Neurona Raises $120M, Buoyed by Early-Stage Data for Epilepsy Cell Therapy – BioSpace

Pictured: 3D illustration of stem cells used for cell therapy/iStock,Maksim Tkachenko

Neurona Therapeutics on Thursday announced that it has secured $120 million in funding, which will help it advance its pipeline of investigational off-the-shelf cell therapies.

The California-based biotech will use the money to advance its lead candidate NRTX-1001, a regenerative neural cell therapy candidate being assessed in a Phase I/II trial for drug-resistant mesial temporal lobe epilepsy (MTLE), the most common form of focal epilepsy that can trigger ongoing seizures despite medical treatment.

Neuronas financing on Thursday was co-led by Viking Global Investors and Cormorant Asset Management. Other institutional supporterssuch as UC Investments, UCB Ventures, Euclidean Capital, The Column Group and Alexandria Venture Investmentsalso participated in the funding round.

Neurona has pioneered development of a fully-differentiated cell therapy for drug-resistant focal epilepsy that is designed to be disease-modifying, repairing the affected neural network, and is yielding very promising initial clinical data, Raymond Kelleher, managing director at Cormorant, said in a statement, adding that NRTX-1001 could be a game-changer with its potential to control seizures and preserve neurocognitive function.

According to the biotechs website, NRTX-1001 is a cell therapy comprised of human inhibitory GABAergic interneurons, which can potentially address the underlying hyperactive neural networks in epileptic seizures.

In December 2023, at the annual meeting of the American Epilepsy Society, Neurona presented data from two patients enrolled in the Phase I/II study of NRTX-1001, who saw at least a 95% drop in overall seizure frequency more than one year after treatment. NRTX-1001 was also safe overall, with adverse events categorized as mild to moderate in severity.

Neurona is also testing NRTX-1001 in Alzheimers disease, for which it is currently in the preclinical evaluation stage.

Beyond NRTX-1001, Neurona will also channel some of Thursdays haul into its other pipeline projects including investigational myelinating glial cells and gene-edited cells, both for yet-undisclosed indications.

Neuronas funding comes nearly a year after the biotech laid off 18 employees, or 25% of its total headcount, to streamline its budget in the face of the current tight funding environment and extend available cash to support the ongoing clinical trial of NRTX-1001, a spokesperson told Fierce Biotech at the time.

The financing could also be a sign of an uptick in the industrys fundraising activities. This week alone, there have been two initial public offeringsKyverna and Metagenomiwhich follow the earlier debuts of Alto Neuroscience, Fractyl Health, ArriVent Biopharma and CG Oncology.

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

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Neurona Raises $120M, Buoyed by Early-Stage Data for Epilepsy Cell Therapy - BioSpace

Unlocking the power of stem cell therapy – Drug Target Review

In this Q&A, founder of Scheer Medical Wellness Dr Alexander Scheer shares his insights, discussing the current challenges in stem cell therapy and how these can be addressed, the advancements in delivery techniques, and stem cells overall potential in regenerative medicine.

Adipose-derived stem cells (ADSCs) have garnered attention due to their remarkable ability to differentiate into various cell types crucial for tissue regeneration and repair. Their plasticity allows them to adapt to the microenvironment of host tissues, facilitating integration and functional restoration. Additionally, ADSCs possess immunomodulatory properties, enabling them to modulate immune responses and promote tissue healing. These characteristics position ADSCs as promising candidates for addressing a wide range of medical conditions, from degenerative diseases to traumatic injuries.

Despite their potential, ADSC therapy faces several challenges in preclinical studies. These include ensuring controlled differentiation of ADSCs into desired cell types, addressing safety concerns such as the risk of tumorigenicity or immunogenicity, and optimising large-scale production methods. To overcome these challenges, researchers employ rigorous preclinical assessments, refine culture techniques to enhance cell purity and functionality, and develop standardised protocols for consistent results.

Researchers commonly use a combination of in vitro cell culture assays and in vivo animal models to assess the therapeutic potential of ADSCs. While in vitro assays provide controlled environments for studying cellular behaviour and differentiation, in vivo models offer insights into tissue-level interactions and responses. However, translating findings from these models to clinical trials requires careful consideration of factors such as disease complexity, species differences, and the relevance of model systems to human physiology.

Recent advancements in delivery techniques have significantly enhanced the precision and effectiveness of ADSC treatments. Biomaterial-based scaffolds provide structural support and mimic the extracellular matrix, facilitating tissue regeneration and integration. Additionally, innovations in 3D bioprinting enable the fabrication of complex tissue structures, offering tailored solutions for patient-specific needs. These delivery techniques improve cell retention, viability, and functionality post-transplantation, maximising therapeutic outcomes.

ADSC therapy holds promise for a multitude of medical conditions, including but not limited to wound healing, organ repair, autoimmune disorders, and cancer treatment. In wound healing, ADSCs accelerate tissue regeneration and reduce scarring, offering hope for chronic wound management. Similarly, in organ repair, ADSCs contribute to tissue regeneration and functional restoration, potentially revolutionising treatments for conditions such as myocardial infarction and liver cirrhosis. Moreover, their immunomodulatory properties make them valuable assets in managing autoimmune diseases by suppressing aberrant immune responses. Furthermore, ongoing research explores the potential of ADSCs in cancer therapy, both in supporting conventional treatments and directly targeting cancer cells. Overall, ADSC therapy represents a versatile and promising approach in personalised and regenerative medicine, with the potential to improve patient outcomes and quality of life across diverse medical fields.

Author bio

Dr Alexander Scheer

Founder of Scheer Medical Wellness

Dr Alexander Scheer, the founder of Scheer Medical Wellness, is a New York-based physician, with more than 20 years of expertise. He serves as the Medical Director (MD) of Scheer Medical Wellness, a cutting edge practice which provides a comprehensive range of high-quality services. These services encompass pain management, neurosurgery, spinal surgery, orthopedic surgery, primary care, physical therapy, physical medicine and rehabilitation, podiatry, plastic surgery, acupuncture, weight loss management, gastroenterology, and sports medicine.

Dr Scheer earned his medical degree from New York Medical College and subsequently completed his Surgical internship at Mount Sinai Beth Israel, followed by training in neurological surgery at the Albert Einstein College of Medicine until 2009. Dr Scheer is licensed to practice medicine in the state of New York and has dedicated his career to enhancing the well-being of his patients, enabling them to lead long and healthy lives.

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Unlocking the power of stem cell therapy - Drug Target Review

Improving stem cell research – Harvard School of Engineering and Applied Sciences

Repetitive, manual tasks are an inevitability in managing daily operations in a research setting. But the more time researchers spend on basic maintenance, the less time they have to do cutting-edge research.

Third-year students at the Harvard John A. Paulson School of Engineering and Applied Sciences (SEAS) worked with the Harvard Stem Cell Institute iPS Core Facility last fall to design a pair of solutions to reduce the time spent on day-to-day operations. They created an automated task management and scheduling system called WorkFlow, and a semi-robotic cell imaging system called CytoScope. The students presented their designs as their final project for ES96: Engineering Problem Solving and Design Project, a core course for third-year SEAS students pursuing S.B. engineering degrees. This section was taught by David Mooney, Robert P. Pinkas Family Professor of Bioengineering.

This was the best design project Ive ever been part of, said project co-lead Ryan Link, a mechanical engineering concentrator. We started from scratch, didnt know what wed have at the end. It was this whole design process: start from literally a blank piece of paper, create each piece, go through and solve problems along the way, and build something in the end.

The iPS Core Facility derives and distributes induced pluripotent stem cells (IPSCs). Unlike embryonic stem cells, IPSCs can be taken from adults and start off as cells that have already differentiated into a specific use, such as kidney or heart cells. The cells can then be regressed into stem cells, which can differentiate into a new function.

I could walk up to an adult, take some cells, turn them into stem cells and use them to recreate their kidney or liver, Link said.

Because IPSCs can be taken from adults, they have the potential to enable stem cell research without the ethical and political issues associated with embryonic stem cells.

As bioengineers, a lot of the stuff that we research has ethical concerns in mind, said Aaron Zheng, a bioengineering concentrator and project co-lead. So, it was very interesting for us to work on this project to further a field that has a lot of scientific implications without the preexisting ethical implications.

The iPS Core Facility challenged the 13 ES96 students to identify ways to improve productivity and operations in the lab. That led to a full month of background research and interviews to identify the most-pressing needs.

We spent a month deriving a one-sentence problem statement, which is what we framed the rest of the semester around, Zheng said. It was about what our client needed the most, what their biggest challenges were, and what solution would best address that problem.

The students then brainstormed potential solutions, slowly whittling down the list based on factors that included cost of materials, level of impact, and feasibility of delivery by the end of the semester.

The students worked hard, demonstrated significant creativity and ingenuity, and I think really learned how to work as a team on a complex, multicomponent project, Mooney said. The Teaching Fellows, Shawn Kang and Kyle Ruark, and Active Learning Labs staff Melissa Hancock and Avery Normandin provided access to critical resources and important training, and the students worked closely with the iPS Core Facility Director Dr. Laurence Daheron to both identify the key issues and develop solutions.

As a problem statement, the students decided the facilitys biggest need was to improve the inefficiencies in its monitoring technology and process of culturing sample cells. The CytoScope addresses those inefficiencies by automating the imaging process for stem cell plates stored overnight in incubators.

When researchers would check on the IPSCs every morning, theyd have to take them out, put them under a microscope, examine them by hand and try to determine what was going on, Link said. Theyd have to do that for every cell plate or cell well, which means a lot of manual labor for a pretty simple task. Our idea was to create a system inside the incubator that could image the cells autonomously overnight, and the researchers could just look at the images in the morning without having to do all these extra steps.

WorkFlow is a software system that combines calendar, messaging, spreadsheet and task-management programs, making it easier for researchers to track what their colleagues are doing and when. Both final products stressed the importance of feasibility, of designing engineering solutions that can be delivered to a client by a specific date.

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Improving stem cell research - Harvard School of Engineering and Applied Sciences

Amneal Announces Complete Response Resubmission for IPX203 New Drug Application

BRIDGEWATER, N.J., Feb. 08, 2024 (GLOBE NEWSWIRE) -- Amneal Pharmaceuticals, Inc. (NASDAQ: AMRX) (“Amneal” or the “Company”) today announced that it has provided a Complete Response resubmission to the U.S. Food and Drug Administration (FDA) for IPX203, a novel, oral formulation of carbidopa/levodopa (CD/LD) extended-release capsules for the treatment of Parkinson’s disease (PD).

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Amneal Announces Complete Response Resubmission for IPX203 New Drug Application

Travere Therapeutics to Report Fourth Quarter and Full Year 2024 Financial Results

SAN DIEGO, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced it will report fourth quarter and full year 2023 financial results on Thursday, February 15, 2024, after the close of the U.S. financial markets. The Company will host a conference call and webcast to discuss the financial results and provide a general business update at 4:30 p.m. ET.

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Travere Therapeutics to Report Fourth Quarter and Full Year 2024 Financial Results

Gritstone bio Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

EMERYVILLE, Calif., Feb. 08, 2024 (GLOBE NEWSWIRE) -- Gritstone bio, Inc. (Nasdaq: GRTS), a clinical-stage biotechnology company that aims to develop the world’s most potent vaccines, today announced that the Compensation Committee of the company’s Board of Directors granted six employees nonqualified stock options to purchase an aggregate of 147,000 shares of its common stock with an exercise price of $2.26, which is equal to the closing price of Gritstone’s common stock on February 6, 2024, the date of the grant. These stock options are part of an inducement material to the new employees becoming an employee of Gritstone, in accordance with Nasdaq Listing Rule 5635(c)(4).

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Gritstone bio Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Immix Biopharma Announces Closing of $15 Million Public Offering of Common Stock

LOS ANGELES, CA, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us” or “IMMX”), a clinical-stage biopharmaceutical company pioneering personalized therapies for oncology and immunology, announced today the closing of its previously announced underwritten public offering of 5,535,055 shares of its common stock. Each share of common stock was sold at a price to the public of $2.71 per share. The gross proceeds to ImmixBio from this offering are expected to be approximately $15 million, before deducting underwriting discounts and commissions and other estimated offering expenses payable by ImmixBio. The Company intends to use the net proceeds from this offering for NXC-201 clinical trials, working capital and general corporate purposes.

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Immix Biopharma Announces Closing of $15 Million Public Offering of Common Stock

Telomir Pharmaceuticals Announces Pricing of Initial Public Offering

BALTIMORE, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Telomir Pharmaceuticals, Inc., a pre-clinical-stage pharmaceutical company focused on the development and commercialization of TELOMIR-1, is developing Telomir-1 as the first novel small molecule, to lengthen the DNA’s protective telomere caps in order to affect age reversal, today announced the pricing of its initial public offering of 1,000,000 shares of its common stock at a public offering price of $7.00 per share. All of the shares of common stock are being offered by Telomir. In addition, Telomir has granted the underwriters a 45-day option to purchase up to an additional 150,000 shares of common stock at the initial public offering price, less underwriting discounts and commissions. The shares are expected to begin trading on the Nasdaq Capital Market on February 9, 2024 under the ticker symbol "TELO." The gross proceeds to Telomir from the initial public offering, before deducting underwriting discounts and commissions and estimated offering expenses, are expected to be $7 million. The initial public offering is expected to close on February 13, 2024, subject to satisfaction of customary closing conditions.

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Telomir Pharmaceuticals Announces Pricing of Initial Public Offering

Molecular Templates to Present at Oppenheimer 34th Annual Healthcare Life Sciences Conference

AUSTIN, Texas, Feb. 08, 2024 (GLOBE NEWSWIRE) -- Molecular Templates, Inc.?(Nasdaq: MTEM, “Molecular Templates,” or “MTEM”), a clinical-stage biopharmaceutical company developing novel therapeutics with potent and differentiated mechanisms of action for cancer, announced participation in the upcoming Oppenheimer 34th Annual Healthcare Life Sciences Conference.

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Molecular Templates to Present at Oppenheimer 34th Annual Healthcare Life Sciences Conference

Metagenomi Announces Pricing of Initial Public Offering

EMERYVILLE, Calif., Feb. 08, 2024 (GLOBE NEWSWIRE) -- Metagenomi, Inc. (Nasdaq: MGX) (“Metagenomi”), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived gene editing toolbox, today announced the pricing of its initial public offering of 6,250,000 shares of its common stock at a price to the public of $15.00 per share. The gross proceeds to Metagenomi from the offering, before deducting the underwriting discounts and commissions and offering expenses, are expected to be $93,750,000. All of the shares are being offered by Metagenomi. In addition, Metagenomi has granted the underwriters a 30-day option to buy an additional 937,500 shares of its common stock at the initial public offering price, less underwriting discounts and commissions.

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Metagenomi Announces Pricing of Initial Public Offering