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Gizmodo
11 hours ago
- Health
- Gizmodo
When Will Genetically Modifying Our Children Go Mainstream?
In late May, several scientific organizations, including the International Society for Cell and Gene Therapy (ISCT), banded together to call for a 10-year moratorium on using CRISPR and related technologies to pursue human heritable germline editing. The declaration also outlined practical steps that countries and research institutions could take to discourage this sort of experimentation, such as strengthening regulations tied to gene editing. 'Germline editing has very serious safety concerns that could have irreversible consequences,' said Bruce Levine, a cancer gene therapy researcher at the University of Pennsylvania and former president of the ISCT, in a statement. 'We simply lack the tools to make it safe now and for at least the next 10 years.' Newer technologies such as CRISPR have made gene editing easier, cheaper, and more practical to carry out in a variety of species, humans included. That reality has made heritable germline editing—altering egg, sperm, and embryos such that they can be passed down to offspring—more feasible than ever. In November 2018, Chinese scientist He Jiankui thrust this issue into the limelight when he announced that his team modified the genes of several human embryos using CRISPR, then implanted them successfully in women volunteers. Eventually three children were born with the modifications, intended to confer natural immunity to HIV infection. He deliberately flouted ethical guidelines and the law in his research, such as doctoring lab results so that HIV-positive men could father the children (according to He, the children were born without HIV and appeared to have avoided any related health issues). He's experiments were roundly condemned by the scientific community and he ultimately served a three-year prison term for his actions, which ended in 2022. Upon release, He went back to working in the gene-editing field, though he promised to abide by domestic and international rules. The episode showed that human heritable germline editing is already clearly possible today, but not necessarily ethical to carry out. Indeed, many scientists and bioethicists believe we're not ready to go down that path just yet. For this Giz Asks, we reached out to several bioethicists to get their take on the moratorium, and more broadly, on the question of when we should be able to genetically modify children, if ever. Founding head of the Division of Medical Ethics at New York University's Grossman School of Medicine's Department of Population Health. I've been thinking about that question for well over 40 years. We didn't always have the technology to go in and modify genes in an egg, sperm, embryo, or fetus for that matter. But it's certainly the case that people have been thinking hard about trying to genetically alter and improve children, probably back to the Greeks. We know that in modern times, Nazi Germany was home to race hygiene theory and a form of eugenics; they would have been very interested in creating better babies. They did have the Lebensborn Program where they tried to force women and men that they deemed especially genetically fit to breed and have kids. It's not really clear whatever happened to those kids. But it's a form, if you will, of trying to get the right genes into your offspring and get them passed along into the future. They practiced that. And we had versions of that in the U.S., believe it or not. We actually had awards given at state fairs to families that were seen as eugenically the best and trying to encourage those families to have bigger families. That's an idea that's still rattling around today, by the way, in the mouths of Donald Trump, Elon Musk, Stephen Miller, etc. Many in the current Trump administration are very concerned about minorities becoming the majority in the U.S. In any event, these are old fashioned ideas, often fueled by dreams of eugenics, shifting the population in the future toward healthier, more competent, more physically able people, trying to get people of the right race or ethnicity so that the society's makeup is proper. They don't rely on engineering a gene. There's no CRISPR. There's nobody going in there and trying to penetrate the cell wall to insert genetic information. But those are just new ways to think about ideas that have been around for a long time. So if you ask me, will we see genetic engineering of children aimed at their improvement? I say yes, undoubtedly. Now when? I'm not sure what the answer to that is. Right now, we have some crude tools. We are seeing some efforts to use gene therapy in kids to repair diseases of their bodies, not things that would be inherited. They work a bit, but I wouldn't say we're really at the sort of utopia of being able to reliably get rid of in a person or a child, sickle cell or other major diseases. The tools, despite a lot of hype and a lot of maybe press release journalism, are not quite there yet to really say we can even do a good job repairing disease in an existing kid. So when it comes to trying to use tools to modify an embryo, I'm going to say flat out we're at least 10 years away from that in any serious way that could be considered safe, targeted, and likely to produce the outcome you want. So the big restriction now is safety. I think we'll get past safety, but it is a reason right now not to do anything. Now, what else might become an objection if we did have accurate, sophisticated tools? I think the first is access. If you make better kids, but only some people can afford it, that wouldn't be fair. And that in itself would be unjust. And you might wind up creating two classes or more of humans on Earth, the genetically engineered superior people and others. And this obviously is a theme all over science fiction. Old-timers will remember the Wrath of Khan from Star Trek for their take on what happens when you get a super genetically engineered race. There's Gattaca, another movie that explored this. But I'm going to say this somewhat controversially. Fairness in access never stopped a technology from going forward. When the rich and the middle class want it, they're not stopped by the fact that the poor can't get it. I would like to see provisions made to say we shouldn't move forward unless those technologies are available to those who want them regardless of cost. But I don't think that's going to happen. It's just never happened. So access is an issue, but I don't think it's a game-breaker for improving your kids. People also say, well, how will we improve? I mean, what's the best state? We can't agree on that. So will we really improve kids? There may be things we disagree about as to whether they're really improvements. Would it be an improvement to diminish pigment in black people? Try and make them less dark. We can certainly see that argued. There are plenty in the deaf community who say, well, deafness is not really something you have to get rid of or try to improve by genetically engineering hearing to make it better. They can get around the world deaf using a different language and different institutions. But there are clearly things that it would be nice to genetically improve in kids. Immunity would be great. We do it now with vaccines. It would be great to find the right genes, tweak them, and build stronger immune systems. It would be great to make sure that we try our best to diminish the extreme pain, that some of us suffer not just as disease, but with respect to certain stimuli. I'm not saying we should genetically eliminate all pain. That would probably put us in danger, but we don't quite have to suffer the way we do. My point being, the fact that we don't agree on everything as to what would be an improvement doesn't mean that we can't agree on anything. The last thing I'll say is this. When you try to make better kids, I think one last concern is: Are you going to make the children have less options rather than more? So if I considered it an improvement in a child to make them a giant, or to make them a tennis player, or to try and figure out perhaps some weird appearance that would make them a celebrity, I'm condemning the child to my choice. They don't have the freedom to run their own life. They don't have the ability to choose what they want to do. I tighten down their future by narrowing the kinds of traits they have. That, I think, is a legitimate objection. We have to think hard about that. Many of the things we do environmentally, learn to read better, learn to do exercise, learn to play games, these are skills that expand capacities in our children, and may in fact be values that are then passed on to future generations. But they don't wind up creating kids who are less capable because of those interventions. That's where genetic change has to be watched very closely. So the bottom line of this gigantic speech is yes, we will see genetic modification of our children. It will come. There are traits that people will eagerly try to put into their kids in the future. They will try to design out genetic diseases, get rid of them. They will try to build in capacities and abilities that they agree are really wonderful. Will we hang up these interventions on ethical grounds? For the most part, no, would be my prediction, But not within the next 10 years. The tools are still too crude. Associate professor of bioethics at Case Western Reserve University's School of Medicine There are children with genetic modifications walking around today, children like KJ, who was treated with personalized CRISPR gene editing at just six months old. There are now kids who are free of sickle cell disease symptoms through CRISPR therapy, the first one ever approved by the FDA. All of these children are 'genetically modified,' and they and their parents couldn't be happier about it. What other conditions could and should be treated through genetic modifications? That's a question that scientists are actively working on, and that social scientists like me are talking about with patients, parents, and communities—because we and they think it's really important for them to be part of those decisions. These 'somatic' gene editing treatments that are already being used aren't the kind that is passed down through our reproductive cells, the germline. Heritable gene modification would involve embryos, eggs, or sperm, or even possibly other cells that could be turned into these kinds of cells. A technology currently being researched, called in vitro gametogenesis, could use gene editing to turn skin cells into reproductive cells, allowing families with infertility to have their own genetically related children. And of course, there are scientists looking at the possibility of editing reproductive cells to allow couples who carry the genes for severe diseases to conceive children without those conditions. Many ethicists and scientists have drawn a hard line between heritable and non-heritable gene editing, but in practice it's not nearly so clear-cut. Off-target effects of gene editing are difficult to predict or control, so there is a chance that reproductive cells could be changed by treatments aimed at other organ systems. Fetal gene editing, which could help babies with some conditions be born with few or no symptoms, will also involve the pregnant bodies of their mothers; those adults could host edited cells even after the pregnancy ends, possibly affecting their future children too. Families dealing with genetic conditions that cause great suffering for their children don't necessarily see a problem with eliminating those conditions forever with heritable gene editing. On the other hand, some people living with genetic conditions, such as deafness or autism, see no reason for treating their condition with gene editing, heritable or not, because their biggest problems come not from the condition itself but from the way society treats them. So there are many questions to be asked about all forms of genetic modification, and how they will be developed and implemented. All the gene editing treatments that exist now or are being imagined over the next decade, heritable or not, involve exorbitant cost and will be inaccessible to most people worldwide. It will be crucial to balance the excitement of these novel technologies with attention to questions of justice, developing new treatments with an eye toward both accessibility and the priorities of those most affected. The only way to do this is to bring more voices into conversation with one another: people living with genetic conditions, scientists and doctors, policymakers of all kinds, and members of the public. Although gene editing is an amazing tool to add to our kit, the work of building more robust healthcare and support for families carrying or living with genetic conditions doesn't begin or end with genetic modification. Bioethicist, sociologist, and executive director of the Institute for Ethics and Emerging Technologies. Yes we should, when it's safe, effective, and voluntary. Calls to permanently ban the creation of genetically modified children often rest on fear, not facts. They mirror past moral panics over interracial marriage, in vitro fertilization, and birth control—all technologies or choices once deemed unnatural or dangerous, and now widely accepted. We should be wary of arguments dressed up as ethics but rooted in anxiety about change. That doesn't mean anything goes. Like any powerful technology, gene editing must be tightly regulated for safety and efficacy. But the agencies we already trust to regulate medicine—the FDA, NIH, and institutional review boards—are largely capable of doing that. We don't need a bioethics priesthood or a new bureaucracy to police reproductive decisions. We need science-based oversight, individual consent, and protection from coercion. One of the loudest objections to genetic editing is the specter of 'eugenics.' But if eugenics means state control over reproduction, then the lesson of the 20th century is to defend reproductive freedom, not curtail it. Governments should not tell parents what kinds of kids to have. Preventing parents from using safe, approved gene therapies to reduce suffering or enhance their children's lives is a strange way to honor that lesson. They should give parents access to all the information and technology for the choices they make. True reproductive liberty includes the right to use the best science available to ensure a child's health. Another objection is that genetic modification could harm people who would rather not participate. But this 'perfection anxiety' ignores how all medical advances shift social norms. We didn't stop improving dental care because it made bad teeth less acceptable. And a healthier society has not led to less compassion for those who remain sick or disabled—if anything, it's strengthened the case for inclusion and support. The goal should be equitable access, not frozen norms. We do need to ensure that parents can access all the gene therapies that actually provide potential benefits for children. Governments with universal healthcare will need to make tough choices about what to cover and what not to cover. For instance, the National Health Service should make gene therapy to remove lethal, painful conditions available for all Britons, but parents may need to pay for medical tourism to some offshore clinic if they want to tweak their embryo's eye color. What about risks we can't foresee? Of course there will be some. All new medical therapies come with uncertainties. That's why we have trials, regulation, and post-market surveillance. There's no reason genetic therapies should be held to an impossibly higher standard. We should start with animal models, and proceed to the most morally defensible gene tweaks, lethal and painful conditions. Over time, as the safety of the techniques are better understood, we can expand the scope of therapeutic choices. Some worry that genetically modified children could disrupt our ideas of family or humanity. But those concepts have already been revolutionized—by urbanization, feminism, economic precarity, and social movements. The family of today would be unrecognizable to most people in 1800. If genetic technologies change our values again, it won't be the first time. Liberal democracies don't freeze culture in place—they ensure people have the freedom to shape it. Ultimately, the question isn't whether we should allow genetically modified children. It's whether we trust parents to make mostly good choices under the oversight of regulators and doctors. We should, because most parents have their children's best interests in mind, as they perceive them. That's why we allow parents to raise their own children in the first place. And we should ensure those choices are equitably available to all, not outlawed out of fear. If we ever find genetic tweaks to reduce suffering, enhance capability, or prevent devastating disease—and we can do so safely and ethically—the real moral failure would be to prohibit it. A Canadian bioethicist and environmentalist currently teaching at the University of Toronto. Well, there's a big difference between genetic enhancement and treatment. And with enhancement, I think we're nowhere near a point where we should be even considering that. But with treatment, the large ethical issue right now is something like single gene mutation. So something like Huntington's disease, muscular dystrophy, or similar diseases, could it be justified to edit the gene for that? The challenge is we don't fully understand all the things. We don't know what we don't know, to put it bluntly. And with germline editing, the changes we would be making are permanent and they run through many generations ahead. So, yes, being able to prevent deadly or debilitating illnesses is absolutely something wonderful. But having said that, you obviously don't have consent of the person who will be born, but you also don't have consent of the generations that come after that. And if there is complications or unexpected problems, you can have an inheritance that just keeps running through generations. But here's the thing with this moratorium; to what end? You can call for a moratorium, but if no one's focusing on anything, if there's no research, no planning, no social discourse, there's just a lot of people with different opinions, and everything gets shelved for 10 years. I'm not sure that's going to be particularly useful. It sounds great if it's going to be 10 concentrated years on building consensus and public engagement and those types of things, but I don't think that's what would actually happen. And also, I'm sure you've noticed, the world's not in good shape, and Western culture is not of one mind these days. And with the ruptures, particularly in the United States, there's a lot of division in Western culture of how people see things. And I'm just not convinced that a moratorium, that people would make use of it in a constructive way. It really needs a coordinated plan, and I'm not sure there is one. So I do see that as quite a problem. The other thing is, we're dealing with high-income countries. So when we look at potential for CRISPR-Cas9 and gene editing, we're dealing with a very small percentage of the world's population. I'm going to guess that it's maybe 15% to 20% of the world's population, because most of the population of the world has no access to things like this and never will. Not never will, but in the foreseeable future, they won't. And I think that's something we miss a lot of the time. And the biggest ethical problem in the world today is not gene editing. It's just access to healthcare. And this doesn't do anything in those domains whatsoever. So from a justice point of view, that is a concern. And I'm going to sound cynical here. Emerging medical technologies are not motivated largely by the social sector. They're motivated by marketing and market forces. So if people can make money on this, somehow, someway, people will proceed. And if gene editing is illegal in Canada and the U.S. and Western Europe and Australia, there's a lot of countries that don't fall into that. And you can set up shop anywhere. Equatorial Guinea or other places are not going to be worried about things like this. They've got enough problems on their hands. And there's a lot of countries out there where this would not be easily called. So I support the essence of it. And I can see why people want to do it. I'm just not convinced it's all that feasible. I think what makes more sense is just not having any germline editing until we have a larger consensus about this technology.
Yahoo
15-05-2025
- Business
- Yahoo
BrainStorm Cell Therapeutics Announces First Quarter 2025 Financial Results and Provides Corporate Update
Conference call and webcast rescheduled for 8:30 a.m. Eastern Time on Monday, May 19 NEW YORK, May 15, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced financial results for the first quarter ended March 31, 2025, and provided a corporate update. "BrainStorm continues to make meaningful progress in advancing our NurOwn® development program. Our priority continues to be initiation of a Phase 3b trial, designed to confirm the product's efficacy in early stage ALS patients and support a new BLA," said Chaim Lebovits, President and Chief Executive Officer of BrainStorm. "We have a clear path forward and ongoing support from the ALS community. We recently submitted an amendment to our IND, which includes updated documentation that is essential for regulatory compliance and trial integrity. We are in the process of finalization clinical trial agreements with leading academic centers and completing other necessary steps on trial execution and manufacturing. We believe that NurOwn, if approved, has the potential to become a valuable treatment option for ALS patients. Our team is fully aligned and executing with discipline to position BrainStorm for success." Recent Highlights NurOwn (MSC-NTF) for ALS IND amendment on NurOwn submitted to FDA BrainStorm submitted an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration for NurOwn. This important milestone sets the stage for the initiation of the planned Phase 3b clinical trial, which has been designed in collaboration with the FDA under a Special Protocol Assessment (SPA). The trial will have a primary efficacy endpoint assessing changes in ALSFRS-R scores from baseline to week 24, and is designed to enroll approximately 200 ALS participants with early stage disease. Successful completion of the double blind part of the study (Part A) is expected to generate the clinical data to support a new BLA submission. Phase 3b trial listed on Details of the trial, known as ENDURANCE, are now available on ID NCT06973629. Included is a list of 15 clinical trial sites that are expected to participate in the trial. NurOwn® data selected as Breakthrough Science for Presentation at ISCT 2025 Meeting The new pharmacogenomic were delivered in a oral presentation at the International Society for Cell & Gene Therapy (ISCT) 2025 Annual Meeting, in New Orleans. The data highlight the impact of the UNC13A genotype on clinical outcomes for ALS patients treated with NurOwn. The presentation was featured in the ISCT public announcement regarding the meeting, which indicated that the data were "carefully reviewed and selected by the ISCT 2025 Planning Faculty, to explore the latest breakthroughs in the clinical translation of Mesenchymal Stem/Stromal Cells and how they will shape the future of cell therapies." Biomarker insights supporting NurOwn's mechanism and clinical impact presented at the 2025 ALS Drug Development Summit BrainStorm's senior leadership team (Dr. Bob Dagher, Dr. Netta Blondheim-Shraga and May Kay Turner) participated in sessions at the summit that highlighted insights and expertise gained throughout the NurOwn® development program. These sessions included a presentation on cerebrospinal fluid (CSF) biomarker pathways associated with NurOwn treatment, including their relationship to clinical outcomes and disease heterogeneity in ALS. Financial Results for the First Quarter Ended March 31, 2025 Cash, cash equivalents, and restricted cash were approximately $1.8 million as of March 31, 2025. Research and development expenditures, net, for the quarter ended March 31, 2025 were $1.3 million, compared to $1.0 million for the quarter ended March 31, 2024. General and administrative expenses for the quarter ended March 31, 2025 were approximately $1.8 million, compared to approximately $1.5 million for the quarter ended March 31, 2024. Net loss for the quarter ended March 31, 2025, was approximately $2.9 million, as compared to a net loss of approximately $3.4 million for the quarter ended March 31, 2024. Net loss per share for the three months ended March 31, 2025, and 2024 was $0.45 and $0.75, respectively. Conference Call and Webcast Monday, May 19, 2025, at 8:30 a.m. U.S. Eastern Time Participant Numbers: U.S. dial in: 888-506-0062 International: 973-528-0011 Participant Access Code: 621608 Webcast URL: The replay of the conference call can be accessed by dialing the numbers below and will be available for 14 days. Teleconference Replay Number: Toll Free: 877-481-4010 International: 919-882-2331 Passcode: 52457 About NurOwn® The NurOwn® technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. About BrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn® platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection. NurOwn® is BrainStorm's lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). A Phase 3 trial in ALS (NCT03280056) has been completed, and a second Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA. The NurOwn clinical program has generated valuable insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A genotype, biomarker data collected at seven longitudinal time points, and a comprehensive analysis of the "Floor Effect" — a critical challenge in measuring clinical outcomes in advanced ALS. BrainStorm has published its findings in multiple peer-reviewed journals. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society. BrainStorm is also advancing a proprietary, allogeneic exosome-based platform designed to deliver therapeutic proteins and nucleic acids. The company recently received a Notice of Allowance from the U.S. Patent and Trademark Office for a foundational patent covering its exosome technology, further strengthening BrainStorm's growing IP portfolio in this emerging area of regenerative medicine. To learn more, visit Notice Regarding Forward-Looking Statements This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. CONTACTSInvestors:Michael WoodPhone: +1 646-597-6983mwood@ Media: Uri Yablonka, Chief Business OfficerPhone: +1 917-284-2911uri@ BRAINSTORM CELL THERAPEUTICS INC. AND SUBSIDIARIES INTERIM CONDENSED CONSOLIDATED BALANCE SHEETS U.S. dollars in thousands (Except share data)March 31,December 31, 20252024 UnauditedAudited U.S. $ in thousands ASSETSCurrent Assets: Cash and cash equivalents$ 1,644$ 187 Other accounts receivable 67 63 Prepaid expenses and other current assets 621 135 Total current assets$ 2,332$ 385Long-Term Assets: Prepaid expenses and other long-term assets $ 22$ 22 Restricted Cash 182 184 Operating lease right of use asset (Note 4) 653 807 Property and Equipment, Net 382 434 Total Long-Term Assets$ 1,239$ 1,447Total assets$ 3,571$ 1,832LIABILITIES AND STOCKHOLDERS' EQUITY (DEFICIT)Current Liabilities: Accounts payables$ 6,797$ 6,080 Accrued expenses 538 619 Short-term loans (Note 7) 1,200 300 Operating lease liability (Note 4) 443 549 Employees related liability 1,923 1,430 Total current liabilities$ 10,901$ 8,978Long-Term Liabilities: Operating lease liability (Note 4) 127 171 Warrants liability (Note 5) - 447 Total long-term liabilities$ 127$ 618Total liabilities$ 11,028$ 9,596Stockholders' Deficit: Stock capital: (Note 6) 15 14 Common Stock of $0.00005 par value - Authorized: 250,000,000 shares at March 31, 2025 and at December 31, 2024 respectively; Issued and outstanding: 7,911,204 and 6,141,762 shares at March31, 2025 and December 31, 2024 respectively (*) Additional paid-in-capital 222,144 218,974 Treasury stocks (116) (116) Accumulated deficit (229,500) (226,636) Total stockholders' deficit$ (7,457)$ (7,764)Total liabilities and stockholders' deficit$ 3,571$ 1,832 The accompanying notes are an integral part of the consolidated financial statements. BRAINSTORM CELL THERAPEUTICS INC. AND SUBSIDIARIES INTERIM CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (UNAUDITED) U.S. dollars in thousands (Except share data) Three months ended March 31, 20252024Unaudited Operating expenses:Research and development, net $ 1,304$ 961 General and administrative1,785 1,513 Operating loss(3,089) (2,474) Financial income, net46 13 Loss on change in fair value of Warrants liability (Note 6)(179) (940) Net loss $ (2,864)$ (3,401) Basic and diluted net loss per share from continuing operations $ (0.45)$ (*) (0.75) Weighted average number of shares outstanding used in computing basic and diluted net loss per share6,342,002 (*) 4,315,903 The accompanying notes are an integral part of the consolidated financial statements. Logo: View original content: SOURCE BrainStorm Cell Therapeutics Inc.
Associated Press
13-05-2025
- Business
- Associated Press
Phase 2 Trial of BRTX-100 in cLDD Continues to Generate Positive Preliminary Blinded Data
MELVILLE, N.Y., May 13, 2025 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. ('BioRestorative', 'BRTX' or the 'Company') (NASDAQ: BRTX ), a clinical stage regenerative medicine innovator focused on stem cell-based therapies and products, is pleased to announce that preliminary 26-, 52- and 104-week blinded preliminary data from the first 15 patients with chronic lumbar disc disease ('cLDD') enrolled in the ongoing Phase 2 clinical trial of BRTX-100 was recently presented by Francisco Silva, Vice President of Research and Development, at the International Society for Cell & Gene Therapy ('ISCT') 2025 Annual Meeting. No serious adverse events (SAEs) were reported, and there was no dose (40X106 cells) limiting toxicity at 26-104 weeks. The U.S. Food and Drug Association (FDA) is requiring at least a greater than 30% improvement in both Oswestry Disability Index ('ODI') and Visual Analog Scale ('VAS' ); ongoing clinical data demonstrates trends greater than 30% improvements in both measures. The following is breakdown of the percentage of subjects that had both a greater than 50% improvement in function, as measured by ODI, and a greater than 50% decrease in pain, as measured by VAS: 'We were pleased that blinded data from the ongoing Phase 2 trial of BRTX-100 in cLDD continues to be in-line to meet the primary safety endpoint of study, and that the positive preliminary efficacy trends also continue,' said Lance Alstodt, Chief Executive Officer of BioRestorative. Mr. Silva's ISCT presentation, titled 'Phase 2 Clinical Safety/Efficacy Data of Intradiscal Injection of Hypoxic Mesenchymal Stem Cells for Lumbar Disc Disease,' can be accessed on the Company's new website under 'Scientific Publications' in the Product Candidate section. About the BRTX-100 Phase 2 Trial in cLDD BRTX-100, a novel cell-based therapeutic engineered to target areas of the body that have little blood flow, is the Company's lead clinical candidate. The safety and efficacy of BRTX-100 in treating cLDD is being evaluated in a Phase 2, prospective, randomized, double-blinded and controlled study. A total of up to 99 eligible subjects will be enrolled at up to 16 clinical sites in the United States. Subjects included in the trial will be randomized 2:1 to receive either BRTX-100 or placebo. About BioRestorative Therapies, Inc. BioRestorative ( ) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. As described below, our two core clinical development programs relate to the treatment of disc/spine disease and metabolic disorders, and we also operate a commercial BioCosmeceutical platform: • Disc/Spine Program (brtxDISC™): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a person's own) cultured mesenchymal stem cells collected from the patient's bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The BRTX-100 production process utilizes proprietary technology and involves collecting a patient's bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patient's damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have commenced a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain arising from degenerative disc disease. We have also obtained FDA IND clearance to evaluate BRTX-100 in the treatment of chronic cervical discogenic pain. • Metabolic Program (ThermoStem®): We are developing cell-based therapy candidates to target obesity and metabolic disorders using brown adipose (fat) derived stem cells ('BADSC') to generate brown adipose tissue ('BAT'), as well as exosomes secreted by BADSC. BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes. BADSC secreted exosomes may also impact weight loss. • BioCosmeceuticals: We operate a commercial BioCosmeceutical platform. Our current commercial product, formulated and manufactured using our cGMP ISO-7 certified clean room, is a cell-based secretome containing exosomes, proteins and growth factors. This proprietary biologic serum has been specifically engineered by us to reduce the appearance of fine lines and wrinkles and bring forth other areas of cosmetic effectiveness. Moving forward, we also intend to explore the potential of expanding our commercial offering to include a broader family of cell-based biologic aesthetic products and therapeutics via Investigational New Drug (IND)-enabling studies, with the aim of pioneering U.S. Food and Drug Administration (FDA) approvals in the emerging BioCosmeceuticals space. Forward-Looking StatementsCONTACT: Stephen Kilmer Investor Relations Direct: (646) 274-3580 Email: [email protected]
Korea Herald
07-05-2025
- Business
- Korea Herald
Minaris Advanced Therapies launches as the leading global partner dedicated to cell therapy development, manufacturing, and testing
New company unveiled at the annual ISCT conference in New Orleans combines two tenured and innovative CDMOs with a testing powerhouse PHILADELPHIA, May 7, 2025 /PRNewswire/ -- Through strategic acquisitions made by New York–based investment firm Altaris, Minaris Regenerative Medicine and the U.S. and U.K. operations of WuXi Advanced Therapies have been combined to form Minaris Advanced Therapies ™, a global cell therapy CDMO and testing partner. The company is headquartered in Philadelphia, Pennsylvania. With modern facilities approved for commercial production in the United States (Allendale, NJ and Philadelphia), Europe (Munich) and Asia-Pacific (Yokohama, Japan), Minaris Advanced Therapies manufactures clinical-stage and commercial products on three continents and has a proven track record of delivering more than 7,500 GMP batches. The company currently manufactures two commercial cell therapies and provides testing services for more than 27 commercial products. The company's capabilities include innovative platforms, development and manufacturing capabilities for cell therapies and viral vectors, and extensive testing services. With more than 25 years of cell therapy CDMO experience and over 40 years of experience in biosafety testing and product characterization, the new company will support the next wave of commercial cell therapies. "CDMOs supporting the cell therapy industry have struggled to evolve from a small-scale cottage industry to deliver cost effective manufacturing at scale," said Iain Baird, chairman of Minaris Advanced Therapies. "Our mission is to solve the development and manufacturing challenges preventing these promising therapies from successful worldwide commercialization." The team includes more than 1,400 industry professionals with deep scientific and regulatory expertise. The company's testing business unit provides unrivalled expertise in analytical method development and GMP-grade biosafety and product characterization testing services for both in-house and external programs. "Minaris Advanced Therapies is designed to help the industry treat more patients by providing the experience, global footprint, and platforms to accelerate timelines and enable commercialization," said Eytan Abraham, Ph.D., chief commercial and technology officer. "Many of the technologies needed to reduce cost of goods sold and improve turnaround time already exist, we will drive their adoption and implementation at scale." Built for speed, scale and science Minaris Advanced Therapies brings together: About Minaris Advanced Therapies Minaris Advanced Therapies is a global contract development and manufacturing organization (CDMO) and contract testing provider focused exclusively on cell and gene therapies. Minaris Advanced Therapies is headquartered in Philadelphia, Pennsylvania, and has more than 730,000 square feet of infrastructure across the United States, Europe and Asia. We have manufactured and released over 7,500 GMP batches and our global network supports therapy developers through early-stage development, clinical trials and commercial manufacturing. By combining scientific expertise with best-in-class manufacturing and testing services, we help bring safe, effective therapies to patients faster and more efficiently. Visit to learn more.
Yahoo
29-04-2025
- Health
- Yahoo
BrainStorm's NurOwn® Data Selected as Breakthrough Science for Presentation at ISCT 2025 Meeting
New findings highlight impact of UNC13A genotype on treatment response in ALS NEW YORK, April 29, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, in collaboration with leading ALS research centers, today announced the acceptance of new pharmacogenomic data for oral presentation of new findings on NurOwn® at the International Society for Cell & Gene Therapy (ISCT) 2025 Annual Meeting, to take place from May 6 - 10, 2025 in New Orleans. The data highlight the impact of the UNC13A genotype on clinical outcomes for amyotrophic lateral sclerosis (ALS) patients treated with NurOwn® (debamestrocel). The presentation was featured in the ISCT public announcement, which indicated that the data were "carefully reviewed and selected by the ISCT 2025 Planning Faculty, to explore the latest breakthroughs in the clinical translation of Mesenchymal Stem/Stromal Cells and how they will shape the future of cell therapies." "We look forward to sharing the latest scientific data on NurOwn at this year's ISCT meeting. These new results are particularly meaningful as they explore a genetic factor that may help predict treatment response. The feedback received from the ALS experts on these new data continues to be encouraging as we advance in our mission to address the unmet needs of patients with this devastating disease," said Chaim Lebovits, President and CEO of BrainStorm. Mr. Lebovits added, "Our immediate priority is to launch the Phase 3b trial of NurOwn to build on our clinical data and demonstrate its potential in early ALS. We are enjoying positive communication with the FDA, have submitted an IND amendment, and are implementing the SPA exactly as agreed with the Agency. We're fully prepared to begin the study." Dr. Bob Dagher, Executive Vice President & Chief Medical Officer at BrainStorm, commented, "These latest results offer additional scientific insights into our completed Phase 3 NurOwn trial and increase our understanding of treatment effects in patient subpopulations. These types of analysis provide important information for the development of future ALS treatments and add to the growing body of evidence supporting NurOwn®." Dr. Dagher added, "This represents a first-of-its-kind pharmacogenomic analysis from the Phase 3 trial in ALS and is among the first ALS studies to analyze how genetic differences, specifically the UNC13A genotype, affect patient response to therapy. Understanding how genetics can influence treatment response will help move us closer to truly personalized medicine for ALS." Presentation details: Title: Debamestrocel Effect on Clinical and Biomarker Endpoints by UNC13A Genotype in Phase 3 ALS TrialLead Author Bob Dagher, MDOral Session: Mesenchymal Stem/Stromal Cells #1Date and time: Date & Time: Thursday, May 8, 2025 | 9:00 – 10:00 a.m. U.S. Central Ernest N. Morial Convention Center, 900 Convention Center Boulevard, New Orleans, Louisiana 70130 A copy of the poster will be available on the BrainStorm corporate website, at the conclusion of the ISCT meeting. About Brainstorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn® platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection. NurOwn® is BrainStorm's lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). A Phase 3 trial in ALS (NCT03280056) has been completed, and a confirmatory Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA. The NurOwn clinical program has generated valuable insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A genotype, biomarker data collected at seven longitudinal time points, and a comprehensive analysis of the "Floor Effect" — a critical challenge in measuring clinical outcomes in advanced ALS. BrainStorm has published its findings in multiple peer-reviewed journals. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society. BrainStorm is also advancing a proprietary, allogeneic exosome-based platform designed to deliver therapeutic proteins and nucleic acids. The company recently received a Notice of Allowance from the U.S. Patent and Trademark Office for a foundational patent covering its exosome technology, further strengthening BrainStorm's growing IP portfolio in this emerging area of regenerative medicine. To learn more, visit Notice Regarding Forward-Looking Statements This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), ADCOM meeting related to NurOwn, the timing of a PDUFA action date for the BLA for NurOwn, the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. CONTACT:Michael WoodPhone: +1 646-597-6983mwood@ Logo: View original content: SOURCE BrainStorm Cell Therapeutics Inc. Sign in to access your portfolio
