorate. “This nano-scale feature translates to more energetically efficient actuation at a macro scale, meaning robots that can do more for the warfighter over a longer amount of time.”

These widely accepted muscle contraction models are akin to a black-box understanding of a car engine,” Culver explained. “More gas, more power. It weighs this much and takes up this much space. Combustion is involved. But, you can’t design a car engine with that kind of surface-level information. You need to understand how the pistons work, and how finely injection needs to be tuned. That’s a component-level understanding of the engine. We dive into the component-level mechanics of the built-up protein system and show the design and control value of living functionality as well as a clearer understanding of design parameters that would be key to synthetically reproducing such living functionality.”

Culver stated that the capacity for Brownian motion to kick a tethered particle from a disadvantageous elastic position to an advantageous one, in terms of energy production for a molecular motor, has been illustrated by ARL at a component level, a crucial step in the design of artificial nanomotors that offer the same performance capabilities as biological ones.

This research adds a key piece of the puzzle for fast, versatile robots that can perform autonomous tactical maneuver and reconnaissance functions,” Culver said. “These models will be integral to the design of distributed actuators that are silent, low thermal signature and efficient – features that will make these robots more impactful in the field.”

Culver noted that they are silent because the muscles don’t make a lot of noise when they actuate, especially compared to motors or servos, cold because the amount of heat generation in a muscle is far less than a comparable motor, and efficient because of the advantages of the distributed chemical energy model and potential escape via Brownian motion.

According to Culver, the breadth of applications for actuators inspired by the biomolecular machines in animal muscles is still unknown, but many of the existing application spaces have clear Army applications such as bio-inspired robotics, nanomachines and energy harvesting.

 

The Journal of Biomechanical Engineering recently featured their research.

Source: https://www.arl.army.mil/