Researchers at UC San Diego created the largest maps of gene regulation to date, capturing the network of RNA binding proteins, the RNA sequences they bind to, and what the proteins do.

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Can you imagine organizing more than 10 terabytes of data into a useful network of information, like creating Google maps from scratch? I certainly can’t, but Eclipse BioInnovations, a San Diego-based biotech start-up, is aiming to do just that for RNA therapeutics.

Researchers at the University of California San Diego, who are also Eclipse co-founders, captured a high-resolution picture of the inner workings of gene regulation. They created the largest maps of the network of RNA binding proteins, the RNA sequences they bind to, and what the proteins do. The better resolution the map, the more researchers understand.

“By identifying where all the RNA binding proteins sit, the floodgates start to open for describing how RNA biology is modulated,” said Gene Yeo, Ph.D., MBA, Professor of Cellular and Molecular Medicine at UC San Diego and co-founder of Eclipse. “From a drug development perspective, we’ve laid out the largest landscape to date of almost 360 RNA binding proteins.” …


Your metabolism has a memory — and it can hold a grudge for years. Researchers turned to the epigenome to figure out how and why it does.

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Photo by Victor Josan on Shutterstock.

Yes, even your metabolism has a memory — and it can hold a grudge for years. In people with diabetes, periods of high blood sugar can negatively impact their health years later, even if they get their blood sugar under control. While this ‘metabolic memory’ phenomenon has been known for years, why it happens is poorly understood.

Rama Natarajan, Ph.D., Professor and Chair of the Department of Diabetes Complications & Metabolism at City of Hope, turned to our epigenome for the answer.

“We’ve shown the first link between DNA methylation in blood and stems cells, blood sugar history, and future development of complications,” said Natarajan. …


The immune response against the AlloPrime® vaccine can influence the response to a virus (like the coronavirus that causes COVID-19).

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Image credit: Dreamstime

Originally published: Jul 08, 2020

What if you could get one vaccine that protects you against a wide spectrum of viruses, even viruses we haven’t discovered yet? That might sound impossible and futuristic, but this is just what Immunovative Therapies and its sister company Mirror Biologics, Inc. are aiming to achieve. Their new ‘pan-viral’ vaccine called “AlloPrime ®” is slated to begin a Phase I/II trial next month.

“Our pan-viral vaccine harnesses the same protection mechanism that naturally protects us from viral diseases — a healthy immune system,” Michael Har-Noy, MD, Ph.D., Founder and CEO of Immunovative Therapies, told BioSpace. …


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Originally published: Jul 14, 2020

What do you get when you mix certain immune cells with vitamin D and a disease-provoking molecule? An innovative new vaccine for type 1 diabetes.

“We are trying to educate the immune system to teach it what not to do,” Bart O. Roep, Ph.D., Chan Soon-Shiong Shapiro Distinguished Chair in Diabetes at City of Hopeand professor/chair of the Department of Diabetes Immunology, told BioSpace. “It is a radical approach that does not involve immunosuppression — it engages the immune system and causes desensitization, like allergy treatments.”

This approach is considered an ‘inverse vaccine’ because it reverses certain immune responses, rather than activating the immune system, like most vaccines do. …


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Originally published: Jul 20, 2020

Another COVID-19 vaccine candidate recently entered the races. The difference — this one is a live attenuated (weakened) virus expressing the coronavirus’s signature spike protein on its surface. And it’s delivered nasally, not a shot.

“COVID-19 vaccine development will be more of a marathon than a sprint,” Martin Moore, Ph.D., co-founder and CEO of Meissa Vaccines, told BioSpace. “A live attenuated vaccine may not be first-in-class, but their historically high efficacy could make it the best-in-class.”

Moore spoke with BioSpace about live attenuated vaccines, interim data from their RSV vaccine candidate, and how they are building on their RSV vaccine platform to create a COVID-19 vaccine. …


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Originally published: Jul 29, 2020

After almost 15 years since the first gene therapy trial for Duchenne muscular dystrophy (DMD) began, the dream of a DMD gene therapy drug is getting closer to a reality.

BioSpace sat down with Sharon Hesterlee, Ph.D., chief research officer at the Muscular Dystrophy Association (MDA), to talk about the history and challenges of developing gene therapy for DMD and the DMD gene therapy field as a whole, including Pfizer’s and Sarepta Therapeutics’ latest clinical data.

Duchenne muscular dystrophy (DMD)

is a progressive muscle wasting disease caused by a genetic mutation. The mutated gene is on the X chromosome, making DMD an X-linked disease. This explains why it largely affects boys as they don’t have a backup copy of the gene (they only have one X chromosome). …


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Originally published: Aug 06, 2020

A small biotech company is aiming to recharge tired cells, giving mitochondrial disease patients new hope of a disease-specific treatment.

“We focus on patients, going after diseases with high unmet needs, like primary mitochondrial myopathies,” Niall O’Donnell, Ph.D., President, CEO and Director of Reneo Pharmaceuticals, told BioSpace.

Despite COVID-19 crashing the party and stopping their Phase Ib trial early, the company still showed that their drug candidate REN001 was safe in PMM patients, paving the way to a large trial slated to begin in early 2021. …


AiCure’s phone app helps trials keep tabs on which participants have taken their medications correctly and which have intentionally not.

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Image provided by AiCure

Originally published: Jul 31, 2020

How can you assess the effects of a drug if clinical trial participants do not take it as instructed? And how do you know if a participant has truly taken their medication? Those are the issues many clinical trials face, even potentially leading to a trial failure or the demise of an otherwise useful medication.

“AiCure is applying modern technology to this problem,” Ed Ikeguchi, M.D., CEO of AiCure, told BioSpace. “Our study is the first time anyone quantified intentional nonadherence in clinical trials.”

AiCure’s phone app helps clinical trials keep tabs on which participants have taken their medications correctly and which have not. By analyzing videos the app takes, they can also spot who is intentionally not taking their pills, known as intentional nonadherence. …


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Originally published: Aug 14, 2020

With only one FDA-approved therapy and standard-of-care treatment including avoidance and carrying an EpiPen in case of accidental exposure, the unmet need for peanut allergies is huge. Not to mention that peanut allergy is the most common food allergy in children, affecting 2.5% of U.S. children — nearly 1.8 million children and adults.

DBV Technologies aims to fill the peanut allergy treatment gap, especially for highly allergic children, with their low dose epicutaneous immunotherapy (EPIT) patch Viaskin™ Peanut.

“There’s an emotional aspect to the product — you’re putting this patch on your young child’s back every day instead of forcing them to eat something they might be dreading,” Pharis Mohideen, MD, Chief Medical Officer at DBV Technologies, told BioSpace. “It’s a nice feeling that you’re doing something you feel is going to make a difference and help them.” …


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Originally published: Aug 26, 2020

Lasers aren’t just for directing presentations, playing tag or annoying cats. They can also serve as virus hunters.

Botanisol Analytics, a start-up company based in Arizona, is developing a screening platform that can identify biological threats, from contaminants to pathogens (like coronavirus), simply by shining a laser onto the sample and interpreting the light that scatters.

“It’s a real-time snapshot of all the molecules in a patient’s sample,” David Talenfeld, JD, MBA, MGM, CEO of Botanisol, told BioSpace. “The device can be calibrated to detect a chemical or pathogen, like coronavirus, whose spectral ‘fingerprint’ is known. In order to get the device to the people who need it as quickly as possible, we are developing it as a screening technology working towards an EUA and not a diagnostic. The system is being designed to produce positive results in two minutes and negative results in under ten. We’ve implemented a design control process and are working towards compliance with CFR 820.30 …

About

Chelsea Weidman Burke, M.S.

Biochemist turned science journalist. Alzheimer’s, immunology, immunotherapy, genetics, cancer. Follow my publication! https://medium.com/chemically-inquisitive

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