Subscribe for email alerts

Don’t miss a single Science & Enterprise post. Sign up for our daily email alerts.

Please share Science & Enterprise

Delivery Tech Licensed for CBD Epilepsy Nasal Spray

Blooming cannibis plant

(My 420 Tours, Wikimedia Commons)

20 Sept. 2021. A company is developing a nasal spray for epilepsy symptoms with a cannabis extract delivered in nanoscale polymer particles to the brain. Virpax Pharmaceuticals Inc. in Berwyn, Pennsylvania is extending its existing license agreement for the drug delivery technology made by Nanomerics Ltd. in London, U.K.

Virpax Pharma develops non-addictive pain treatments and neurological therapies delivered as directly as possible to the source of the conditions, instead of most current systemic therapies given as oral or injected drugs. One of its product lines delivers therapeutic peptides, short chains of amino acids, as a nasal spray where, according to the company, the inhaled peptides can cross the blood-brain barrier to ease pain from cancer and other sources, and treat post-traumatic stress disorder or PTSD. The nasal sprays are packaged in single-use cartridges, and sent through the nose with an inert gas, which can be administered by a clinician or caregiver if needed.

Delivery of these therapies uses a process licensed from Nanomerics called molecular envelope technology. With this technology, Nanomerics encapsulates therapeutic molecules in nanoscale biocompatible polymer particles, 50 to 300 nanometers in size. Nanomerics says the polymers self-assemble around insoluble or peptide molecules, providing stability in transit and releasing their contents rapidly on delivery. As a result, says Nanomerics, higher doses of drugs can be delivered more safely to their targets, including to brain cells.

Under the agreement, Virpax Pharma is receiving an exclusive worldwide license from Nanomerics to apply molecular envelope technology to a nasal spray treating epilepsy-type seizures with a cannabidiol or CBD compound. The deal also calls for Nanomerics to conduct preclinical animal tests of Virpax’s CBD candidate code-named VRP324. Financial terms of the agreement were not disclosed.

Through olfactory nerve to the brain

CBDs are derived from the Cannabis sativa plant, but do not produce an emotional high associated with marijuana, also a derivative of cannabis. National Library of Medicine in the U.S. says some 80 chemicals, known as cannabinoids, are associated with these plants. One of those chemicals, CBD, a non-intoxicating derivative, makes up about 40 percent of cannabis extracts and has been studied extensively for a range of disorders. Epilepsy and related disorders are shown to respond to CBD, and in 2018 the Food and Drug Administration approved a CBD-based therapy for Lennox-Gastaut syndrome and Dravet syndrome, two rare and severe forms of epilepsy.

Virpax Pharma is developing VRP324 for patients one year or older, delivered as a powder into the nose and through the olfactory nerve to the brain for seizures associated with tuberous sclerosis complex, a rare genetic condition where benign tumors grow in the brain. Tuberous sclerosis complex can also cause impaired intellectual development, autism, and behavioral problems, but may take years for symptoms to appear. Virpax says VRP324 is being developed as well for treating Lennox-Gastaut syndrome and Dravet syndrome.

Anthony Mack, chairman and CEO of Virpax Pharma, says in a company statement that the company’s tests show delivery through the nose bypasses the liver, and “we believe that since the CBD will not be metabolized in the liver, this may reduce the concern of drug-to-drug interaction and/or the need to adjust the dosage of other related medications.” Mack adds, “We believe VRP324 is the first step toward building our neurological disorder therapy pipeline, expanding the use of our novel delivery platform technologies to multiple categories of neurology.”

More from Science & Enterprise:

*     *     *

Infographic – Digital Therapeutics Funding on the Rise

Chart: digital therapeutics funding

Click on image for full-size view (CB Insights)

18 Sept. 2021. As we’ve noted on these pages for some time, health care technologies are a favorite target for venture investors. This week, the technology intelligence company CB Insights reported on new investments in one of those technologies, digital therapeutics, and found a record-setting funding pace this year.

CB Insights defines digital therapeutics as “evidence-based, software-driven therapeutic interventions to prevent, manage, or treat medical conditions.” In its report on health care technologies funding for the second quarter of this year (registration required), CB Insights found the number of venture deals in digital therapeutics worldwide rose somewhat from 18 transactions in the first quarter to 20 in the next three months. But the total number of investment dollars fell to $580 million in the second quarter from nearly $700 million in the previous quarter.

In its update — and our infographic — this week, CB Insights found a turnaround in digital therapeutics investments. So far this year, digital therapeutics companies raised a record $1.6 billion from venture investors, eclipsing the $1.06 billion collected in all of 2020, and projected to reach nearly $2.5 billion by the end of the year. And the number of investment deals is on track to reach 87 by the end of 2021, if the current rate continues, also a record.

Digital therapeutics is a technology sector we also follow at Science & Enterprise. See below for our stories on this topic in just this month.

More from Science & Enterprise:

*     *     *

Neuroscience Software Company Raises $23M in Early Funds

Brain circuits illustration

(HypnoArt, Pixabay)

17 Sept. 2021. A company developing neuroscience data collection and analytics tools for clinicians and researchers raised $22.8 million in its first venture round. Rune Labs, a three year-old enterprise in San Francisco, designs software that integrates data from medical equipment and wearable devices to provide more precise diagnostics for neurodegenerative diseases and analytical tools for clinical trials.

Rune Labs aims to bring together neurological data from a wide range of sources into databases for analysis. The company’s technology seeks to integrate and synchronize these data from electrophysiology systems, brain images, and wearable devices from large numbers of patients and over longer periods of time. Rune Labs says its databases provide large-scale data volumes for detailed analytics, including deep learning algorithms. And the company says it partners with drug makers and academic medical labs to help design therapeutics for neurodegenerative disorders.

As an example of the large-scale neurological data becoming available for analysis, Rune Labs points to a recent study published in the journal Nature Biotechnology that captures data from Parkinson’s disease patients undergoing deep-brain stimulation. This technique implants electrodes in affected parts of the brain to treat the disorder, which up to recently was conducted mainly in hospitals. In this study, the researchers collected data from more modern and compact implanted devices patients could wear over longer periods of time, including at home.

The study team captured two-way data from five patients for up to 15 months, amassing more than 2,600 hours of data that enabled the researchers to identify and validate neurological biomarkers for each patient during their normal daily activities. The researchers were led by Ro’ee Gilron, a postdoctoral fellow at University of California in San Francisco, and lead neuroscientist at Rune Labs.

Expand technology platform to more disorders

In a blog post about the study, the company says …

This is a big deal because Parkinson’s, like most neurological conditions, can vary significantly over the day, day-to-day, in response to different environmental pressures, and in response to medication and diet: any in-clinic snapshot will only give a limited picture of a patient’s overall disease phenotype.  By contrast, this publication shows 2500 hours of brain sensing over days, weeks, and months across 5 individual patients, including quantitative tremor and dyskinesia labels from wearable devices and patient-reported outcomes for medication and symptoms.

Rune Labs raised $22.8 million in its first venture funding round, led by technology investor Eclipse Ventures in Palo Alto, California. Joining the round are Rune Labs’ earlier investors DigiTx Partners and Moment Ventures. Rune Labs, according to Crunchbase, raised nearly $6.5 million in two seed rounds between September 2019 and October 2020.

“Since our founding in 2018,” says Rune Labs founder and CEO Brian Pepin in a company statement, “we have made substantial progress in the development and implementation of our brain data software platform. This financing will enable us to expand the number of patients, clinicians, and researchers using our platform to inform precision therapy development and delivery for patients with Parkinson’s disease, multiple sclerosis, and depression.”

Eclipse Ventures identifies health care infrastructure as one if its investment targets. The investment company also announced yesterday a $19.5 million first-round investment in Safely You, a spin-off enterprise from University of California in Berkeley, developing systems using artificial intelligence to detect detect and help prevent falls in people in assisted-living facilities.

More from Science & Enterprise:

*     *     *

Yom Kippur 5782

Shofar

Shofar, a ram’s horn sounded during Jewish high holiday services (A. Kotok)

16 Sept. 2021. We’re observing Yom Kippur today, the day of atonement and holiest day in the Jewish calendar. Regular posting will resume tomorrow, Friday 17 Sept.

*     *     *

FDA Okays One-Time HIV Treatment Clinical Trial

HIV released from cell

Illustration of HIV released from the surface of an infected cell (Bette Korber, Los Alamos National Lab)

15 Sept. 2021. A company developing a one-time treatment for HIV infections using the gene-editing technique Crispr says it received FDA clearance to begin a clinical trial. Excision BioTherapeutics Inc. in San Francisco says the Food and Drug Administration accepted the company’s investigational new drug application for its HIV treatment candidate code-named EBT-101.

Excision Bio employs gene editing to remove large sections of DNA from HIV viruses, taking out the viruses’ ability to replicate and escape. For these edits, the company uses Crispr, short for clustered regularly interspaced short palindromic repeats, a process based on bacterial defense mechanisms that use RNA to identify, monitor, and edit targeted locations in DNA. Excision Bio says one Crispr treatment disables human immunodeficiency type 1 viruses, or HIV-1 viruses, preventing their replication, thus stopping further infection.

According to Centers for Disease Control and Prevention, 36,801 Americans were diagnosed with HIV infections in 2019, a decrease of nine percent from 2015. About seven in 10 (69%) of those new cases were gay or bisexual men, with nearly a quarter (23%) heterosexual, and seven percent people who inject drugs. Globally, according to World Health Organization, nearly 38 million people were living with HIV in 2020, with 1.5 million new cases reported.

Functional cure for HIV

The standard of care for HIV infections is antiretorviral therapy, which suppresses and controls, but does not eliminate the virus. Antiretorviral medications must be taken periodically, usually each day, to prevent viral loads from rebounding, since residual levels of HIV remain in infected individuals. Excision Bio says EBT101, its one-time treatment candidate, is a functional cure for HIV.

Excision Bio says EBT-101 works in the body using Crispr to remove proviral DNA, the DNA that enters and integrates with host cells. The company says EBT-101 is guided to the HIV target with RNA, targeting three sites in the HIV genome. The Crispr edits remove the virus’s ability to replicate and minimizes its chance to escape. As reported by Science & Enterprise in December 2020, researchers from Temple University in Philadelphia used Crispr to remove genes gained from infections similar to HIV in lab monkeys. Excision Bio licenses the technology from the lab of Kamel Khalili, professor of virology and neurology at Temple and scientific founder of the company.

“EBT-101 has demonstrated removal of proviral DNA in multiple animal models,” says Excision Bio chief medical officer Lisa Danzig in a company statement released through Globe Newswire, “and offers an opportunity for individuals living with HIV to potentially cease life-long therapies.”

An investigational new drug application officially seeks permission from FDA to ship new drug candidates across state lines to clinical trial sites, in effect requesting permission for a clinical trial. Excision Bio plans a combined early- and mid-stage clinical trial testing the safety, tolerability, and efficacy of EBT-101 in individuals living with HIV, to begin later this year.

More from Science & Enterprise:

*     *     *

Trial Shows VR Training Helps Correct Lazy Eye in Kids

Child with VR headset

(Prashant Sharma, Pixabay. https://pixabay.com/photos/augmented-reality-vr-virtual-reality-3468596/)

14 Sept. 2021. Clinical trial findings show a digital therapy using virtual reality headsets improves vision in children with amblyopia, or lazy eye, more than corrective lenses alone. Results of the trial, testing the Luminopia One system made by Luminopia Inc. in Cambridge, Massachusetts, appear in yesterday’s issue of the journal Ophthalmology.

Amblyopia is a neurological and vision disorder occurring from a breakdown in coordination between one eye and the brain. As a result, the affected eye weakens and often wanders, while the other eye strengthens to compensate. The condition is usually detected in children up to age seven. Amblyopia is a leading cause of decreased vision in children, which according to data cited by Luminopia, affects three percent of children worldwide. Common treatments for amblyopia are wearing an eye patch over the stronger eye to strengthen the affected eye, or wearing prescription glasses to correct impaired vision symptoms.

The Luminopia One system presents videos for children using commercially available virtual reality headsets. The videos — children’s television shows and movies — are digitally altered with algorithms in real time to re-balance visual stimuli between the two eyes. Patients use the system for an hour a day for three to six months. The company says it consulted researchers from Boston Children’s Hospital and the Picower Institute, a neuroscience research center at MIT, in developing the system. Representatives from both institutions are scientific advisors to Luminopia.

The late-stage clinical trial enrolled 105 children at 21 sites in the U.S., age four to seven, diagnosed with amblyopia. Participants were randomly assigned to use a Luminopia One headset for an hour a day, six days a week, for 12 weeks, or continue using their glasses with corrective lenses over this time for comparison. Participants using Luminopia One also wore their corrective-lens glasses at other times.

Greater adherence to headsets than eye patches

The study team looked primarily for changes in scores on standard electronic eye chart tests of visual acuity in both eyes, tested separately. The researchers also measured visual acuity changes after four and eight weeks, and tracked adherence to treatments among Luminopia One users, as well as signs of adverse effects.

The results show more improvement in visual acuity among Luminopia One users than children wearing corrective-lens glasses alone. After 12 weeks, Luminopia One users could read an average of 1.8 more lines on the eye chart tests compared to 0.8 more lines on average for glasses wearers. Moreover, roughly six in 10 (62%) of Luminopia One users advanced two lines or more on eye chart tests after 12 weeks, compared to one-third (33%) of glasses wearers. In both cases, differences were large enough for statistical reliability.

In addition, half or more of the Luminopia One users kept to their prescribed treatment schedules at least 88 percent of the time, which is higher than parents’ reports of previous adherence to eye patches by their children. No serious adverse effects were reported. Because of the consistent differences between treatment and comparison groups, the study team stopped the trial earlier than expected.

“The data from this pivotal trial,” says Robert Langer, biomedical engineering professor at MIT and co-author of the paper, in a company statement released through BusinessWire, “validate Luminopia One as a safe and effective potential new treatment option for amblyopia in young children. These positive data also illustrate the potential of digital therapeutics as a new class of treatments for serious diseases.” Langer is a member of Luminopia’s board.

Luminopia says the Food and Drug Administration is evaluating findings from the trial in its review for medical device clearance of the Luminopia One.

More from Science & Enterprise:

*     *     *

Gene-Edited Treatments in Works for Safer Cancer Therapies

T-cells and cancer cells

Killer T-cells surround a cancer cell (NICHD, Flickr)

13 Sept. 2021. A cancer center and biotechnology enterprise are developing gene-edited therapies working in the gut to reduce adverse effects from cancer immunotherapies. The project brings together M.D. Anderson Cancer Center, part of the University of Texas system in Houston, and the company Snipr Biome in Copenhagen, Denmark.

M.D. Anderson and Snipr Biome aim to validate gut microbe targets and design therapies with the gene-editing technique Crispr that reduce adverse intestinal effects of immune checkpoint inhibitors, a type of immunotherapy for cancer. Immune checkpoint inhibitors block the ability of cancer cells to turn off the immune system, allowing T-cells in the immune system to attack tumors. While checkpoint inhibitors are effective in some forms of cancer, they also are associated with inflammatory bowel diseases, such as colitis, in patients receiving the therapy.

In a study published in July in Nature Medicine, a team led by Jennifer Wargo, professor of genomic medicine and surgical oncology at M.D. Anderson, found about about half of melanoma patients treated with immune checkpoint inhibitors that the team assessed, experienced severe or life-threatening adverse effects. The researchers went on to identify two specific types of gut bacteria produced in patients receiving immune checkpoint inhibitor therapies, who also developed colitis. The cancer therapies addressed CTLA-4 and PD-1 proteins often targeted by immune checkpoint inhibitors.

Snipr Biome develops therapies addressing gut microbes using the gene editing technology Crispr, short for clustered regularly interspaced short palindromic repeats. Crispr is a genome-editing process based on bacterial defense mechanisms that use RNA to identify and monitor precise locations in DNA. The company’s technology selectively delivers edited DNA killing harmful gut bacteria, like an antibiotic, or preventing bacterial infection. Snipr Biome says its Crispr process can also edit DNA to add genes coding for therapeutic proteins or peptides.

Regulate gut microbes to reduce toxicity

Snipr Biome says it uses Crispr’s RNA-guiding property to deliver therapies, either with bacteriophages, a type of virus that infects bacteria, or bacterial conjugation, where one organism transfers material to another through direct contact. The company says it can deliver therapies as oral drugs, injections, or through the skin.

“Treatment with immune checkpoint inhibitors and other forms of immunotherapy,” says Wargo in an M.D. Anderson statement, “has dramatically improved outcomes for patients with cancer, but these therapies can cause significant side effects. Microbes within the gut of patients have been shown to influence responses to immunotherapy, and we now have evidence that they can impact toxicity as well.”

Snipr Biome and M.D. Anderson first plan to validate microbiome targets associated with immune-related adverse effects from immune checkpoint inhibitors. The project team will then develop therapies for regulating gut microbes to reduce toxicity of those therapies. The parties expect to conduct preclinical studies, but also note the potential for a clinical trial. Financial and intellectual property terms of the agreement were not disclosed.

M.D. Anderson is conducting the work under its Program for Innovative Microbiome and Translational Research, or Prime-TR, that studies microbial communities in the body for ways to improve cancer prevention, diagnosis, and treatment. Wargo is director of Prime-TR.

More from Science & Enterprise:

*     *     *

Infographic – US Employer Vaccine Mandates Tick Up

Chart: US employer mandates

Click on image for full size view (Statista)

11 Sept. 2021. A new poll shows a jump in the percentage of American workers now required by employers to get vaccinated against Covid-19. Data from a Gallup survey conducted last month shows about one in five workers (19%) in the U.S. are covered by employer vaccine mandates, triple the percentage reported just two months earlier.

From May through July, five to nine percent of workers said their employers required Covid-19 vaccinations to return to their in-person jobs. Gallup’s latest data in August show 19 percent of employers require vaccination, with another 55 percent encouraging the shots, or about three in four workers (74%) combined. That represents a small increase from the roughly seven in 10 — 71 or 72 percent — from May through July.

The business research company Statista published its chart with the Gallup findings on Thursday. That same day the White House released plans to require employers with more than 100 workers to mandate vaccines or weekly tests of their workforces, as well as federal workers, contractors, and health care staff at facilities receiving Medicare or Medicaid funds. Also that day, the Business Roundtable, a group representing CEOs of large U.S. companies, endorsed the government’s plan.

In response to another Gallup question, about half (52%) of workers in August say they favor employer vaccination mandates, the same percentage as reported in July. However the number of respondents strongly in favor of mandates increased from 36 to 41 percent in August, also 12 points more than in May. The 31 percent strongly opposed to employer mandates in August is the same percentage recorded in May.

Gallup conducted its latest survey 16 to 22 August, with 1,870 individuals working full or part-time, taking part in its nationally representative panel.

More from Science & Enterprise:

*     *     *

Essential Skills For Starting A Business

– Contributed content –

Chart on Imac screen

(Serpstat, Pexels. https://www.pexels.com/photo/silver-imac-displaying-line-graph-placed-on-desk-572056)

10 Sept. 2021. If you are looking into starting your own business, there are a few things you need to consider. It is not something that you should ever take lightly as it requires a lot of work, however, it can turn out to be one of the best things that you will ever do. By being equipped with the right skills, the chance of your business succeeding is a lot greater than if you just try and wing it as you go along. Here are some top skills you should add to your roster when you are looking to start up your own business…

Copywriting

Copywriting is an extremely important skill when you are looking to start a business. Even if you just get up to scratch with basic grammar and how to write compelling copy, then you are onto a winner. You will need copy for everything from populating your website, to writing a press release or pitching emails to potential clients and customers. You might need to create flyers or banners and will want to ensure that your social media is updated and sounds effective. This can all be done through well-written copy that is persuasive, engaging and interesting.

SEO

When you are creating a business, it is important that other people can find it. And this is where an SEO strategy comes in. SEO is essential for getting your business higher on search engines and for new customers to discover you. There are many elements to SEO and further down the line, when you have the budget, it can be a good idea to get an agency to sort it for you. In the short term however, it can be useful to learn the basics so you can do it yourself. This can include things such as optimizing your image sizes, adding in alt tags and filling in all the relevant metadata and descriptions for the pages of your website. Use a tool such as Google Analytics to measure how your website is performing and what might need amending along the way.

Financial management

A key to any business is finances – and this is something you don’t want to end up running out too quickly. You will need to be able to figure out your gains and losses, your profits and how many outgoings you have each month. It is important to be as sustainable as possible and to keep costs to a minimum while also remaining true to yourself and your business model. The last thing you want is to let a customer down due to poor quality services or products.

These are just a few fundamental skills that will really help you on your way. Through learning things such as how to stay on top of your finances, how to give your website the best chance of appearing on search engines and how to write copy that will engage people, you will be in the best possible position. So ask yourself: What are some top skills you find useful to have when running your own business?

*     *     *

Pharma, Synthetic Bio Companies to Find Antibody Therapies

DNA chip graphic

(Gerd Altmann, Pixabay)

10 Sept. 2021. A pharmaceutical company and developer of synthetic genomic materials are collaborating on discovery of therapeutic antibodies to treat a range of diseases. The deal with drug maker Boehringer Ingelheim International GmbH in Ingelheim, Germany could bring Twist Bioscience Corp. in South San Francisco as much as $710 million in milestone payments.

Twist Biosciences develops synthetic genetic materials on a silicon platform, patterned after semiconductors, instead of traditional plastic plates and receptacles. This process, says the company, overcomes conventional limitations and inefficiencies to design and construct genes, and from these synthetic genes, produce libraries of genetic variations. Moreover, Twist Biosciences created a subsidiary, Twist Biopharma, that applies the technology to create collections of synthetic antibodies the company says match genomic sequences occurring in the body.

These collections, says Twist Bio, are made from phage display libraries, protein interactions derived from viruses that infect bacteria called bacteriophages, and a tool used increasingly to discover immunotherapies. Under the agreement, Twist Biopharma will use its collections of phage display libraries, or “library of libraries” as the company calls it, to identify therapeutic antibody candidates that match existing human genomic sequences.

Antibodies that control blood glucose

Twist Bio and Boehringer Ingelheim will collaborate on optimizing and validating these antibody candidates. And Boehringer Ingelheim will gain exclusive worldwide rights to further develop and commercialize antibody therapy candidates discovered in the partnership. Twist Bio will receive an undisclosed initial payment for each antibody program created from the partnership, and as much as $710 million for achieving clinical, regulatory, and commercial milestones for the anticipated multiple antibodies.

As reported by Science & Enterprise in March 2021, Twist Bioscience published a paper on synthetic antibodies it discovered that control blood glucose levels in lab animals. The company discovered these antibodies from a phage display representing some 10 billion receptor proteins, with the researchers focusing on glucagon-like peptide 1, or GLP-1 receptor agonists known to promote insulin production. This screening tool, say the authors, made it possible to find 13 antibody candidates with desirable binding properties and peptide chemistry. Also among the candidates are antibodies that depress GLP-1 receptors, which can help reduce hypoglycemia incidents.

From these antibodies, the Twist Bio team created synthetic antibody drug candidates addressing GLP-1 receptors. And the researchers designed another synthetic antibody, this one acting as an antagonist or reducer of GLP-1 receptors.

Boehringer Ingelheim expects the partnership to add more innovative product candidates to its pipeline. Clive Wood, who heads drug discovery research at the company says in a statement, “Boehringer Ingelheim believes Twist’s ability to generate potent, diverse therapeutic antibodies by mining its comprehensive libraries, combined with our extensive capabilities and experience in drug discovery and development, will enable us to deliver breakthrough opportunities to patients.”

Emily Leproust, Twist Bio’s co-founder and CEO adds, “We have the ability to generate precise antibodies to a diverse range of targets, which together with Boehringer Ingelheim’s strength in drug development capabilities, could mean multiple new, more personalized treatments in the future for patients.”

More from Science & Enterprise:

*     *     *