Subscribe for email alerts

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

Donate to Science & Enterprise

Please share Science & Enterprise

RNA Cancer Immunotherapy Shown Feasible in Preclinical Test

Lung cancer illustration

(NIH.gov)

7 Oct. 2022. Tests with lab animals show synthetic viral RNA particles, encased in natural oils, can safely invoke an immune response to attack solid tumors. Results of preclinical tests conducted by the biotechnology company Oncorus Inc. in Andover, Massachusetts, appear in today’s issue of the journal Nature Communications.

Oncorus is a seven year-old enterprise developing cancer therapies with synthetic viruses engineered with RNA to generate an immune response against solid tumor cancers. Up to now, says the company, most cancer immunotherapies are designed to invoke a response from T-cells in the immune system, with mixed results. Some cancer patients, says Oncorus, don’t respond to these therapies, while others who may respond initially, later relapse. The company says much of the problem can be traced to neutralizing antibodies also generated by the immune system, which limit the effects of therapy and prevent subsequent treatments.

The technology designed by Oncorus aims to address these issues. Oncorus engineers natural viruses, such as herpes simplex or Seneca Valley virus, with tumor-specific RNA. These synthetic viral RNA strands are designed to generate responses from multiple immune system cells to attack tumors and degrade the tumor’s protective environment, while sparing healthy cells and tissue. The company says this approach invokes both the innate or general immune system, as well as the adaptive immune system that responds to specific threats.

Infectious viral particles that replicate and spread within tumors

In addition, Oncorus says it delivers its synthetic viral RNA therapies as nanoscale particles packaged in lipids or natural oils. This lipid packaging, says the company, hides synthetic viruses from the immune system long enough for delivery to targeted tumors, to prevent generation of neutralizing antibodies that would otherwise damage the viral therapies. As a result, says Oncorus, its therapies retain their efficacy, making possible multiple treatments for patients.

The Nature Communications paper presents findings from tests of the company’s self-amplifying viral RNA therapies in lab cultures, then given intravenously to mice and monkeys induced with solid tumor cancers. The tests show engineered Seneca Valley virus and Coxsackievirus treatments in lipid nanoparticles or LNPs produce infectious viral particles that replicate and spread within tumors. The researchers tested these engineered viruses with RNA therapies in lab mice grafted with non-small cell lung cancer and melanoma, showing the tumors regress after receiving treatments.

Moreover, the tests show the mice tolerate the treatments, at doses above levels needed to regress tumors, with little body weight change, and minimal effects on their organs. Similar tests with macaque monkeys, infused three times every two weeks with engineered Seneca Valley virus therapies, show delivery of lipid-encased viral nanoparticles to tissues and organs in their bodies, with few adverse effects on those organs. Plus, when challenged with additional neutralizing antibodies, the lipid-encased engineered Seneca Valley virus therapies remain effective in test animals.

Oncorus believes the findings advance the company’s first cancer treatment with this specific technology, code-named ONCR-021. “As demonstrated in animal models outlined here,” says Oncorus vice-president for research Matthew Kennedy and lead author on the paper in a company statement, “we’ve established a way to simultaneously cause direct tumor cell killing in addition to broad immune stimulation in multiple tumors through an IV-administered self-amplifying RNA encapsulated within an LNP. We look forward to progressing our first candidate from this platform, ONCR-021, in patients with non-small cell lung cancer, renal cell carcinoma, melanoma, and hepatocellular carcinoma.”

More from Science & Enterprise:

We designed Science & Enterprise for busy readers including investors, researchers, entrepreneurs, and students. Except for a narrow cookies and privacy strip for first-time visitors, we have no pop-ups blocking the entire page, nor distracting animated GIF graphics. If you want to subscribe for daily email alerts, you can do that here, or find the link in the upper left-hand corner of the desktop page. The site is free, with no paywall. But, of course, donations are gratefully accepted.

*     *     *

 

Nasal Spray Vaccines Get CEPI, NIH Funding

Nasal spray

(Wikimedia Commons)

6 Oct. 2022. A developer of infectious disease vaccines is gaining funds for new nasal sprays protecting against a range of coronaviruses and the sexually-transmitted disease gonorrhea. Intravacc B.V. in Bilthoven, the Netherlands, received a grant from the Coalition for Epidemic Preparedness Innovations or CEPI, an international health partnership based in Oslo, Norway, and a contract from National Institute of Allergy and Infectious Diseases or NIAID, part of U.S. National Institutes of Health.

Intravacc is a biotechnology company that designs protective and therapeutic vaccines with several processes that address a variety of infectious diseases and cancer. Many of their vaccines are developed without water, using freeze- or spray-drying, which the company says makes possible more stable vaccines that can be given without a syringe, and stored and shipped without refrigeration.

One of those processes creates round nanoscale particles resembling naturally emitted spheres from gram-negative bacteria. These outer membrane vesicles, as they’re called by Intravacc, contain many bacterial antigen proteins in their natural state, and are engineered by the company to express other needed properties, such as faster cell uptake, additional immune response without an adjuvant, and lower toxicity. In Jan. 2022, Science & Enterprise reported on a clinical trial in the Netherlands testing an Intravacc nasal spray vaccine protecting against Covid-19 infections developed with the outer membrane vesicle process.

The award of up to $4.8 million from CEPI funds work by Intravacc on a vaccine that protects against a broad range of betacoronaviruses, a collection of coronaviruses including the now notorious SARS-CoV-2 responsible for Covid-19 infections, as well as the earlier SARS-CoV-1 that emerged in China in 2003, and Middle East respiratory virus or MERS-CoV first found in Saudi Arabia in 2012. Betacoronaviruses are notable for their zoonotic property, the ability to jump from animals to humans.

Common sexually-transmitted disease among young adults

Intravacc plans to use the CEPI funds to advance its candidate nasal spray code-named Avacc 101 through preclinical stages as a broad-range betacoronavirus vaccine. The company says Avacc 101 will be designed to generate immune responses to a universal betacoronavirus spike protein, as well as T-cells from the immune system, in nasal mucous membranes to prevent personal infections and spread of the virus to others.

“To secure the gains we’ve made,” says CEPI CEO Richard Hatchett in a statement, “we must continue to develop vaccines that provide broad protection against these variants to mitigate the need for regular variant boosters, and which can also provide protection against other, more lethal, coronavirus threats including MERS-CoV.”

Yesterday, Intravacc announced NIAID is awarding the company a $14.6 million contract to develop a nasal spray vaccine against gonorrhea, a common sexually-transmitted disease among young adults. The agency is funding some $2 million for work through October 2023. The new vaccine, says Intravacc, will be designed with engineered outer membrane vesicles to protect against infections from Neisseria gonorrhoeae or NG bacteria responsible for the disease.

The company’s vaccine code-named NGoXIM is made with outer membrane vesicles from NG bacteria, enhanced with microspheres containing the human immune system cytokine interleukin-12. Intravacc says NGoXIM is already in preclinical testing, with the company expected to advance the vaccine to produce doses for an early-stage clinical trial in healthy adults.

More from Science & Enterprise:

We designed Science & Enterprise for busy readers including investors, researchers, entrepreneurs, and students. Except for a narrow cookies and privacy strip for first-time visitors, we have no pop-ups blocking the entire page, nor distracting animated GIF graphics. If you want to subscribe for daily email alerts, you can do that here, or find the link in the upper left-hand corner of the desktop page. The site is free, with no paywall. But, of course, donations are gratefully accepted.

*     *     *

 

Yom Kippur 5783

Shofar

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

5 Oct. 2022. We’re observing Yom Kippur today, the day of atonement and holiest day in the Jewish calendar. Regular posting will resume tomorrow, Thursday, 6 Oct.

*     *     *

 

Algae-Grown Non-Needle Malaria Vaccine in Preclinical Test

Malaria clinic

Malaria clinic in Mali (USAID.gov)

4 Oct. 2022. A vaccine grown in genetically-altered algae, designed as a nasal-spray and edible drug, is shown to protect lab mice against parasite infections causing malaria. Researchers from the biotechnology company Lumen Bioscience Inc. in Seattle and University of Washington describe the vaccine and their findings in today’s issue of the journal NPJ Vaccines.

According to World Health Organization, malaria in 2020 affected some 241 million people worldwide leading to 627,000 deaths, with 95 percent of cases occurring in sub-Saharan Africa. Children under the age of five are particularly vulnerable. The disease is caused by infections from the Plasmodium parasite, transferred to humans by bites from female Anopheles mosquitoes. Current malaria vaccines are injected with syringes, and require refrigeration during transport and storage, which is difficult in parts of the world most affected by the disease.

Lumen Bioscience is a five year-old company developing biologic drugs with a process it says is simpler and less expensive than conventional techniques for both producers and consumers. Lumen Bio says it genetically engineers the blue-green algae spirulina to express therapeutic molecules, with the altered spirulina producing and storing the molecules inside their cells. The engineered algae cells are grown and produced into biomass from water, salt, carbon dioxide, and light, with the biomass then dried into a powder.

Complex shape-shifting organisms

The company says the therapeutic molecules are preserved inside surviving cell membranes, which allows the powder to be packed inside capsules, and taken as oral drugs. The capsules, says Lumen Bio, can be shipped and stored at room temperatures. Once swallowed, the capsules are protected against degradation in the stomach, and release their therapeutic payloads in the small intestine, where they neutralize the targets. In April 2022, Science & Enterprise reported on FDA clearance for a clinical trial of a Lumen Bio treatment for Clostridium difficile or C. difficile infections, often contracted in health care facilities.

Researchers from Lumen Bio and the lab of microbiologist Sean Murphy at University of Washington applied the Lumen process to produce a vaccine to protect against Plasmodium parasite infections. Murphy and colleagues study the Plasmodium parasite, noting that the shape-shifting organisms are complex and dangerous, with the ability to live in multiple environments in mosquitoes and humans. For this study, the team genetically altered spirulina cells to express virus-like particles with a Plasmodium protein antigen on their surface.

The researchers tested the algae-grown vaccine in lab mice as a nasal spray, with an edible booster fed to the mice. The team found the nasal spray followed by edible boosters are well tolerated by mice, and produce antibodies with the ability to neutralize Plasmodium proteins. The researchers also found female mice produce higher concentrations of anti-Plasmodium antibodies after the nasal spray and edible boosters, while male mice need an extra temperature-stable adjuvant to achieve similar antibody concentrations. In subsequent challenge tests against Plasmodium proteins delivered under the skin to simulate mosquito bites, the combination of nasal spray and edible booster protect female mice against infection, with the vaccines plus adjuvant protect male mice.

“Tremendous progress has been made in the field of recombinant malaria vaccines,” says Murphy in a Lumen Bioscience statement, “but real-world access remains challenging due to infrastructure and supply chain limitations and to cost sensitivity in most regions where malaria is endemic.” James Roberts, Lumen Bio’s chief scientist and co-author of the paper notes, “The use of edible spirulina as a malaria vaccine platform offers a new approach to vaccine development at scales, costs, and delivery that could greatly improve access compared with traditional vaccination approaches.”

More from Science & Enterprise:

We designed Science & Enterprise for busy readers including investors, researchers, entrepreneurs, and students. Except for a narrow cookies and privacy strip for first-time visitors, we have no pop-ups blocking the entire page, nor distracting animated GIF graphics. If you want to subscribe for daily email alerts, you can do that here, or find the link in the upper left-hand corner of the desktop page. The site is free, with no paywall. But, of course, donations are gratefully accepted.

*     *     *

 

Patent Issued for Crispr-Edited Viruses in Microbiome

Crispr-Cas9

Crispr editing with Cas9 enzyme (Broad Institute, NIH)

3 Oct. 2022. A biotechnology company developing gene-edited therapies for bacterial infections in the gut received a U.S. patent for viruses engineered as treatments. Eligo Bioscience says the U.S. Patent and Trademark Office or USPTO granted patent number 11,452,765 to inventors at Rockefeller University in New York for their Crispr technology with engineered viruses, licensed exclusively to the company.

Eligo Bioscience, based in Paris, designs engineered bacteriophage viruses that are natural enemies of bacteria. Bacteriophages, or phages, are viruses that infect and destroy bacteria with lysis, a process for breaking through outer cell membranes. The company says it genetically edits phage viruses with Crispr, short for clustered regularly interspaced short palindromic repeats, to precisely target disease-bearing bacteria in the gut, bypassing beneficial bacteria in the microbiome. Eligo says RNA guiding the gene-editing payloads finds matching RNA sequences in the bacterial community, then unleashes the editing enzymes to break up the bacterial DNA. Gut microbes not matching the targeted RNA are left alone.

The patent, issued on 27 September 2022 covers techniques for reducing disease-causing or antibiotic-resistant bacteria found mixed with healthy or beneficial bacteria in the gut, using Crispr to target the precise DNA sequences for attack. The company says the new patent is the latest of three documents protecting its intellectual property, based on research at Rockefeller University on using Crispr to find and destroy harmful bacteria in the gut. The earlier two documents cover genetically modified phagemids, circular non-replicated DNA molecules called plasmids found in phage viruses, while the latest patent applies to replicable engineered phage viruses.

Patents released to academic and not-for-profit labs

Eligo Bio and Snipr Biome, a biotechnology company in Copenhagen are embroiled in a patent dispute going back to 2020. Snipr Biome is also developing Crispr-edited phage viruses to attack antibiotic-resistant bacteria, such as E. coli in the gut, among patients with blood-related cancers. Science & Enterprise reported in April 2022 on an early-stage clinical trial testing the safety of Snipr Biome’s gene-edited phage viruses in healthy volunteers. In Dec. 2021, the Patent Trial and Appeal Board at USPTO ruled that Snipr Biome interfered with Eligo’s patents first granted to Rockefeller University, indicating that five of Snipr Bio’s patents benefited unfairly from the university’s prior inventions.

“Each additional patent granted in this family, which has the earliest priority date in the field of Crispr-based killing of prokaryotes,” says Eligo Bioscience CEO Xavier Duportet, referring to single-cell organisms with a nucleus such as bacteria, in a company statement released through Cision, “further strengthens Eligo’s dominant patent position. Eligo is definitively a key partner to any entity intending to use Crispr to kill bacteria.”

For its part, Snipr Biome announced today it’s making available its patent portfolio of Crispr-based technologies to edit prokaryotes to academic and not-for-profit research labs without a written license. The company says this release covers more than 20 patents applied to technologies for cancer and immunology therapies.

More from Science & Enterprise:

We designed Science & Enterprise for busy readers including investors, researchers, entrepreneurs, and students. Except for a narrow cookies and privacy strip for first-time visitors, we have no pop-ups blocking the entire page, nor distracting animated GIF graphics. If you want to subscribe for daily email alerts, you can do that here, or find the link in the upper left-hand corner of the desktop page. The site is free, with no paywall. But, of course, donations are gratefully accepted.

*     *     *

 

Infographic – Biotech Index Q3 Rollercoaster

NBI Year to date 30 Sept 2022

Click on image for full-size view. (Nasdaq)

1 Oct. 2022. It’s time again for our quarterly look at the Nasdaq Biotechnology Index, an indicator of investor sentiment in science-based businesses. For six weeks after 30 June, NBI rose about 14 percent to close at 4,281 on 15 Aug., but then bounced up and down over the next six weeks to close yesterday (30 Sept.) at 3,768, slightly higher from where it started for the quarter at 3,749.

For the year-to-date, as noted in today’s chart, NBI shows a general decline, losing more than one-fifth of its value since January. While the NBI’s close yesterday at 3,749 is somewhat of a rebound from the low for the year on 13 June at 3,368, the overall trend for the year is a decline from opening on 3 Jan. at 4,729. By comparison, the Nasdaq Composite Index, an indicator of investor sentiment in the overall technology sector, is down about one-third for the year.

Nasdaq computes NBI from share prices of 365 biotech and pharma companies publicly traded on its exchange. The Nasdaq Composite Index is calculated from share prices of 3,762 companies, nearly all stocks traded on the exchange.

More from Science & Enterprise:

We designed Science & Enterprise for busy readers including investors, researchers, entrepreneurs, and students. Except for a narrow cookies and privacy strip for first-time visitors, we have no pop-ups blocking the entire page, nor distracting animated GIF graphics. If you want to subscribe for daily email alerts, you can do that here, or find the link in the upper left-hand corner of the desktop page. The site is free, with no paywall. But, of course, donations are gratefully accepted.

*     *     *

 

Start-Up Developing Efficient RNA Process, Raises $12M

Vials and bottles

(National Cancer Institute, Unsplash)

30 Sept. 2022. A company formed earlier this year is commercializing a more efficient process for making synthetic RNA molecules and raising $12 million in seed funds. EnPlusOne Biosciences Inc. in Watertown, Massachusetts is spun-off from labs at the Wyss Institute for Biologically Inspired Engineering, a research center at Harvard University.

EnPlusOne Biosciences offers a process for synthesizing ribonucleic acid, or RNA, that carries instructions from genetic codes in DNA for producing proteins in cells. Synthetic RNA is used increasingly in therapies, showcased by the recent rapid development and efficacy of Covid-19 vaccines based on programmed synthesized messenger RNA. For treatments or vaccines, RNA is produced in short sequences called oligonucleotides, often used in diagnostics, but also serve as building blocks for more complex synthesized RNA chemistry.

As explained last year by EnPlusOne Bio co-founder Daniel Wiegand in a Wyss Institute blog post, current processes for synthesizing RNA oligonucleotides work fine for producing small quantities in the lab or limited clinical use, but are difficult to scale-up for population-wide public health or more complex chemical requirements. Instead of the current phosphorous-based chemical methods, EnPlusOne Bio employs a synthesis process with naturally occurring enzymes to produce RNA molecules. Wiegand says in the Wyss Institute post, “we believe that we can produce oligonucleotides more cheaply at substantially higher purities and commercially relevant scales.”

“A clear vision for effectively delivering RNA to the world.”

EnPlusOne Bio says it can also design synthetic RNA oligonucleotides in longer strands than currently produced, and in novel configurations to meet the needs of industry partners. Plus the company plans to develop a manufacturing facility in the near future. “Our platform,” adds Wiegand in a company statement released through BusinessWire, “represents a new way of addressing this need utilizing next-generation technology for the scaled production of high-quality RNA oligonucleotides. We’ve made significant progress developing this technology, and we have a clear vision for effectively delivering RNA to the world.”

Wiegand designed the EnPlusOne process as a Wyss Institute research scientist in the lab of geneticist and serial entrepreneur George Church, a co-founder of the company with Wiegand, biochemist Jonathan Rittichier, and biotech engineer Dan Ahlstedt. Wiegand is EnPlusOne Bio’s CEO, while Rittichier is chief scientist, Ahlstedt is chief operating officer, and Church is an advisor. “I remember bringing Daniel and Jon together to tackle this challenge,” notes Church, “and how enthusiastic they were working through it. Our lab culture is driven by seeing every bit of basic science for how it might be stretched and recombined to solve a societal problem.”

EnPlusOne Bio was formed earlier this year under Northpond Labs, a joint undertaking of Northpond Ventures, a life science and health technology investor in Cambridge, Mass., that supports commercially feasible technologies being developed at the Wyss Institute. In May 2022, Science & Enterprise reported on SomaCode, a Northpond Labs/Wyss Institute initiative to extend the reach and targeting of cell therapies. Northpond Ventures is leading the seed round for EnPlusOne Bio raising $12 million, joined by Breakout Ventures, Coatue, and angel investors. The company expects to apply the proceeds to further develop its synthetic RNA process.

More from Science & Enterprise:

We designed Science & Enterprise for busy readers including investors, researchers, entrepreneurs, and students. Except for a narrow cookies and privacy strip for first-time visitors, we have no pop-ups blocking the entire page, nor distracting animated GIF graphics. If you want to subscribe for daily email alerts, you can do that here, or find the link in the upper left-hand corner of the desktop page. The site is free, with no paywall. But, of course, donations are gratefully accepted.

*     *     *

 

Stem Cell Lab Planned for Commercial Space Station

Orbital Reef

Orbital Reef illustration (Sierra Space)

29 Sept. 2022. A collaboration between university researchers and a space science company is designing a stem cell research lab on a proposed commercial space station. The partnership between Sierra Space in Louisville, Colorado and the Sanford Stem Cell Clinical Center at University of California in San Diego expands on the university’s current studies of stem cell development in microgravity.

Sierra Space is one of the lead developers of Orbital Reef, a commercial space station designed for low-Earth orbit that aims to offer a business park for space-based commerce, research, and tourism. Blue Origin, the other lead developer for Orbital Reef, is providing reusable launch vehicles for the project, while Sierra Space provides its habitation modules and Dream Chaser space plane, a winged spacecraft similar to the NASA space shuttle. Orbital Reef is expected to become operational in 2027, orbit 500 kilometers (311 miles) above the Earth, and accommodate up to 10 people in living space comparable to the International Space Station.

The Sanford center at UC San Diego studies treatments for regenerative medicine with stem cells, including clinical trials of stem cell therapies. In April 2020, the Sanford center began a collaboration with the company Space Tango to begin studies of stem cell development in microgravity on the International Space Station or ISS. That project became the Integrated Space Stem Cell Orbital Research, or ISSCOR, funded by a $5 million NASA grant. As reported by Science & Enterprise at the time, the UC San Diego researchers are designing experiments to be conducted in weightless conditions on stem cells derived from blood and immune-system cells, looking for changes in biomarkers indicating cellular malfunctions associated with cancer.

Research labs and biomanufacturing

“Microgravity and radiation exposure in low-Earth orbit,” says Catriona Jamieson, director of the Sanford center in a Sierra Space statement, “offers a unique opportunity to study stem cell aging and pre-cancer development in a compressed time frame in a manner that is unavailable on Earth.” Jamieson adds, “We are learning things that we never could under normal gravity; knowledge that can elevate the search for new pre-cancer diagnostics and therapeutics that eradicate cancer at its earliest stages into addition to a broad array of degenerative diseases that arise as a result of stem cell dysfunction.”

Under the new agreement, Sierra Space and the Sanford center are collaborating on extending the ISSCOR labs into Orbital Reef. A UC San Diego team is contributing design and operational planning expertise for stem cell research labs, as well as biomanufacturing and fabrication on the new space station. The university is also taking part in a biomanufacturing research consortium led by Sierra Space, for advice on R&D objectives and technical requirements. Sierra Space is responsible for development of the labs and biomanufacturing facilities, as well as their launch and deployment.

“As the ISS completes its time in service,” notes Sierra Space CEO Tom Vice, “UC San Diego will now have a place to grow and expand its vital research in biotech and biopharma with full, on-orbit biomanufacturing and biofabrication centers to foster breakthrough advancements and products in medical science that will benefit all life on Earth.”

More from Science & Enterprise:

We designed Science & Enterprise for busy readers including investors, researchers, entrepreneurs, and students. Except for a narrow cookies and privacy strip for first-time visitors, we have no pop-ups blocking the entire page, nor distracting animated GIF graphics. If you want to subscribe for daily email alerts, you can do that here, or find the link in the upper left-hand corner of the desktop page. The site is free, with no paywall. But, of course, donations are gratefully accepted.

*     *     *

 

Company Formed to Boost Clinical Trial Success Odds

Human machine interface

(Gerd Altmann, Pixabay. https://pixabay.com/illustrations/digitization-particles-smartphone-7261158/)

28 Sept. 2022. A pharma/tech industry group is starting a new company to use algorithms for finding gaps in preclinical data that can impair clinical trial progress. Omec.Ai is the start-up enterprise being formed by AION Labs in Rehovot, Israel, a joint venture of pharmaceutical companies AstraZeneca, Merck, Pfizer, and Teva, with the Israel Biotech Fund and Amazon Web Services.

AION Labs aims to apply artificial intelligence and other computational techniques across the pharmaceutical industry to find solutions to long-standing problems affecting industry performance. Omec.Ai, says AION Labs, is the first new company formed by the consortium, in this case to improve the odds of success for treatment candidates in clinical trials, based on the data generated in preclinical studies. Even with advanced analytics from data produced through genomics and related “omics” technologies with tissue and single-cell profiling, says AION Labs, many drug candidates still fail in clinical trials, due to safety issues or efficacy shortfalls.

AION Labs traces some of the continuing problems to lack of consistency in preclinical data, making it difficult for biotech and pharma companies to assess the predictive value of preclinical findings. “There is currently no automated solution,” says Omec.Ai co-founder and CEO Ori Shachar in an AION Labs statement released through Cision, “that employs all preclinical data in a way that allows a reliable assessment of the clinical trial readiness of a drug candidate. We are aiming to fill this gap.”

New business formed to create proposed solution

AION Labs holds crowd-sourced challenge competitions to identify promising teams and solutions to intractable issues in drug development. The consortium works with BioMed X, a research institute in Heidelberg, Germany that sponsors crowd-sourced studies in molecular, cell, and computational biology, and diagnostics by scientists in academic labs and start-up companies. The winning entry is selected to form a new business to pursue its solution, with funding, mentorship, and datasets provided by the sponsors.

In 2021, Science & Enterprise reported on two other AION Labs challenges, to apply computational techniques to design and development of antibodies. In Feb. 2021, AION Labs put out a call for a new A.I. technology that enhances existing antibody treatments. And in Oct. 2021, AION Labs issued a challenge for algorithms that help design new synthetic antibodies.

AION Labs opened the competition for preclinical data assessment in December 2021, sponsored by drug makers Pfizer, Merck, and AstraZeneca that are also the primary investors in Omec.Ai. The Israel Innovation Authority that promotes industrial R&D in the country is also an investor. In addition, the pharmaceutical companies are providing data sets to help build and train machine learning algorithms. Plus, Amazon Web Services is providing technical resources.

Omec.Ai is still in formation. “With the support of AION Labs and its partners,” adds Shachar, “we hope to develop a cutting-edge solution to significantly improve the probability of success of drug candidates that make it to the clinical trial phase.”

More from Science & Enterprise:

Disclosure: The author owns shares in Pfizer.

We designed Science & Enterprise for busy readers including investors, researchers, entrepreneurs, and students. Except for a narrow cookies and privacy strip for first-time visitors, we have no pop-ups blocking the entire page, nor distracting animated GIF graphics. If you want to subscribe for daily email alerts, you can do that here, or find the link in the upper left-hand corner of the desktop page. The site is free, with no paywall. But, of course, donations are gratefully accepted.

*     *     *

 

Start-Up Licenses Gene-Edited Drug Discovery Tech.

African clawed frog

African clawed frog (Brian Gratwicke, Flickr. https://flic.kr/p/dFHy8R)

27 Sept. 2022. A new company is acquiring a technology using genome-edited tadpoles that finds current drugs addressing precise genetic conditions underlying rare diseases. Unravel Biosciences, a one year-old enterprise in Boston, is licensing the technology from the Wyss Institute for Biologically Inspired Engineering, a research center at Harvard University.

The technology called CogniXense is developed by researchers at Wyss Institute and Tufts University, and seeks to discover drugs meeting a rare-disease patient’s unique medical conditions, defined by symptoms and RNA transcribed from that person’s genetic code. The technology first creates computational models of the disease focusing on anomalies in the RNA transcribed from a patient’s genes. Next, CogniXense extends those algorithms to screen currently approved drugs with potential to address the identified transcription anomalies for restoring health to the patient.

The technology then uses the genome editing technique Crispr to genetically engineer tadpoles from the Xenopus laevis or African clawed frog species, to reflect the patient’s symptoms and genetic conditions. African clawed frogs are a common model organism and widely studied in biology labs because of their rapid development cycle. In this case, the tadpoles’ response to the identified drugs are observed and assessed in neurological tests, such as ability to learn and navigate simple mazes. CogniXense also tests for possible toxicities.

The Wyss Institute researchers, led by Richard Novak, then a staff engineer, applied CogniXense to Rett Syndrome as a test case. Rett syndrome is a genetic neurological disorder affecting girls caused by a mutation in the MECP2 gene. The disease occurs in 1 in 9,000 to 10,000 female births, with symptoms somewhat similar to autism, including language and communication problems, learning difficulties, and lack of coordination. The MECP2 gene gives instructions for making a protein needed to maintain synapses or connections between neurons in the brain.

“Computationally predict which existing drugs could restore health.”

The Rett syndrome case shows the CogniXense algorithms can replicate behavioral differences between healthy individuals and those with Rett syndrome, as well as identify transcription characteristics of drug candidates for reversing symptoms of the disease. When tested with genetically edited tadpoles to display Rett syndrome characteristics, CogniXense identified several drugs that reverse symptoms of the disease.

Harvard University is granting Unravel Biosciences an exclusive license to apply and commercialize the technology for prevention, diagnostics, and treatment of specified but undisclosed neuro-developmental disorders, with new and current drugs discovered by the technology. Financial details of the license agreement were also not disclosed.

Novak founded Unravel Bio in Sept. 2021 with Wyss Institute director Donald Ingber, staff scientist Frederic Vigenault, and Tufts biology professor Michael Levin, also an associate faculty member at Wyss Institute. Novak and Vigenault are the company’s CEO and chief scientist respectively, while Ingber and Levin are advisors to the company.

“We computationally predict which existing drugs could restore health in that patient,” says Novak in a Wyss Institute statement, “validate those drugs in our engineered animal models and focused clinical trials, and from there identify the underlying molecular targets that could be drugged to treat the disease across patient populations.”

Unravel Bio now calls its platform BioNav, with algorithms trained by more than 12,000 gene interactions and some 40,000 drug compounds. The company says it can produce Crispr-edited tadpole models of disease and screen for relevant solutions in about three weeks.

More from Science & Enterprise:

We designed Science & Enterprise for busy readers including investors, researchers, entrepreneurs, and students. Except for a narrow cookies and privacy strip for first-time visitors, we have no pop-ups blocking the entire page, nor distracting animated GIF graphics. If you want to subscribe for daily email alerts, you can do that here, or find the link in the upper left-hand corner of the desktop page. The site is free, with no paywall. But, of course, donations are gratefully accepted.

*     *     *