Ryoncil, the first FDA-approved mesenchymal stromal cell therapy
Regenerative medicine, a rapidly advancing field, can be generally defined as a therapeutic methodology for the repair, regeneration, or replacement of damaged or diseased human cells and tissues. Ryoncil is an allogeneic mesenchymal stromal cell (MSC) therapy indicated for the treatment of acute graft-versus-host disease (aGvHD) for children older than two months, including adolescents and teenagers. aGvHD is a common complication of allogeneic haematopoietic stem cell transplantation (alloHCT) and is a detrimental condition with a high mortality rate. Systemic steroid therapy is generally the first line of treatment for aGvHD. According to a study by Malard and colleagues, which was published in Nature in 2020, approximately 35-50% of patients develop systemic-refractory acute graft-versus-host disease (SR-aGvHD). Furthermore, an optimal second-line therapy has not yet been introduced, highlighting the significant need for researchers to develop a safe and effective novel therapy for patients with SR-aGvHD.
Cell therapies are a fundamental aspect of regenerative medicine and consist of the administration of living cellular material into a patient to replace or regenerate damaged tissue for functional restoration. There are a variety of cells used within cell therapy, including embryonic stem cells (ESCs), induced pluripotent stem cells, and MSCs. MSCs are stromal cells that exhibit self-renewal and multipotent properties with the capacity to differentiate along mesenchymal and non-mesenchymal lineages. These include osteoblasts, myocytes, adipocytes, and chondrocytes. MSCs can be isolated from various tissues, including bone marrow, cord cells, and molar cells, and pose fewer ethical concerns compared to those associated with the collection of ESCs. SR-Ryoncil was developed and patented by Osiris Therapeutics, which later sold the intellectual property to Mesoblast Limited to initiate Phase III trials. In a single-arm, multi-centre, Phase III trial in children with SR-aGvHD, 89% of which had a severe form of the disease, 70% of children achieved an overall response by Day 28 of treatment using Ryoncil. This measure predicts the survival rates of patients with aGvHD. According to leading data and analytics company GlobalData's Making a Difference with Regenerative Medicine, 2025 report, allogeneic cell therapies have the potential to reduce manufacturing burdens within the pharmaceutical industry. This is due to their advantageous scalability in comparison to autologous treatments as multiple patients can be treated from a single batch, consequently increasing patient accessibility. However, the usage of allogeneic cell therapies exhibits a variety of challenges. For example, larger batches of allogeneic cells create difficulties in preserving cell consistencies, requiring advanced bioprocessing techniques and intricate machinery. Furthermore, the regulatory landscape for allogeneic cell therapies presents complex procedures due to their relative novelty in comparison to autologous treatments. The multipotent characteristics of MSCs have the potential to expand into multiple clinical developments and fulfil the unmet medical demand of various diseases. As the number of patients with alloHCT increases, the development of novel and alternative means of regenerative therapy becomes increasingly important within the pharmaceutical landscape. This highlights the potential of Ryoncil's immunomodulatory effects for the treatment of SR-aGvHD while simultaneously advancing therapeutic applications for other inflammatory diseases, thus allowing researchers to utilise MSC-based therapy options to ultimately improve patient outcomes.
"Ryoncil, the first FDA-approved mesenchymal stromal cell therapy" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand.
The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Tom's Guide
a day ago
- Tom's Guide
Trouble sleeping? 'Moon breathing' can reduce anxiety at night, says expert
Even when you've created the perfect environment for sleep and are exhausted from a busy day, sleep can be elusive if you experience nighttime anxiety — which can include trouble relaxing, night sweats, panic attacks and increased waking during the night. But what if we told you that an ancient yogic breathing technique could be a tool in your arsenal to challenge it? Moon breathing is a term that has become more popular more recently thanks to social media platforms like TikTok. This breathing technique is focused on using your left nostril, and can lead to a range of benefits, including activating the parasympathetic nervous system (the part of our autonomic nervous system associated with relaxation), lowering the heart rate and preparing the body and mind for sleep. To explore more about what moon breathing is, exactly why it can help with nighttime anxiety and sleep, and how you can try it, we've spoken to licensed clinical psychologist specializing in sleep and trauma, and yoga teacher, Dr. Leah Kaylor Ph.D. PLLC.. "Moon breathing is a calming yogic breathwork technique rooted in ancient Indian practices of pranayama. In yogic philosophy, the left nostril is associated with lunar (moon) energy—cool, calm, and introspective," says Dr. Kaylor. It's a breathing exercise that involves inhaling slowly through your left nostril only, while keeping the right nostril closed, then exhaling through the right nostril while closing the left. A paper published in Nature exploring the changes in brain activity recorded by EEG during nasal breathing noted that, "Yogic practices suggest, and scientific evidence demonstrates, that right-nostril breathing is involved with relatively higher sympathetic activity (arousal states), while left-nostril breathing is associated with a relatively more parasympathetic activity (stress alleviating state)." "By breathing exclusively through the left nostril, this technique is thought to activate the parasympathetic nervous system, promoting relaxation and emotional ease," explains Dr. Kaylor. Get instant access to breaking news, the hottest reviews, great deals and helpful tips. The sympathetic nervous system (SNS) and the parasympathetic nervous system (PNS) are both part of our autonomic nervous system, with research noting that the former is connected to our alert, "fight or flight," response and the latter with "rest and digest" processes, when we are in a relaxed state. "Moon breathing works by channeling breath solely through the left nostril, which is believed to stimulate the brain's right hemisphere — the part associated with calmness, intuition, and emotional regulation," says Dr. Kaylor. The benefits of this, she says, can include reducing sympathetic (stress) arousal, activating the parasympathetic nervous system and lowering heart rate and blood pressure. Plus, moon breathing can help promote mental stillness and emotional grounding, and prepare our bodies for introspection, rest or sleep. One study looked at the effect of different yogic breathing techniques that utilise the left and right nostrils, and both, to see if they had differing effects on reaction time, heart rate and blood pressure. The researchers concluded from their results that moon breathing was among the left nostril techniques that "may induce a state of parasympathetic dominance as seen in (cardiovascular) parameters." They further noted that their findings are in line with "the traditional swara yoga concept that... the flow through left nostril (chandra nadi and ida swara) is relaxatory." Dr. Kaylor explains that moon breathing is particularly effective for pre-sleep anxiety because it: If you're experiencing regular nighttime anxiety, it's important to consult a medical professional for advice. If you want to incorporate moon breathing into your nighttime routine as a tool to combat it, Dr. Kaylor has some simple steps you can follow. The 4-7-8 method is a breathing technique that gained popularity after Dr. Andrew Weil began teaching it, and involves breathing in through your nose for a count of four, holding that breath for a count of seven, and then slowly breathing out through your mouth for a count of eight. Dr. Kaylor explains that the main difference between these two breathing techniques is that "moon breathing is through one nostril." "Moon breathing is best for highly anxious or overstimulated individuals," she says, explaining that it is "ideal for those experiencing hyperarousal or sympathetic overdrive (e.g., anxiety, racing thoughts, agitation)." Because moon breathing does not involve holding your breath, it can also be good breathing technique for those who are sensitive to breath retention, she says, or, those who find it distressing or physiologically uncomfortable. That could include people with a panic disorder or asthma. "4-7-8 is best suited for people with experience in controlled breathing or mindfulness," says Dr. Kaylor. "The extended breath-hold can feel challenging for beginners or those with respiratory sensitivity," she adds, noting that it can suit others too, including those who benefit from structure and counting, and those with sleep-onset insomnia. This is because "studies and anecdotal evidence suggest that 4-7-8 is particularly effective at quieting the mind and inducing a sleep-conducive state." Stress and anxiety are typically associated with higher levels of cortisol, which can increase heart rate and blood pressure. Cortisol is also the sleep hormone associated with making us feel awake and alert, which means that when it's increased, sleep will be more difficult. However, a review of studies looking at the effects of breathing exercises found "that breathing exercises have positive effect on decreasing the blood pressure and heart rate." Dr. Kaylor says, deep breathing can also help to "release physical tension in the body, particularly in the shoulders, chest, and jaw." And, in giving our brains something simple and rhythmic to focus on, deep breathing techniques can "interrupt cognitive rumination." Deep breathing has even been shown to increase melatonin production, the hormone our bodies produce in the evening and which helps us to feel sleepy. One study found that a group of 15 participants who practiced deep breathing alongside meditation and yogic postures for a period of three months saw an increase in plasma melatonin (which is the concentration of melatonin in our blood). In addition, Dr. Kaylor explains that deep breathing can "reduce cortisol levels, allowing melatonin to rise naturally." As well as helping you drift off in a calmer state, it can also help your overall sleep. Research showed that when a group of students practiced moon breathing for four weeks it 'led to improvement in sleep quality." Deep breathing is an incredibly affective way of activating our parasympathetic nervous system, creating a sense of calm and relaxation needed for sleep. Slower, deeper breaths into your diaphragm, "increases the pressure of oxygen in the air sacs, making it easier for oxygen molecules to move into the blood via the capillaries," neuroscience researcher Dr. Yewande Pearce explains in this useful Headspace video.
Forbes
2 days ago
- Forbes
Why The ‘Strawberry Moon' Will Be Lowest Until 2043 — And How To Photograph It
Tuesday's full strawberry moon occurs during two-year period known as the 'major lunar standstill" ... More or "lunistice," when a once-in 18.6-year event will create the lowest-hanging full moon since 2006 and until 2043. The full moon is seen here rising behind Stonehenge in England. (Photo by) The full strawberry moon will put on a dramatic show at moonrise on Tuesday, June 10 — low, luminous, and colored orange as it climbs into the southeastern sky. Get to an observing location that looks southeast — preferably low to the horizon — and at the specific time of moonrise where you are (during dusk), you'll see the red-orange orb rise before your eyes. Seen from the Northern Hemisphere, the full moon will rise far to the southeast, move across the sky close to the southern horizon, and set in the southwest close to dawn. It's happening because we're in the midst of a rare two-year period known as the 'major lunar standstill" or "lunistice," when a once-in 18.6-year event will create the lowest-hanging full moon since 2006 and until 2043. Earth's axis is tilted by 23.5 degrees with respect to the ecliptic, the path of the sun through the daytime sky, and, in effect, the plane of the solar system. That's what gives us seasons, and that's why planets are always found close to the ecliptic (hence the misused "planetary alignment" claims despite planets always being somewhat aligned with each other). The moon's orbit crosses the ecliptic twice each month, and when those crossings align with a new or full moon, eclipses can occur — hence the shared root in the words 'ecliptic' and 'eclipse.' While the sun's rise and set points vary throughout the year, changing by 47 degrees — and reaching the extreme points at the solstices — the moon's range is bigger, with that 5-degree tilt giving its rise and set points a 70-degree range near a major standstill, according to Griffith Observatory. Mount Coot-tha Lookout, Brisbane A major lunar standstill is a period when the northernmost and southernmost moonrise and moonset are furthest apart. Unlike a solstice (Latin for "sun stand still"), which lasts for one day, a major lunar standstill lasts for two years. These events are most noticeable during a full moon. Essentially, the swiveling and shifting orbit of the moon — a consequence of the sun's gravitational pull — is tilted at its maximum angle relative to the ecliptic. Every 18.6 years, the tilts combine to cause the moon to rise and set as much as 28.5° north or south of due east and west, respectively. Most people won't notice the major lunar standstill, but if you regularly watch the full moon rise from a particular place, go there — you'll get a shock when the moon rises at an extreme position much farther from where you might imagine it will rise. Imaging a full moon using a smartphone isn't easy, but it is possible to capture something special. First, switch off your flash and turn on HDR mode (if available) to better capture both the moon and the landscape as the light fades during dusk. Don't zoom in because digital zoom only blurs the details. Instead, frame the moon within a landscape for more impact, which is especially effective during this month's unusually far-southeast moonrise. If you use a manual photography app, stick to an ISO of 100 for a clean shot and experiment with slower (but not too slow) shutter speeds. A tripod will help, especially when using slow shutter speeds. However, if you don't have one, you can balance your phone on a wall or ledge. The key is to image it when it's low on the horizon and glowing orange. You've got a short window to capture that color, even with this low-hanging full moon, which will turn bright white as it lifts above the horizon. Wishing you clear skies and wide eyes.
Yahoo
2 days ago
- Yahoo
Resilience, a Private Japanese Spacecraft, Crash-Landed on the Moon
A Japanese spacecraft has probably crashed on the Moon, the second failed landing attempt for Tokyo-based private firm ispace. The HAKUTO-R Mission 2 (M2) lander — also called Resilience — began its landing sequence from a 100-kilometre-altitude orbit at 3.13am local time on 5 June. The craft was due to land near the centre of Mare Frigoris (Sea of Cold) at 4.17am. The ispace team said at a press conference that it lost contact with M2 when the craft was 192 metres above the Moon's surface and descending faster than expected. An attempt to reboot M2 was also unsuccessful. [Sign up for Today in Science, a free daily newsletter] M2 didn't receive measurements of the distance between itself and the lunar surface in time to slow down and reach its correct landing speed, the team said. 'It eventually slowed down, but not softly enough,' says Clive Neal, who studies the Moon at the University of Notre Dame in Indianapolis, US. He speculates that the failure was probably caused by a systems issue that wasn't identified and addressed during the M1 landing attempt. 'It's something that I believe will definitely be fixable, because getting that close means there's a few tweaks that are going to be needed for the next one,' he adds. If M2 had successfully landed on the lunar surface, the mission would have been the second time a commercial company had achieved the feat and a first for a non-US company. ispace's Mission 1 (M1) probably crashed during a landing attempt in April 2023. Lunar landings are challenging. When M1 crashed, Ryo Ujiie, ispace's chief technology officer said the telemetry — which collects data on the craft's altitude and speed — estimated that M1 was on the surface when it wasn't, causing the lander to free fall. Speaking to Nature last week, Ujiie said the company had addressed the telemetry issue with M2 and modified its software. 'We also carefully selected how to approach the landing site,' he added. Had M2 landed successfully, the craft would have supplied electricity for its cargo, including water electrolyzing equipment and a module for food production experiments — developed by Japan-based Takasago Thermal Engineering and biotechnology firm Euglena. A deep space radiation probe made by Taiwan's National Central University, and the 54-centimetre Tenacious rover were also be on board. The rover, created by ispace's European subsidiary in Luxemburg, was going to be released from the lander to collect imagery, location data and lunar sand known as regolith. Tenacious also carries a small red house made by Swedish artist Mikael Genberg. The craft launched on 15 January from Cape Canaveral, Florida, onboard a SpaceX Falcon 9 rocket. The rocket was also carrying the Blue Ghost Moon lander — developed by Firefly Aerospace, an aerospace firm based in Texas — which landed on the Moon on 2 March. M2 took a longer path to the moon than Blue Ghost, performing a lunar flyby on 15 February and spending two months in a low-energy transfer orbit before entering lunar orbit on 7 May. Ujiie says the path was slower because it was a low-energy trajectory, meaning that less fuel was used to move between Earth and lunar orbit. Richard de Grijs, an astronomer at Macquarie University in Sydney, Australia, says there will likely be more private companies trying to land their own crafts on the Moon. 'It seems that the big government players like NASA are quite keen to partner with commercial companies,' he says, because they can develop and launch crafts more cheaply than government bodies. He also expects that more missions will be launched in clusters, like the launch of M2 and Blue Ghost. This article is reproduced with permission and was first published on June 6, 2025.
