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Rosh Hashanah 5783

Harvesting apples

(Lumix2004, Pixabay. https://pixabay.com/photos/apples-fruits-orchard-nature-trees-1872997/)

26 Sept. 2022. Science and Enterprise is taking off today to observe Rosh Hashanah, the Jewish new year. We wish everyone of any faith or without, a happy and healthy new year, 5783 in our calendar. We will resume regular posting tomorrow.

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Infographic – Earth Orbits Getting More Crowded

Chart: Increasing space junk24 Sept. 2022. Space may be the next frontier, but it’s quickly becoming a place where you won’t be alone. According to a report issued on 15 Sept. from the Organization for Economic Cooperation and Development or OECD, the amount of debris floating in orbit around the earth has increased markedly in the past two years, and only shows signs of accelerating.

The business research company Statista displays the OECD data in chart form, published on Thursday. The data and chart show in 2021 alone, more satellites were launched into space than in the previous decade. And the increasing density of space junk increases the risk of a phenomenon called Kessler syndrome, where the amount of debris in orbit causes a chain reaction of collisions and even more debris.

Moreover, the rate of new earth-orbit launches is accelerating, with OECD expecting tens of thousands of new satellites launched in the next five years. A major contributor to the crowd is the SpaceX’s Starlink program that plans to launch, in its words, “the world’s first and largest satellite constellation using a low earth orbit to deliver broadband internet.” SpaceX plans to eventually launch thousands of these satellites, with 52 more satellites scheduled for lift-off tonight from Florida.

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NIH Funds Decentralized Rare Disease Data Network

Blockchain

(Gerd Altmann, Pixabay. https://pixabay.com/illustrations/blockchain-data-records-system-3513216/)

23 Sept. 2022. A start-up company is building a secure decentralized network of genomic and other medical data on rare diseases, funded by a fast-track award from an NIH agency. Geneial LLC, a two year-old enterprise in Houston, is receiving an initial $350,000 grant from the National Human Genome Research Institute or NHGRI, part of National Institutes of Health, that’s expected to reach $2.3 million by the project’s end.

Geneial is developing a platform for secure data sharing among researchers studying rare diseases. Almost by definition, rare diseases involve small research efforts and samples, often scattered and independent. These data, such as genomic sequences and health records of individuals with rare diseases and their families, can help advance understanding of their conditions and design of treatments. Yet maintaining and exchanging data on rare diseases still calls for meeting stringent privacy and security requirements under laws in the U.S. and most other advanced countries.

Geneial aims to provide a technology for researchers in rare genetic diseases to aggregate their data sets and collaborate on creating precision diagnostics and therapies. The company’s system is based on so-called Web3 networking, a decentralized Internet technology using blockchain processes to enable encrypted data exchanges without trusted third parties such as encryption keys in conventional methods.

Blockchain captures data about transactions in a networked ledger, but with data distributed among various parties to the transactions. Individual transaction data are broken into blocks, with each block connected in a chain. The blocks are also individually time-stamped and encrypted with an algorithm giving them unique identifiers, also linked mathematically to the previous blocks in the chain. Linking uniquely identified and encrypted blocks in this way ensures integrity of the data and protects the data from hacking.

Community engagement platform

Geneial’s founder Adam Hansen designed a prototype decentralized secure network for rare disease research while earning a Ph.D. in genetics at Baylor College of Medicine in Houston. Working with Baylor’s Human Genome Sequencing Center, Hansen applied the technology to create a registry of Xia-Gibbs syndrome patients for prompting research on that rare disease. Xia-Gibbs syndrome or XGS is a genetic neurological condition marked by weak muscle tone, intellectual disability, and delayed development, resulting from in mutations of the AHDC1 gene. It’s considered a very rare disease, with only the first four cases discovered in 2014 at Baylor. The registry developed by Hansen is now maintained by the Xia-Gibbs Society.

The NHGRI award supports development of a “community engagement platform” based on the XGS patient registry to make possible shareable and interoperable data on rare diseases that researchers can find, access, and reuse. The project calls for starting with the XGS registry to prove the concept of a decentralized secure data network that can be generalized to other rare diseases. In the second phase of the project. Geneial plans to expand the design to two more rare disease communities and research data sets.

“Over 400 million individuals worldwide suffer from a rare disease,” says Hansen in a Geneial statement, “yet only five percent of these have an available treatment. This is largely because data is highly fragmented across silos, which impedes the progress of research and therapeutic development.”

The NHGRI grant is made under NIH’s small business programs that set aside a part of the agency’s research funding for U.S.-based and owned companies. This grant uses a special mechanism called a small business transition grant for early career scientists that both encourages entrepreneurship among new scientists, and also promotes technology transfer from academic labs to business. NIH says it’s a new mechanism, begun in 2021, and combines the usual two steps in small business grants, prototype and scale-up, into a single fast-track award.

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FDA Clearance Requested for Behavioral Diabetes Therapy

Blood glucose test

(Peejhunt, Pixabay)

22 Sept 2022. A digital health company is asking for FDA authorization to market a mobile app that helps treat type 2 diabetes with cognitive behavioral therapy. The app code-named BT-001 is made by Better Therapeutics Inc. in San Francisco, a developer of prescription digital software with treatments for cardiovascular and metabolic disorders.

Better Therapeutics says it applies principles of cognitive behavioral therapy to type 2 diabetes and several cardiovascular and metabolic conditions including nonalcoholic fatty liver disease, nonalcoholic steatohepatitis or NASH, hypertension, and hyperlipidemia. Type 2 diabetes is the most common form of diabetes affecting 90 to 95 percent of people with the disease, which translates to about 37 million people in the U.S. In people with type 2 diabetes, cells in the body do not properly respond to the hormone insulin that transforms glucose or sugar in the blood to energy. If left untreated, type 2 diabetes can lead to serious complications, including heart disease, vision loss, and kidney malfunctions.

Cognitive behavioral therapy or CBT is a form of psychotherapy where a clinician helps a patient verbalize and identify harmful patterns of thoughts and actions, then guide the patient through actions to develop healthier behaviors. CBT often involves testing and restructuring false or negative feelings or beliefs. Better Therapeutics says its software is designed to address cognitive patterns and behaviors associated with diet and associated lifestyle choices. The company says its BT-001 mobile app is the first digital CBT-based therapy for type 2 diabetes delivered directly to patients, without the need for a psychotherapist. However, the app is prescribed to patients by a physician as part of an overall program to treat their type 2 diabetes.

Declines in HbA1c levels at 90 and 180 days

Better Therapeutics tested BT-001 in a late-stage clinical trial, which enrolled 668 individuals age 18 to 75 with type 2 diabetes at six sites in the U.S. Participants in the trial were randomly assigned to use the BT-001 app along with standard medical care for type 2 diabetes, or standard care alone. The study team looked primarily for changes in hemoglobin A1c, or HbA1c, the standard measurement for concentration of glucose attached to hemoglobin in the blood after 90 days, between the two groups. The researchers then tracked participants’ HbA1c levels for another 90 days.

The company released results of the trial on 28 July, reflecting 180 days of data. The findings, published on the Better Therapeutics web site and not peer-reviewed, indicate HbA1c levels declined more for participants using BT-001 plus the standard type 2 diabetes care at both 90 and 180 days, compared to participants receiving standard care alone. The differences in HbA1c levels were large enough for statistical reliability. Plus, more participants receiving standard type 2 diabetes care alone needed to use additional medications to control their blood glucose levels than participants also using BT-001. In addition, participants in the BT-001 group experienced fewer adverse effects, including serious adverse effects, during the trial than participants receiving standard care alone.

Better Therapeutics is asking for the Food and Drug Administration to authorize marketing of BT-001 under the agency’s De Novo classification pathway. The De Novo classification pathway is reserved for new types of medical devices rated at low or moderate risk, where no other comparable devices or systems yet exist.

“As the first digital therapeutic submitted to the FDA for the treatment of type 2 diabetes,” says Better Therapeutics chief medical officer Mark Berman in a company statement, “BT-001 marks a new kind of treatment paradigm with the potential to address the root causes of this and other cardiometabolic diseases.”

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Trial Underway Assessing Gut Microbiome-Cancer Links

Gut microbes

Gut microbes cultured in an artistic design (Nicola Fawcett, Wikimedia Commons)

21 Sept. 2022. A clinical trial began collecting stool and blood samples to find connections between microbial communities in the gut and four types of solid-tumor cancer. The study, conducted by Persephone Biosciences Inc. in San Diego, aims to better understand links between the make-up of a person’s gut microbiome and immune health among cancer patients, for creating new diagnostics and treatments.

Persephone Bio develops engineered biologics addressing disorders in the microbiome, collections of bacteria and other microbes living in the gut. The microbiome is an emerging area for health researchers, with more recent discoveries pointing to links between conditions of microbes in the gut and disorders elsewhere in the body, including those not often associated with the gut.

The five year-old company is currently conducting large-scale observational studies to produce data that connect details of people’s gut microbes with their genomic profiles and health status. Participants provide stool and other samples, as well as permission to access to their health records. Persephone Bio then associates gut microbe compositions with genomic and metabolic conditions, to guide development of therapies, including probiotics and synthetic live biologics that work in the gut. In August, Science & Enterprise reported on the start of a clinical trial collecting infants’ stool samples to produce data on characteristics of healthy microbial communities in young children.

Also collecting data on chemo- and immunotherapies

Persephone Bio says it enrolled the first of 4,000 participants in a study to connect the state and composition of gut microbial communities with four types of solid tumor cancer: colorectal cancer, pancreatic cancer, non-small cell lung cancer, and triple negative breast cancer. Non-small cell lung cancer makes up 80 to 85 percent of all lung cancer cases in the U.S., and triple-negative breast cancer cells have no or few receptors for estrogen, progesterone, nor HER2 proteins, making the cells non-responsive to many hormonal treatments.

Participants in the clinical trial called Argonaut are patients with one of the four types of cancer who give two stool and blood samples within six months, and are then tracked at 12, 18, and 24 months for tumor scans and related health data. At each of the follow-up points, the study team also collects data on cancer medications taken by participants, either chemo- or immunotherapies. The company then plans to apply its computational tools, including machine-learning algorithms, to find associations between gut microbe profiles and immune system biomarkers or molecular indicators of these solid tumor cancers.

“As the largest study ever of its kind in the U.S.,” says Stephanie Culler, CEO and co-founder of Persephone Biosciences, in a company statement released through Globe Newswire, “Argonaut will utilize large-scale, high-throughput data collection coupled with machine learning.”

Persephone Bio says the study plans to enroll a highly diverse group of participants since colorectal cancer affects Black Americans to a greater extent than other racial or ethnic groups. For the colorectal cancer part of the trial, the company is working with Janssen Research & Development, a division of Johnson & Johnson, to also enroll a group of healthy participants but still with a high cancer risk. The microbiome-cancer connections are expected to help develop precision microbiome treatments and companion diagnostics.

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Disclosure: The author owns shares in Johnson & Johnson.

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Challenge Seeks Animal-Free Testing for Precision Meds.

Bioprinting

Bioprinting (Phillip Ezze, Wikimedia Commons. https://commons.wikimedia.org/wiki/File:Bioprinting.jpg)

20 Sept. 2022. A charitable foundation promoting research on human longevity unveiled a challenge competition to develop animal-free testing for personalized medicine. David Gobel, CEO of Methuselah Foundation, announced the $1 million challenge today at the Longevity Summit in Dublin, Ireland, but few other details of the competition are available for participants.

Methuselah Foundation, based in Springfield, Virginia, funds research on human longevity and invests in companies developing technologies supporting that goal. The Animal Free Precision Medicine Innovation Prize, as the competition is called, aims to encourage development of alternatives to animal testing of new drugs that the foundation says adds to the extended time and cost of drug development. “Society can no longer afford the time and cost of bringing new drugs to market by relying on traditional animal testing,” says Gobel in a Methuselah Foundation statement released through Cision, “particularly as we are poised to make broad use of precision medicine to more effectively treat patients.”

Earlier in September, the foundation published a blog post identifying organs-on-chips as a promising alternative to animal testing for toxicity of new drugs. Organs-on-chips are three-dimensional models of organs on miniaturized platforms analogous to semiconductors, thus the reference to chips. In this case, an organ’s functions are simulated with microfluidics that provide fine channels and wells with engineered cells and tissue found in human organs. A review of organs-on-chips published in the journal Nature Reviews Methods Primers in May 2022 outlined a multitude of materials and processes for making the devices.

Focus on bioprinted tissue

The foundation’s blog post notes the proliferation of organizations and companies getting into organs-on-chips and calls for standards to ensure the devices work in predictable ways. The group’s statement suggests bioprinted human tissue is the favored technology, at least for this challenge. “We must accelerate use of bioprinted human tissue,” says Gobel in the foundation’s statement, “to eliminate the harm to animals and humans, improve clinical accuracy and speed our ability to deliver the best patient outcomes.”

Methuselah Foundation is offering $1 million in prize money to encourage new organ-on-chips for animal testing with bioengineered tissue. Chip devices entered in the competition should mimic functions of human organs and contribute to an eventual body-on-a-chip for drug development. Specific prizes, review processes, deadlines, and entry forms are not yet published, but a foundation spokesperson tells Science & Enterprise those details will be available shortly on the Methuselah Foundation web site.

The 6 Sept. blog post also notes the Food and Drug Administration “is evaluating this new testing technology” for its regulatory functions in drugs and food safety. In Oct. 2020, Science & Enterprise reported on an R&D agreement between FDA and the company Emulate Inc. in Boston for the company’s lung chip model to demonstrate mechanisms for immune system protection to prevent Covid-19 infections. In that agreement, the agency also planned to make use of Emulate’s brain, intestine, and liver chips.

Emulate Inc. is a spin-off enterprise from the Wyss Institute, a biomedical engineering center at Harvard University and a pioneer in organs-on-chips. However, organs-on-chips developed by Wyss Institute and Emulate use human cells to line microfluidic channels or organoids grown from adult stem cells, as well as bioprinted tissue.

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ALS Treatment Shown Safe, Affects Key Proteins

neurons

(NIH.gov)

19 Sept. 2022. Results of a clinical trial show a combination of two approved drugs changes levels of proteins associated with ALS, with patients experiencing few adverse effects. Researchers from the biopharmaceutical company Neurosense Therapeutics Ltd. in Herzliya, Israel and Cambridge, Mass. reported the findings last week in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration.

Neurosense Therapeutics develops therapies for neurodegenerative disorders, with its lead product called PrimeC designed as a treatment to limit the progression of ALS. Amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, is a progressive disease where neurons controlling muscles in the body start wasting away, and can no longer send or receive signals from the brain or spinal cord. As neurons cease functioning, muscles in the limbs, and later speech and breathing muscles, weaken and eventually stop functioning. Most people with ALS die of respiratory failure. There are currently no cures for ALS, and few effective treatments for slowing its progression.

PrimeC is Neurosense’s own combination of two drugs, ciprofloxacin and celecoxib, currently approved for other diseases. Ciprofloxacin is an antibiotic prescribed to treat bacterial infections such as pneumonia, gonorrhea, and typhoid fever, as well as biological weapons such as anthrax. Celecoxib is a nonsteroidal anti-inflammatory drug given to relieve pain, stiffness, and swelling from osteoarthritis, rheumatoid arthritis, and ankylosing spondylitis, a form of arthritis affecting the spine. The drug works by blocking cyclooxygenase or COX enzymes that cause pain and inflammation.

Two protein indicators in blood samples

The mid-stage clinical trial enrolled 16 people living with ALS at Sourasky Medical Center in Tel Aviv, Israel, with 15 of those individuals taking part and 12 completing the study. Participants took PrimeC capsules three times a day for 12 months. The trial’s primary goal was to assess the safety of PrimeC in ALS patients, but also reveal chemical changes in participants that suggest the treatments are working as intended.

To assess chemical changes in the body, researchers tested patients’ blood samples for exosomes, natural oil extracellular vesicles or bubbles emitted by neurons, in blood serum. The Neurosense team looked mainly for two protein indicators in the blood serum samples: TAR DNA-binding protein 43, or TDP-43 and light chain 3, or LC3. Accumulations of TDP-43 are common in people with neurodegenerative diseases, including ALS, while LC3 is associated with autophagy, a process for clearing the body of misfolded or aggregated proteins.

Findings show four of the 15 participants experienced adverse effects from PrimeC, with most rated mild or moderate, and none considered serious. At the start of the study, the researchers tested trial participants for their levels of TDP-43 and LC3 proteins compared to matched healthy individuals, which shows ALS patients with more TDP-43 than healthy individuals, but lower levels of LC3. After 12 months, the team found TDP-43 levels decrease in ALS patients, but LC3 levels increase, compared to the start of the study.

“This clinical study is a very important achievement and milestone for the ALS community,” says Vivan Drory, director of neuromuscular diseases at Sourasky Medical Center and principal investigator in a Neurosense Therapeutics statement. “It is very encouraging to see that there was both biological activity and clinical signals of a treatment effect.”

Based on the findings, Neurosense Therapeutics says it began another mid-stage clinical trial, testing PrimeC against a placebo among ALS patients.

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Infographic – Smart Home Technologies Emerge

Chart: Smart home technologies

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

17 Sept. 2022. During the Covid-19 pandemic, the home became much more of a central institution in people’s lives, for families and individuals to do their work, school, and commerce. But even before the pandemic, technologies began emerging to connect the home more extensively than before. Earlier this month, technology intelligence company CB Insights issued a report (registration required) cataloging these smart home technologies, and their likely effects on our daily lives.

The report lists six categories of smart home systems, some of which we explore in Science & Enterprise. For example, CB Insights expects robotics to provide more assistance as well as companionship in the home, which will help older people stay in their homes rather than moving to assisted living facilities. Augmented and virtual reality (AR and VR) are also expected to play a larger role, particularly for conducting collaborative work at home. And dynamic monitoring systems, says the report, are expected to increase comfort for people at home and make their residences more energy-efficient.

While these systems promise a more connected, efficient, and pleasant home, they require continued development of underlying basic technologies, calling for continued research in computer science labs. Artificial intelligence needs to advance for learning and adapting to more complex living patterns and interactions. Plus, smart homes need better security and privacy protocols to protect individuals and families in their highly connected environment.

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Addiction Biotech Using Crowdfunding for Investments

Clock and coins graphic

(Tumisu, Pixabay)

16 Sept. 2022. A start-up enterprise developing treatments for drug and alcohol addiction is raising investment capital with a direct pitch to small investors through a crowdfunding platform. Addiction Cure Therapeutic or ACT Sciences Inc., a two year-old biotechnology company in Orlando, Florida, posted its regulation crowdfunding offering earlier this week.

ACT Sciences develops therapies for alcohol and drug addiction that the company says address more of the causes of addiction rather than the usual strategy of countering changes in brain chemistry of people with addictions. The company says its solutions are based on epigenetics, chemicals in the body and environmental factors outside of DNA sequences that influence gene expression.

As an example, ACT Sciences cites the long-term consumption of alcohol, which contributes to genetic changes leading to greater production of alcohol dehydrogenase, an enzyme that breaks down alcohol into molecules more easily tolerated in the body. As a result, this epigenetic change increases the body’s tolerance of alcohol, allowing for more consumption than before. Alex Federation, the company’s lead scientist, is a chemical biologist and entrepreneur in Seattle who studies epigenomics and bioinformatics.

First addressing meth addiction

ACT Sciences says its first products target cannabinoid type 2 or CB2 receptors that limit reward pathways in the brain, thus decreasing the reinforcing effects of addictive drugs and alcohol. The company says its research revealed an endocannibinoid compound that reduces the reinforcement conditioning induced by methamphetamine and also reduces toxicity to brain cells and cognitive dysfunction from excessive dopamine. Moreover, ACT Sciences says it found similar results from this compound with cocaine, oxycodone, and alcohol, and plans to begin proof-of-concept testing.

“When we started ACTS we wanted to address meth addiction first,” says ACT Sciences co-founder and chief operating officer Mike Lambert in a company statement released through Cision, “it’s the only drug addiction without a replacement drug therapy, let alone a cure.” Lambert adds, “Our first two theories showing promising potential, use endocannibinoid and epigenetic compounds, and are being tested against meth addiction first.”

ACT Sciences is taking advantage of changes in U.S. securities trading laws that makes it possible for small investors to own shares in private companies. The legislation, enacted in 2016, allows for a type of online securities trading called regulation crowdfunding. Privately-owned companies can use regulation crowdfunding to raise up to $5 million a year from small investors that before required high-value accredited investors, such as wealthy individuals, or angel and venture capital investors.

Regulation crowdfunding is permitted only on licensed platforms, in this case NetCapital.com. ACT Sciences posted its offering page on 12 Sept., and as of 16 Sept. raised $2,100, with shares selling at $0.50. A minimum investment of $99 is required, with a closing date of 14 Nov. 2022.

Jack Levine, the company’s co-founder and CEO, notes “we are one of the first biotech companies using regulation crowdfunding to help fund our pursuit of an Rx cure for chemical addiction.”

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Please note this story reports on developments in a science-based company and is not a solicitation for the company’s shares. Readers are encouraged to consult their financial advisors before making any investments.

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Eye Disease Gene-Therapy Biotech Adds Venture Funds

Monitor eyes

(Gerd Altmann, Pixabay)

15 Sept. 2022. A biotechnology company designing treatments for inherited eye diseases with Crispr gene editing is raising €75 million in venture financing. SparingVision, a six year-old business in Paris, is developing genetic therapies for retinitis pigmentosa, other inherited retinal disorders (IRDs), and dry age-related macular degeneration (AMD).

Retinitis pigmentosa or RP is a group of rare diseases of the retina, the light-sensitive tissue at the back of the eye. RP is a progressive disorder, where retina cells degrade and breakdown leading increasing loss of vision over time. According to National Eye Institute, RP symptoms often start in childhood, beginning with loss of night vision. People with RP usually experience a narrowing field of vision, losing peripheral vision, then eventually loss of central vision and blindness. SparingVision cites data showing some 2 million people affected by RP worldwide, including 40,000 in France.

SparingVision designs gene therapies delivered to the retina to treat inherited eye diseases. The company’s lead product, code-named SPVN06, delivers two small proteins called neurotrophic factors naturally produced by photoreceptor cells in the retina, expected to protect remaining photoreceptors and prevent further retinal degradation. The company says SPVN06 is in preclinical testing.

Deliver Cas9 enzymes to remove disease-causing mutations

In Oct. 2021, as reported in Science & Enterprise, the company began a collaboration with the biotech enterprise Intellia Therapeutics, a developer of genetic treatments using the gene editing technology Crispr, short for clustered, regularly interspaced short palindromic repeats. Crispr is adapted from a natural process used by bacteria to protect against attack by viruses, where an enzyme that deactivates or replaces genes binds to targeted RNA molecules generated by the genome. The RNA molecules then guide an editing enzyme, known as Crispr-associated protein 9 or Cas9, to specific genes needing changes.

SparingVision says it uses adeno-associated viruses, or AAVs, to deliver gene therapies to the retina. These viruses are benign, naturally occurring microbes that infect cells, but do not integrate with the cell’s genome or cause disease, and generate at most a mild immune response. With Crispr, the company says AAVs deliver Cas9 enzymes to remove disease-causing mutations in retinal cells, after which AAVs deactivate. SparingVision says its work with Intellia also includes investigating genetic therapy delivery with nanoscale lipid or natural oil particles, a technique used with Covid-19 vaccines.

The company is raising €75 million ($US 75 million) in its second venture funding round. Leading the financing are life science investor Jeita Capital in Paris and UPMC Enterprises in Pittsburgh. Taking part in the round are  4BIO Capital, Bpifrance, the RD fund — venture arm of Foundation Fighting Blindness — and Ysios Capital. Proceeds from the round are expected to advance SPVN06 to clinical trials, as well as its other gene therapies in development. Crunchbase says the company so far raised a total of $127.6 million.

“With this financing,” says SparingVision CEO Stéphane Boissel in a company statement, “we are taking a significant step towards achieving clinical validation of our two lead assets and bringing mutation-agnostic genomic medicines to millions of patients affected by IRDs and dry AMD.”

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