Pioneering Technologies in Regenerative Medicine
Key Growth Drivers and Opportunities
Rising Number of Aging Population: With the global aging phenomenon, there will be increased incidences of chronic and degenerative diseases such as osteoarthritis, cardiovascular ailments, neurodegenerations, and musculo-skeletal problems, very much in demand for advanced treatment options. Regenerative medicine comprising stem cell therapy, gene therapy, and tissue engineering, among others, presents viable and sustainable alternatives in contrast to conventional treatments such as medication and surgery, which provide temporary relief. Given that regenerative medicine addresses the actual cause of tissue and organ damage, it significantly enhances the quality of life and decreases long-term care burdens, serving as the prime growth driver for the regenerative medicine.
Challenges
The regenerative medicine is limited by high treatment costs, difficult regulatory approvals, not enough long-term clinical data, and problems in big manufacturing and making everything the same which can block easy acceptance and availability.
Innovation and Expansion
Beam Therapeutics' BEAM-302 has granted RMAT status by the FDA for Alpha-1 Antitrypsin Deficiency
In May 2025, The U.S. Food and Drug Administration (FDA) has designated BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor, as Regenerative Medicine Advanced Therapy (RMAT). This designation is intended to correct the disease-causing mutation in patients with alpha-1 antitrypsin deficiency (AATD).
Beam Therapeutics Inc. is a biotechnology company that develops precision genetic medicines through base editing. There is a substantial unmet need for efficient treatments that may address the whole range of symptoms for AATD, an inherited genetic illness that affects the lungs and/or liver and causes early onset emphysema and liver disease.
The FDA Gives BrainChild Bio's CAR T Therapy for Pediatric Brain Tumors RMAT Status
In May 2025, The U.S. Food and Drug Administration (FDA) has designated the investigational B7-H3 targeting autologous CAR T-cell therapy as a Regenerative Medicine Advanced Therapy (RMAT) for the treatment of diffuse intrinsic pontine glioma (DIPG), an incurable pediatric brain tumor. This news was released by BrainChild Bio, Inc., a clinical-stage biotechnology company that is developing CAR T-cell therapies to treat tumors in the central nervous system (CNS).
The FDA Designates MeiraGTx's AAV-GAD Gene Therapy as an RMAT for Parkinson's disease
In May 2025, AAV-GAD has been designated by the U.S. Food and Drug Administration (FDA) as Regenerative Medicine Advanced Therapy (RMAT) for the treatment of Parkinson's disease that is not sufficiently controlled with anti-Parkinsonian drugs. This announcement was made today by MeiraGTx Holdings plc, a vertically integrated, clinical-stage genetic medicines company.
This RMAT was granted after the FDA received favorable results from three clinical trials showing the advantages of AAV-GAD when given as a single stereotactic infusion to the brain's subthalamic nucleus.
Inventive Sparks, Expanding Markets
Key players in the regenerative medicine market are R&D Systems, Inc., Moderna, Novartis AG and others. These key players are focusing on increasing the research and development of advanced therapies and innovative strategies for 3D bioprinting advancement, and more acquisitions to enhance product offerings through collaborations will be projected to drive the target market.
About Author:
Prophecy is a specialized market research, analytics, marketing and business strategy, and solutions company that offer strategic and tactical support to clients for making well-informed business decisions and to identify and achieve high value opportunities in the target business area. Also, we help our client to address business challenges and provide best possible solutions to overcome them and transform their business.
TIME BUSINESS NEWS
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
The Hill
17 minutes ago
- The Hill
Mars says M&M's, Starbursts without synthetic dyes coming in 2026
Video above: FDA announces in April 2025 that it will phase out food dyes. (NEXSTAR) — While several other food companies had confirmed they would drop artificial food dyes in response to a mission set forth by Health and Human Services Secretary Robert F. Kennedy Jr. earlier this summer, Mars Wrigley was considered a holdout. That quietly changed late last month. In a late-July statement, which was first reported by Bloomberg on Thursday, Mars Wrigley North America announced it plans to release 'product options' made without artificial dyes in 2026. These options without synthetic dyes will be available only across four of its brands to start, according to the statement: M&M's, Skittles Original, Extra Gum Spearmint, and Starburst Original fruit chews. They'll be available to purchase online throughout the U.S. A spokesperson confirmed to Nexstar that the 'choice products' without artificial dyes will be additions to the current brands. They declined to say whether currently available products will be affected by the dye changes. AT&T reached a $177M settlement over data breaches: Will you get a payment? Mars did not immediately respond to Nexstar's request for additional information, like whether these options will replace others within each brand's portfolio, or if currently available products would be otherwise impacted. 'When we have identified fully effective, scalable solutions across the entire portfolio, we will share additional item commitments and timelines,' the company said in its statement. In April, Food and Drug Administration Commissioner Marty Makary said the agency would move to eliminate several synthetic dyes by the end of next year. That includes Green 3, Red 40, Yellow 5, Yellow 6, Blue 1, and Blue 2. Red 3 was set to be banned in food by 2027 because it caused cancer in laboratory rats; the FDA called for that deadline to move up. The FDA has been relying on companies voluntarily ditching the aforementioned dyes, but does plan to establish a standard and timeline for the food industry to switch to natural alternatives and revoke authorization for dyes not in production. Additionally, the FDA said it would authorize four new natural color additives. Several food manufacturers have already announced plans to ditch synthetic colors. That includes General Mills, Pepsico, ConAgra, Nestle, McCormick, Tyson Foods, Sam's Club, JM Smucker, Hershey, Kraft Heinz, and dozens of companies that make ice cream and frozen dairy desserts. Mars, however, was a holdout as of mid-July. The company now stands to be among the first to have products without artificial dyes available. Artificial dyes are used widely in U.S. foods. In Canada and in Europe — where synthetic colors are required to carry warning labels — manufacturers mostly use natural substitutes. Several states, including California and West Virginia, have passed laws restricting the use of artificial colors in foods. Health advocates have long called for the removal of artificial dyes from foods, citing mixed studies indicating they can cause neurobehavioral problems, including hyperactivity and attention issues, in some children. The FDA has maintained that the approved dyes are safe and that 'the totality of scientific evidence shows that most children have no adverse effects when consuming foods containing color additives.' The FDA currently allows 36 food color additives, including eight synthetic dyes. Removing dyes from the food supply will not address the chief health problems that plague Americans, said Susan Mayne, a Yale University chronic disease expert and former director of the FDA's food center. 'With every one of their announcements, they're focusing in on something that's not going to accomplish what they say it is,' Mayne said of Kennedy's initiatives. 'Most of these food dyes have been in our food supply for 100 years. … So why aren't they driving toward reductions in things that do drive chronic disease rates?'
New York Post
an hour ago
- New York Post
Costco won't sell abortion pills after outcry from religious groups
Costco said it will not offer abortion pills at any of its more than 500 pharmacies nationwide, a move celebrated by religious groups that lobbied against the drug being dispensed by retailers. The Washington state-based wholesaler insisted its decision was based on a lack of customer interest in the medication, Mifepristone, which together with another medicine called misoprostol, is used to end a pregnancy within 10 weeks of gestation. It didn't address whether pressure from conservative organizations influenced the move. 4 A Costco location in the US, one of more than 500 company pharmacies that will not provide Mifepristone. Getty Images A coalition of religious groups including Idaho-based Inspire Investing, the conservative legal organization Alliance Defending Freedom and treasurers and other financial officials from Ohio, Texas, Nevada and Utah had contacted the retailer last year requesting it not carry the drug, according to Bloomberg News. 'It's a very significant win and it's one we hope to build on this coming year,' said Michael Ross, an attorney working with Alliance Defending Freedom's corporate engagement division. Ross indicated the coalition plans to target Walgreens Boots Alliance and CVS next. Both pharmacy chains currently provide mifepristone in states that don't have abortion restrictions. People living in states that have severely limited abortion can obtain the medication by mail from states without such bans. The religious investors sent similar requests last year to Kroger, Walmart and Albertsons alongside Costco, urging them against carrying the medication. Kroger responded that it has never provided abortion pills and continues monitoring regulatory developments. Neither Walmart nor Albertsons currently stocks the medicine. Costco's decision intensifies the ongoing debate about access to the medication. 4 Mifepristone tablets are part of a two-drug regimen to terminate early pregnancies. AP New York City Comptroller Brad Lander had written to various retailers in July last year representing city pension funds, encouraging them to pursue authorization for offering the drug. 'Costco's decision to refuse to dispense mifepristone is disappointing and short-sighted,' a representative from Lander's office told Bloomberg. 'Failure to provide access to proven safe and FDA-approved medication under the guise of 'weak demand' risks isolating customers and undermines the company's credibility.' The Post has sought comment from Costco and Lander. The religious organizations have promised to continue to ramp up pressure. 'We have this momentum,' said Tim Schwarzenberger, who leads corporate engagement at Inspire, which manages the biggest religious exchange-traded fund. 'Now there is a chance to turn to some of the other retailers.' doesn't presently sell the medication, and Schwarzenberger indicated his organization will work to maintain that status. 4 A patient prepares to take mifepristone, which blocks progesterone to end an early-stage pregnancy. AP Amazon didn't provide a response to inquiries. Walgreens declined to comment but referenced information on its website stating: 'The US Supreme Court's ruling on mifepristone access allows Walgreens to continue to dispense mifepristone under the FDA guidelines.' CVS confirmed it distributes mifepristone in certain states where permitted after completing what it called a thorough certification procedure. 'We have a long history of supporting and advancing women's health and we remain focused on meeting their unique health needs. This includes providing access to safe, legal, and evidence-based reproductive health services,' the company stated. The elimination of nationwide abortion protections has resulted in uneven access throughout the US, leading telehealth providers to address some service gaps. Various state legislators have proposed legislation to designate mifepristone as a restricted drug and reduce telehealth availability. According to tracking by the Guttmacher Institute, a nonprofit research organization, lawmakers introduced 32 proposals to prohibit medication abortion and 38 to restrict access during the 2025 legislative period, representing the highest number since the overturn of Roe v. Wade. Data from the Society of Family Planning, an abortion rights organization, shows most abortions in America still occur either at medical facilities or through prescriptions from doctors filled at pharmacies like CVS or Walgreens. A smaller percentage involves prescriptions delivered by mail. 4 Anti-abortion demonstrators rally outside the US Supreme Court in March of last year as debate over medication abortion continues. AP Following increased state-level abortion restrictions, more women have pursued online mifepristone prescriptions for mail delivery. These shipments now represent approximately 25% of American abortions, up from 5 percent before the 2022 Supreme Court decision that ended federal abortion protections. Eight states permitting abortion have enacted protective legislation shielding doctors from prosecution when prescribing mifepristone to patients in states with abortion prohibitions. Ushma Upadhyay, a reproductive sciences professor at the University of California, San Francisco, explained that CVS, Walgreens and similar pharmacies don't provide the medication in states prohibiting abortion, creating difficulties for women seeking the procedure. Telehealth providers can utilize protective laws to ship pills directly to states with restrictions, she noted. This has generated legal disputes. A Texas resident filed a lawsuit in July against a California physician for prescribing abortion medication to his girlfriend. A New York doctor faces criminal charges in Louisiana and a lawsuit from Texas's Attorney General for shipping pills to patients in those states.
Medscape
2 hours ago
- Medscape
FDA Panel Seeks More Data on Filler Use in Decolletage Area
A FDA advisory panel recommended that manufacturers of dermal fillers collect more information on use in the decolletage area of the body and said that some patients might be at higher risk of complications from injections because of the proximity to breast tissue. The FDA has not approved dermal fillers for use in the decolletage — a body area that advisory panel members said was not well-defined. It is generally considered the triangular area that runs from the neck and clavicle area to in between the breasts. Agency officials and committee members noted that fillers are increasingly being used off-label to improve skin texture, crepiness, skin thickness, fine lines, and wrinkles in the decolletage. The most common fillers used in the neck and decolletage are made up of hyaluronic acid (HA), calcium hydroxylapatite (CaHA), or poly-L-lactic acid (PLLA), according to the American Academy of Dermatology Association (AADA). At a meeting on August 13, the FDA's General and Plastic Surgery Devices Panel was asked to review safety concerns, in anticipation that manufacturers will soon seek FDA approval of fillers for use in the decolletage area and need guidance on trial design and post-marketing studies. The agency raised the possibility that fillers could migrate from the injection site or form nodules and/or granulomas and interfere with mammograms, cause false positive readings on breast imaging or clinical exams, or impact breast feeding and lymphatic drainage. The committee — made up of dermatologists, plastic surgeons, oncologists, and radiologists — did not formally vote. The panel members agreed that patients who are breastfeeding or pregnant should be excluded from receiving injections because of the unknowns. Individuals with darker skin types or known wound-healing issues — both of whom might easily form keloids or nodules — or those with a history of radiotherapy, lymphoma, or other blood cancers were also seen as potentially higher risk populations, said panel chairman Hobart Harris, MD, MPH, the J. Engelbert Dunphy endowed chair in surgery at the University of California, San Francisco. Sandra R. Shuffett, MD, a breast imaging specialist in Lexington, Kentucky, and temporary panel member, said she was concerned that fillers could obscure tumors on breast imaging tests. 'My focus is to find a cancer as small as possible,' she said, adding that an unseen tumor could quickly grow larger, necessitating more serious treatment. The FDA has not received reports of problems with breast feeding or imaging but a post-approval study of Radiesse (CaHA) found that it obscured bone visualization. There have also been reports of lymph node enlargement near dermal filler injection sites. FDA reviewer Sung Yoon, MD, said at the meeting that the agency is 'not aware of specific reports where breast cancer was missed' because of dermal filler but noted that off-label use might mean less reporting of adverse events. Yoon told the panel that an analysis of the FDA's Medical Device Reporting database found 20 reports of serious injury events associated with the use of dermal fillers in the decolletage area since 2007 out of 186 reports related to unapproved upper body use. It is not possible to know how many decolletage procedures have been performed, she said. The FDA reported that an estimated 6.2 million dermal filler procedures were performed in 2024. Radiesse manufacturer, Merz Aesthetics, told the panel that, between 2018 and 2025, it received 44 reports of potential adverse events in the decolletage area, with none reporting migration of material or radiological interference. Radiesse is approved for decolletage wrinkles in the European Union and Canada. Social media may be fueling more use of fillers in the decolletage, especially among those taking GLP-1 receptor agonists for weight loss who are seeking 'to improve the skin rippling in the chest,' said Karol A. Gutowski, MD, a Chicago-based plastic surgeon who spoke to the committee. Representatives from dermatology and plastic surgery organizations said they had crafted guidelines for safe use of fillers in the decolletage but warned that filler use was often unregulated. 'Filler adverse events are likely under reported, and they're increasing in frequency as the popularity of injectable fillers increases,' said M. Laurin Council, MD, director of dermatologic surgery at Washington University School of Medicine, St. Louis, who spoke to the panel on behalf of the American Society for Dermatologic Surgery. Many panelists suggested women undergo baseline breast imaging before receiving filler in the decolletage area and collecting more data — such as on the volume of filler used during procedures — and added that perhaps a registry should be created. But some were skeptical. 'Probably 75% of these injections are done by non-medical people,' such as attendants at medical spas or storefront wellness centers, said panelist Alan Matarasso, MD, a New York City-based plastic surgeon and past president of the American Society of Plastic Surgeons. Matarasso said that manufacturers should be responsible for tracking their products, not clinicians. 'When these things are being done in strip malls and other places, we're not going to get the data that we need, because people are not going to cooperate with this,' said Gutowski. There is no approved method of removing dermal fillers. That gave some panel members pause. But dermatologists and plastic surgeons said that HA-based fillers could be dissolved with hyaluronidase. Even so, CaHA and PLLA fillers can't be dissolved and 'must break down naturally over time,' said Natalie Curcio, MD, MPH, a Nashville-based dermatologist who spoke to the panel on behalf of the AADA. Temporary committee member Karla V. Ballman, PhD, professor of biostatistics at the Mayo Clinic College of Medicine and Science, Rochester, Minnesota, said that patients should be informed, perhaps via wording on a product label that 'at the current time, there is no approved method of removal' of a filler. The FDA has periodically examined dermal filler safety. In July 2023, the agency updated its informational webpage to describe a new risk of delayed-onset inflammation near treatment sites that had been reported to occur following viral or bacterial illnesses or infections, vaccinations, or dental procedures. The webpage also lists common reactions such as bruising, redness, swelling, and pain. At the panel meeting, consumer advocate Diana Zuckerman, PhD, president of the National Center for Health Research, said that listing adverse events was not enough. 'Risks should be quantified with meaningful statistical data on the short term and long term risks,' said Zuckerman, who spoke during the open public hearing. 'FDA should require well designed and full clinical trials so that patients have the information they need to make informed decisions,' she said. 'FDA really appreciates the thoughtful discussion questions and recommendations that have been provided today, and we will certainly take all of these comments into consideration as we move forward,' said Cynthia Chang, PhD, an acting division director within the agency's Center for Devices and Radiological Health.
