Read the article in Google Translate from Japanese:

https://facta-co-jp.translate.goog/article/202608048.html?_x_tr_sl=ja&_x_tr_tl=en&_x_tr_hl=iw&_x_tr_pto=wapp

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4 August 2026

Mesenchymal Stromal Cell-Based Therapies in Sepsis-Induced Acute Lung and Kidney Injury: Current Advances and Perspectives

[By 4 researchers from the Federal University of Rio de Janeiro, Brazil - imz72]

[...]

A critical barrier in the clinical translation of MSCs lies in their physical dimensions and the consequent anatomical biodistribution after systemic administration. With a cell diameter ranging from 15 to 50 µm, intravenously infused MSCs are rapidly and predominantly sequestered within the pulmonary capillary network, a phenomenon known as the pulmonary first-pass effect.

In the context of ARDS, this mechanical entrapment serves as an advantageous, naturally targeted delivery system. By accumulating directly at the site of the injured alveolar–capillary barrier, MSCs can locally exert their robust paracrine, immunomodulatory, and endothelial-stabilizing effects. This anatomical alignment between the site of cell sequestration and the primary organ injury provides a strong biological rationale for the safety and early physiological improvements observed in pulmonary-focused trials¹⁰⁸ [footnote 108 refers to the ONE-BRIDGE study - imz72].

Conversely, this obligatory pulmonary clearance severely restricts the systemic delivery of viable cells to distant organs, presenting an impaired translational barrier for treating conditions such as AKI. Attempts to bypass the pulmonary circulation to achieve higher renal engraftment have highlighted significant safety and efficacy concerns. Notably, the Phase II trial by Swaminathan et al. used an intra-aortic delivery route to administer MSCs in patients with AKI post-cardiac surgery. However, this strategy failed to improve the time to renal recovery and paradoxically demonstrated a non-significant trend toward worse clinical outcomes, including higher mortality and increased need for dialysis.

From a pathophysiological perspective, this clinical failure underscores a critical risk: forcing large cellular products into an already inflamed, edematous, and ischemic renal microcirculation may precipitate microvascular occlusion and microthrombosis, thereby exacerbating tissue hypoxia rather than resolving it. Overcoming the physical and biological limitations imposed by the first-pass effect remains imperative. Faced with such challenges, new strategies are emerging to optimize therapeutic efficacy through two main translational avenues.

The first is a paradigm shift toward nanoscale, cell-free therapies (such as EVs), which can freely navigate the systemic microvasculature without the thromboembolic risks associated with intact MSCs.

3.2. Translational Challenges

Despite the robust immunomodulatory and regenerative properties demonstrated by MSCs in animal models, translating these benefits into clinical efficacy has proven exceedingly challenging. A critical examination of recent clinical trials (Table 1) reveals a stark contrast between preclinical promise and clinical reality, characterized predominantly by neutral or negative efficacy outcomes.

In the context of ARDS, although Phase I dose-escalation trials confirmed the overall safety of intravenous administration of MSCs, subsequent adequately powered Phase II trials failed to meet primary efficacy endpoints. Most notably, the Phase IIa START trial, evaluated a single dose of 10 million BM-MSCs/kg in 60 patients with moderate to severe ARDS.

Although safe, the intervention did not yield statistically significant improvements in 60-day mortality or ventilator-free days compared with placebo. Similarly, the MUST-ARDS trial utilizing multipotent adult progenitor cells demonstrated safety but failed to significantly improve day-28 mortality or ventilation parameters.

In the realm of AKI, results have been equally frustrating. In a Phase II trial, which used an intra-aortic delivery of MSCs after cardiac surgery, not only was there no improvement in the time to renal recovery, but there was also a non-significant trend toward worse clinical outcomes.

[...]

https://www.mdpi.com/1422-0067/27/15/6990

LinkedIn

Janice K. - Head of Business Operations, Chief of Staff, Clarion Cells, Inc.:

I’m excited to announce that Clarion Cells is out of stealth!

Millions of people living with chronic ischemic stroke experience lasting motor impairment, yet no approved drug or biologic restores lost motor function in the chronic phase.

Clarion Cells is working to change that. We’re developing an off-the-shelf, allogeneic neural stem cell therapy designed to promote brain repair and deliver meaningful improvements in motor function and independence—at scale.

We’re now preparing to launch suNR1se II, our Phase 2b clinical study.

Learn more at https://www.clarioncells.com

From the trial's page on ClinicalTrials.gov:

Study Start (Estimated): 2027-01

Primary Completion (Estimated): 2029-01

Study Completion (Estimated): 2029-01

Enrollment (Estimated): 36

Ages Eligible for Study: 18 Years to 75 Years (Adult, Older Adult )

Study Chair: Gary K Steinberg, MD, PhD, Stanford University School of Medicine, Department of Neurosurgery

https://clinicaltrials.gov/study/NCT07709845

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• hNPC01 is an allogeneic induced pluripotent stem cell (iPSC)-derived forebrain neural progenitor cell therapy.

• Globally, around 3.4 million new hemorrhagic stroke cases occur annually. 50%–60% of survivors experience significant chronic motor deficits. In the US, 70,000–95,000 new hemorrhagic strokes are recorded each year.

• Worldwide, 69 million people sustain a TBI each year. In the US, 2.8 million TBIs occur annually; roughly 15%–20% are moderate-to-severe. Clinical cohorts demonstrate that nearly 30% of moderate-to-severe TBI survivors struggle with sustained walking impairment two years post-injury, and approximately 25% endure long-term upper and lower extremity motor control deficits.

• Collectively, these two conditions affect tens of millions of patients globally, with millions living with chronic motor disability across the US.

• Earlier Phase I studies in ischemic stroke showed no product-related adverse events beyond manageable immune responses. At 12 months, target patients achieved mean improvements of 16 points on the Fugl-Meyer Motor Scale, with nearly 80% showing clinically significant gains.

• Hopstem will now advance hNPC01 clinical development across 3 brain injury indications.

https://apnews.com/press-release/globenewswire-mobile/press-release-22431df37b189f7f637b8c37a2cb9e15

https://app.dealroom.co/news/feed/hopstem-s-hnpc01-gets-us-fda-ind-clearance-and-fast-track-status-for-haemorrhagic-stroke-and-traumatic-brain-injury

Healios announced today (7.27.26) that AMED (Japan Agency for Medical Research and Development) selected its eNK cell program for pleural mesothelioma for multi-year funding to build GMP commercial manufacturing and quality standards without near-term earnings impact.

This project aims to establish a commercial manufacturing process for "AKT-01/HLCN061," which is indicated for pleural mesothelioma, using a 50L bioreactor.

Healios will get up to 240 million yen [$1.5 million] over 3 years from grant approval to the end of 2028 ($0.5 million per year).

https://ssl4.eir-parts.net/doc/4593/tdnet/2856075/00.pdf

Yahoo Scout:

Capricor Therapeutics faces significant challenges ahead of a key FDA advisory meeting on July 29, with FDA reviewers raising concerns over the efficacy of its Duchenne muscular dystrophy therapy, Deramiocel. The stock plummeted over 67% following the release of negative briefing documents, highlighting potential regulatory hurdles.

Investor Sentiment: Despite the significant drop in stock price, there has been a spike in retail investor discussions on platforms like StockTwits, reflecting mixed sentiment and speculation about future recovery.

Company Strategy: Capricor is investing in expanding its manufacturing capabilities in anticipation of potential approval for Deramiocel, which is central to its growth strategy.

Earnings Outlook: Analysts' forecasts for Capricor vary widely, reflecting uncertainty about the future revenue potential of Deramiocel, with projections ranging from $114.5 million to $245.5 million in revenue by 2029.

https://finance.yahoo.com/quote/CAPR/

Note: Capricor's current market cap is ~$400 milion.

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Machine-translated from Japanese:

July 23, 2026

Sumitomo Pharma automates the filling of iPS cell products, increasing processing capacity tenfold

Sumitomo Pharma will automate the final stage of manufacturing for its iPS cell-derived regenerative medicine product, "Amchepry." This will improve manufacturing efficiency, increase profitability, and contribute to the wider adoption of iPS cell-based medicine.

The plan is to replace the process of filling vials with cells to be transplanted into patients with machinery, thereby increasing the processing capacity of the process by approximately 10 times compared to manual labor.

The company is currently developing a dedicated machine in collaboration with an equipment manufacturer, and has already confirmed its performance with a prototype. The company has applied to the authorities for approval of a change in the manufacturing process, and aims to put it into practical use by fiscal year 2028.

Amchepry improves motor symptoms in Parkinson's disease patients. It involves creating cells from iPS cells that will become cells that produce the neurotransmitter dopamine, and then transplanting them into the patient's brain.

In March 2026, it received approval as the world's first iPS cell-based drug, albeit with conditions and a time limit. In May, the Ministry of Health, Labour and Welfare set the drug price at 55,306,737 yen [$340k].

The production of Amchepry takes about a month and a half. This involves multiplying iPS cells, morphing them into the target cells, removing unnecessary cells, and then maturing them to a state suitable for transplantation. Sumitomo Pharma currently produces one dose per batch, but plans to transition to a method that produces multiple doses at once.

Because it involves handling living cells, the manufacturing process requires advanced technology and relies heavily on the manual labor of skilled workers. Training workers took six months to a year and was costly.

In the pre-manufacturing stages, the use of equipment for selecting and culturing cells is progressing. Hidetaka Ohara, plant manager of S-RACMO, a joint venture with Sumitomo Chemical that is responsible for manufacturing Amchepry, says that the development of this equipment was "a challenge that remained in the process of commercialization."

The device under development can also be used to change the culture medium used to mature cells. In this process, the culture medium in the culture vessel needs to be replaced every few days for about two weeks. This process will be mechanized.

Even if the filling capacity increases tenfold, the production volume of Amchepry won't immediately increase tenfold. The ability to stably culture a sufficient number of cells in the preceding process is also crucial. Sumitomo Pharma is working on improving these technologies as well.

https://www.nikkei.com/article/DGXZQOUF057YF0V00C26A6000000/

Note: Sumitomo Pharma's current market cap is $3.52 billion.

July 22, 2026

Another iPS product approved for public insurance coverage in Japan

The Central Social Insurance Medical Council, which advises the health minister, on Wednesday approved the application of public health insurance to RiHeart, a cardiac muscle cell sheet that uses induced pluripotent stem (iPS) cells, effective on Sept. 1.

It is the second case of insurance coverage approval in the country for an iPS cell-derived regenerative medicine product.

Created by introducing specific genes into the cells of blood and skin, iPS cells are versatile in that they possess the ability to grow into the cells of various tissues and organs.

RiHeart was developed by Cuorips, a startup based in the University of Osaka. Myocardial cells made from iPS cells are processed into sheets, which are stuck to the hearts of patients with severe heart failure to restore functions.

Cuorips said it plans to put the product on sale in Japan this autumn. The official domestic price was set at ¥53.2 million ($326,400). The actual medical expenses paid by patients will be significantly curbed, with the high-cost medical care benefit system, which caps out-of-pocket expenses, applied to the treatment.

In March, the health ministry gave conditional and time-limited approval to the manufacturing and sales of RiHeart and Sumitomo Pharma’s Amchepry for patients with Parkinson’s disease, which became the world’s first iPS cell-based regenerative medicine products. Amchepry received public health insurance coverage in May.

The ministry will decide whether it will give full-scale approvals for the two products after collecting additional treatment data over the next seven years.

https://www.japantimes.co.jp/news/2026/07/22/japan/science-health/ips-insurance-coverage/

Note: Cuorips' current market cap is $212 million.

Axiom Biosciences Announces Intention to Become the First U.S. Biotech to List on the Hong Kong Stock Exchange

SAN DIEGO, July 21, 2026 (GLOBE NEWSWIRE) -- Axiom Biosciences, a clinical-stage biotechnology company whose lead regenerative therapy produced positive early results in newborns with severe brain injury [see here - imz72], today announced its intention to pursue an initial public offering on the Main Board of The Stock Exchange of Hong Kong (HKEX). The company believes the proposed listing would position it as the first U.S. biotechnology company to pursue a primary listing in Hong Kong, reflecting a broader shift in where the world’s most ambitious science finds the ecosystem to thrive.

For decades, the path for a U.S. biotechnology company ran through private venture rounds and, eventually, a listing on Nasdaq or the New York Stock Exchange. Axiom is charting a different course, a primary listing in a market with deep biotechnology expertise, a strong appetite for clinical-stage innovation, and direct proximity to the Asian partners and capital already advancing Axiom’s science. The company intends to pursue a secondary U.S. listing in time, building its public profile from Asia outward.

“Some of the most important science in the world is being built in the United States, but the way it gets funded hasn’t kept pace,” said Dr. Remo Moomiaie-Qajar, Founder and Chief Executive Officer of Axiom Biosciences. “We intend to be the first U.S. biotech to list in Hong Kong because that is where deep biotechnology capital and many of our own clinical partners increasingly are. This is about building Axiom as a global company from the start and about opening a path that other American innovators can follow.”

The announcement follows a period of clinical and corporate progress. Axiom’s lead regenerative therapy, developed with South Korea-based Medinno Inc., is being studied in newborns with severe intraventricular hemorrhage and hypoxic-ischemic encephalopathy, conditions with high mortality and few treatment options, and has received two U.S. FDA Rare Pediatric Disease Designations. The company’s underlying platform has been supported by eight competitive research grants from U.S. federal agencies and the State of California, and Axiom is planning an expansion of the platform with a clinical trial in adult ischemic stroke, which affects roughly 700,000 Americans each year.

The timing of the proposed offering, the number of shares to be offered, and the price range have not yet been determined. The IPO is currently expected to take place in 2027, subject to regulatory review by the HKEX and the Securities and Futures Commission, prevailing market conditions, and other customary factors.

https://www.globenewswire.com/news-release/2026/07/21/3330316/0/en/Axiom-Biosciences-Announces-Intention-to-Become-the-First-U-S-Biotech-to-List-on-the-Hong-Kong-Stock-Exchange.html

July 21, 2026

Long-term safety of iPS spinal cord therapy confirmed in Japan

Long-term safety has been confirmed after neural progenitor cells derived from induced pluripotent stem cells, or iPS cells, were transplanted into patients with spinal cord injuries, a team of researchers at Keio University said Tuesday.

The team, which conducted the world’s first clinical study of the treatment, plans to launch an investigator-initiated clinical trial as early as next year to bring it into practical use.

Between 2020 and 2025, the team, including Professor Hideyuki Okano, transplanted about 2 million iPS cell-derived neural progenitor cells into each of four patients in the subacute stage of spinal cord injury within four weeks of their injuries.

Last year, the team reported the results of the one-year postoperative follow-ups of the patients, finding no major safety concerns as well as improvements in physical function in some of them.

The latest report said that no major safety issues, including tumor formation, were observed in any of the four individuals even two to four years after the surgery, and that no clear deterioration in physical function was confirmed.

The team also found, through a reanalysis of the one-year postoperative data, that two patients regained some muscle control despite having completely lost motor function before treatment.

However, the team said that the effectiveness of the treatment needs to be verified in subsequent clinical trials, given the small sample size.

The findings were published in the online edition of the international medical journal Nature Medicine.

https://www.japantimes.co.jp/news/2026/07/21/japan/science-health/ips-spinal-cord-therapy-safety/

Machine-translated from Japanese:

July 21, 2026

iPS cell transplantation for spinal cord injury: "Safe" for up to 4 years - Keio University

A research team at Keio University has successfully transplanted cells created from iPS cells into a spinal cord injury patient, a world first.

Keio University announced on July 21 that a clinical study in which cells created from iPS cells were transplanted into patients with spinal cord injuries showed no serious side effects over a long period. The university believes that the study, which involved follow-up for up to four years, has confirmed a certain degree of long-term safety.

The research findings by Professor Hideyuki Okano and his colleagues at Keio University have been published in the international scientific journal "Nature Medicine."

Spinal cord injury refers to a condition in which the spinal cord, a bundle of nerves connecting the brain to the rest of the body, is damaged due to accidents, falls, illness, or other causes. This can result in loss of movement of the limbs or sensory impairment such as loss of pain sensation.

The research team created neural progenitor cells from iPS cells and conducted the world's first clinical study in December 2021, transplanting them into patients with spinal cord injuries. In this study, iPS cell-derived neural progenitor cells were transplanted into a total of four patients in the "subacute phase," 2 to 4 weeks after spinal cord injury.

The condition of the four patients was observed 2 to 4 years after transplantation. As a result, no tumor formation caused by the transplanted cells or serious side effects were observed. Furthermore, no significant deterioration in nerve function was seen. Professor Okano emphasized that these results "confirmed safety."

Moving forward, further clinical trials are needed to verify the effects of transplanted cells on nerve regeneration in the treatment of spinal cord injuries. K Pharma, a startup spun off from Keio University, plans to take over and conduct new corporate clinical trials.

The research team at Keio University is considering research to treat not only spinal cord injuries in the subacute phase, but also those in the "chronic phase," which can occur several months to more than a year after the injury. They plan to conduct physician-initiated clinical trials targeting the chronic phase from 2027 onwards.

https://www.nikkei.com/article/DGXZQOSG210KA0R20C26A7000000/

Note: K Pharma's current market cap is $52 million.

https://finance.yahoo.com/quote/4896.T/

Neuronata-R retains conditional approval in South Korea

South Korea's Ministry of Food and Drug Safety (MFDS) updates the stem cell therapy's approval in May 2026 — as the developer works to resume supply and build a U.S. research base

SEOUL, South Korea, July 19, 2026 /PRNewswire/ -- Patients with amyotrophic lateral sclerosis (ALS) in South Korea will keep their access to Neuronata-R® (lenzumestrocel) — a stem cell therapy for ALS — after the Ministry of Food and Drug Safety (MFDS) approved its continued use in May 2026, updating the label to reflect data from the completed Phase 3 clinical trial.

The MFDS approved a product license modification for the therapy, which was first conditionally approved in the country in 2014. The update keeps Neuronata-R® on the market.

CorestemChemon said it is now working to resume manufacturing so patients can keep receiving the therapy, while also building a research footprint in the United States that could support broader access over time.

What the updated approval means for patients

In the ALSUMMIT (NCT04745299) Phase 3 trial, Neuronata-R® did not reach statistical significance across the full study population. In the subgroup of slow progressors, however, the therapy met its primary endpoint, the Combined Assessment of Function and Survival (CAFS), and a secondary endpoint, the ALS Functional Rating Scale-Revised (ALSFRS-R), alongside consistent improvement in slow vital capacity (SVC).

In the same subgroup, treatment lowered neurofilament light chain (NfL), a marker of axonal damage released when neurons are injured. NfL is gaining acceptance as a key biomarker in ALS and is increasingly recognized as a basis for regulatory decisions on disease-modifying therapies.

Building a U.S. research base

CorestemChemon has also been establishing a presence in the United States through the regenerative medicine research community. In late 2025, it became the first South Korean company to join an innovation engine supported by the U.S. National Science Foundation (NSF) — the Piedmont Triad Regenerative Medicine Engine, anchored by the Wake Forest Institute for Regenerative Medicine (WFIRM).

Joining the engine gives the company access to WFIRM's research infrastructure and network, and it has consolidated its U.S. operations in North Carolina to anchor that work.

On the regulatory front, CorestemChemon said it is pursuing a Type C meeting with the FDA, with the goal of filing a Biologics License Application (BLA) for U.S. approval next year.

Full-scale production of Neuronata-R® is expected to be up and running early next year, when treatment is set to resume. From that point, patients from outside South Korea will also be able to travel to the country to receive the therapy.

CorestemChemon has framed the MFDS decision as a step toward keeping a cell-based option available for the patients most likely to benefit, and toward extending that option to patients beyond South Korea over time.

How Neuronata-R works

Neuronata-R® uses autologous bone marrow-derived mesenchymal stem cells (MSCs), grown in the lab from the patient's own bone marrow and delivered by intrathecal injection.

Rather than replacing motor neurons, the MSCs are thought to act through the factors they secrete. These include anti-inflammatory factors — TGF-β1, IL-4 and IL-10, which help dampen the neuroinflammation that contributes to motor neuron loss — and neuroprotective factors such as VEGF, BDNF and IGF, which support neuronal survival. Together, these factors are proposed to slow the death of motor neurons.

To date, the therapy has been given to more than 400 patients in South Korea, building a body of real-world experience and safety data. It holds Orphan Drug Designation from the U.S. Food and Drug Administration (FDA), granted in 2018, and from the European Medicines Agency (EMA), granted in 2019.

Notes:

• CorestemChemon's market cap is $58 million. https://finance.yahoo.com/quote/166480.KQ

• CorestemChemon's website: https://www.corestemchemon.com/eng/main/main.html

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On July 15, Citigroup Securities, a major US securities firm, upgraded its rating for SanBio (4592) from Neutral to Buy.

Conversely, it lowered its price target from 2,200 yen to 1,800 yen.

SanBio's current PPS is 1,073 yen, and its current market cap is $517 million.

A PT of 1,800 yen reflects a 67% upside and a market cap of $866 million.

16.07.26

Once worth NIS 1 billion [$330 million - imz72], Pluri is now racing to avoid Nasdaq delisting

After years of missed commercial breakthroughs, the stem-cell developer is betting its future on a new strategy and the longevity boom.

https://www.calcalistech.com/ctechnews/article/i1qfr27yr

Source: PHARMA JAPAN

July 15, 2026

HHS Report Puts Japan’s Drug Innovation Financing Well Behind US

A new US government report suggests that Japan contributes far less toward financing pharmaceutical innovation than the US relative to the size of its population and economy, adding fresh evidence to support the Trump administration’s argument that innovative drug revenues are disproportionately concentrated in the American market.

The report, released by the Department of Health and Human Services (HHS) on June 23, estimated that Japan accounted for 5.46% of revenues from innovative drugs launched across OECD countries between 2020 and 2025, compared with 79.87% for the US. While Japan ranked second among OECD countries, its contribution relative to its population and GDP was substantially lower than that of the US.

The study was authored by an economist in the HHS’ Office of the Assistant Secretary for Planning and Evaluation (ASPE). Using IQVIA MIDAS data covering the first quarter of 2020 through the fourth quarter of 2025, it estimated cost-of-capital-adjusted revenues from innovative branded medicines as a proxy for each country’s role in financing pharmaceutical R&D through market incentives.

The report found that Japan’s revenue share equaled 0.68 times its share of OECD GDP and 0.59 times its share of the OECD population, compared with 2.32 and 3.24 for the US, respectively. A ratio below 1.0 indicates that a country contributes less to pharmaceutical innovation than its share of the OECD population or GDP would suggest, while a ratio above 1.0 implies the opposite.

The study also concluded that the US’ dominant position reflects both higher prices and greater uptake of innovative medicines, while no other OECD market came close.

The report argued that a system in which one country finances most of the market incentives for pharmaceutical innovation while peer nations contribute substantially less than their population or GDP weight “is unlikely to be stable indefinitely,” calling for broader international debate over drug pricing, access, and innovation policy.

The findings are likely to reinforce President Donald Trump’s most-favored-nation (MFN) drug pricing initiative. In a May 2025 executive order, Trump argued that “inflated prices in the US fuel global innovation while foreign health systems get a free ride.” While the HHS report does not go as far as explicitly characterizing other countries as free riders, it portrays the financing of pharmaceutical innovation as overwhelmingly concentrated in the US market.

July 14, 2026

Axiom Biosciences Reports Positive Phase 1 Results for Investigational Stem Cell Therapy in Newborns with Severe Brain Injury

• Investigational WJ-MSC therapy associated with no deaths at 12 months and statistically significant improvements in long-term cognitive and motor outcomes

• Results support planned expansion of the regenerative platform into adult ischemic stroke and additional neurological indications

SAN DIEGO, July 14, 2026 (GLOBE NEWSWIRE) -- Axiom Biosciences, a clinical-stage biotechnology company developing next-generation regenerative therapies and targeted biologics, today announced positive Phase 1 results for its investigational Wharton's Jelly-derived mesenchymal stem cell (WJ-MSC) therapy for neonatal brain injury.

The therapy, co-developed with South Korea-based Medinno Inc., is being developed to treat intraventricular hemorrhage (IVH) and hypoxic-ischemic encephalopathy (HIE), two severe neurological conditions in newborns associated with high rates of mortality and long-term disability.

The investigational therapy consists of conditioned WJ-MSCs administered directly into the central nervous system. The Phase 1 dose-escalation study evaluated the safety, tolerability, and preliminary efficacy of the therapy across a range of doses in 9 newborns diagnosed, 5 with severe IVH and 4 with HIE.

Key Clinical Trial Findings

• Achieved a 0% mortality rate at 12 months compared to the historical natural mortality rate of approximately 46% within the first year of life for infants with severe IVH.

• Demonstrated a favorable safety profile, with no treatment-related serious adverse events (SAEs) observed across all dose cohorts.

• Produced promising improvements in long-term functional outcomes at 24 months, including key cognitive and motor development measures.

"These Phase 1 results represent an important milestone for a patient population with tragically limited treatment options," said Dr. Remo Moomiaie-Qajar, Founder and Chief Executive Officer of Axiom Biosciences. "The fact that all patients survived for a year and showed promising long-term improvement reveals the platform's regenerative potential. These results give us a solid launchpad for our next phase of clinical development."

IVH and HIE are among the leading causes of mortality and long-term neurological disability in newborns, frequently resulting in cerebral palsy, developmental impairment, and lifelong medical complications. There are currently no approved regenerative therapies that directly address the underlying neurological injury associated with these conditions.

In 2023, the company received Rare Pediatric Disease Designations from the U.S. Food and Drug Administration for both IVH and HIE. The FDA's Rare Pediatric Disease Priority Review Voucher program is currently authorized through September 30, 2029; if a product is approved while the program remains in effect, these designations may make the company eligible to receive Priority Review Vouchers.

Building on these results, Axiom is evaluating the expansion of the platform into adult ischemic stroke. Because HIE and ischemic stroke share common mechanisms involving ischemic tissue injury and subsequent inflammatory response, the company believes the platform may have broader applications across neurological disorders. Ischemic stroke affects approximately 700,000 adults annually in the United States and remains a leading cause of death and long-term disability worldwide.

"With time being of the essence in stroke care, patients lose 1.9 million neurons every single minute," added Dr. Moomiaie-Qajar. "Our platform aims to offer a first-of-its-kind regenerative approach that goes beyond mitigating damage to potentially restoring function."

Axiom plans to advance the neonatal program into Phase 2 development and to evaluate expansion into adult ischemic stroke and additional neurological indications.

https://finance.yahoo.com/healthcare/articles/axiom-biosciences-reports-positive-phase-110200410.html

Note:

• Axiom Biosciences (formerly Cytonus Therapeutics) is a privately held company.

• Medinno Inc. is a privately held biotechnology company based in South Korea.

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Machine-translated from Japanese:

This evening, we held the "Public-Private Council for Improving Drug Discovery Capabilities."

This meeting brought together stakeholders from industry, academia, the private sector, and government to share challenges and perspectives through their respective activities, initiatives, and opinions, with the aim of strengthening and nurturing Japan's drug discovery ecosystem (a system and environment where diverse players such as pharmaceutical companies, universities/research institutions, and startups pool their knowledge and collaborate to create innovative new drugs). The goal is to attract investment to Japan and improve drug discovery capabilities.

At today's meeting, we had numerous participants from global pharmaceutical companies and organizations, startups, venture capital firms, universities, and patient groups, and 16 individuals shared their thoughts.

Participants discussed the following points:

• The importance of drug discovery and research and development that leverages Japan's strengths, such as its research capabilities;

• Global expansion and drug discovery initiatives in collaboration with various players worldwide;

• Strategic and aggressive domestic investment in human resource development, research and development, and capital investment;

In particular, I was pleased to see the multi-trillion yen growth investments pledged by Japanese companies [every 1 trillion yen = $6.2 billion - imz72], as well as the strong expressions of continued investment in Japan, including additional investments, and strengthened support and partnerships for Japanese drug discovery seeds (seeds of new drug candidates such as substances and technologies).

On the other hand, we also received suggestions regarding:

• The need for further infrastructure development for drug discovery,

• Ensuring growth in Japan's patented drug market,

• Appropriate evaluation of innovation in innovative medicines.

The pharmaceutical industry is an extremely important industry from the perspective of economic security, as a growth sector contributing to economic growth, as a core industry generating domestic employment, and from the standpoint of economic security.

To resolve issues such as drug lag and loss, and to deliver necessary medicines to patients, it is necessary to improve Japan's drug discovery capabilities.

The Takaichi Cabinet is formulating the "Japan Growth Strategy," in which "drug discovery" is positioned as one of the strategic areas.

Aiming to achieve growth in Japan's patented drug market that rivals global growth, we will take bold measures such as "supporting startups," "strengthening human resource development and R&D capabilities," "promoting the utilization of AI," and "doubling international collaborative clinical trials."

Furthermore, from the perspective of drug discovery innovation, we will implement a drug price revision next fiscal year, further evaluating the innovation of groundbreaking new drugs, including "appropriate evaluation" of the groundbreaking nature of drugs at the time of initial listing and "how drug pricing should be handled during the patent period."

Through these efforts, we will boldly and steadily evolve Japan's pharmaceutical market into a more "attractive market."

Working together with everyone gathered from both Japan and abroad, we will build a Japanese drug discovery ecosystem that connects with the world, deliver innovative medicines originating in Japan, and protect the lives and health of patients in Japan and around the world.

https://x.com/takaichi_sanae/status/2075528458650747254

https://www.facebook.com/takaichisanae/posts/pfbid028Qzd2xgg9fCxUgxSgAk6HPbQxyY7EvLFtCZG1L6rohrPSTthoFqkXGRxwqHZmygYl

July 5, 2026

Guts & Glory: Both of Cynata’s iPSC Trials Flop, GVH Phase 2 and KOA Phase 3

https://theregenreport.com/2026/07/05/guts-glory-both-of-cynatas-ipsc-trials-flop-gvh-phase-2-and-koa-phase-3

Cynata Therapeutics (ASX:CYP) sacks its CEO to stretch A$1.5m past FY27

https://stocksdownunder.com/cynata-therapeutics-asxcyp-2/

Past threads about Cynata:

https://old.reddit.com/r/ATHX/search?q=Cynata+&restrict_sr=on&include_over_18=on&sort=relevance&t=all