Paraplegia

Pictured: diverse group over colorful abstract background/Taylor Tieden for BioSpace Rare diseases currently afflict 300 million people worldwide and 30 million people in the U.S. alone. However, 95% of these diseases lacked an FDA-approved treatment as of January 2023. One reason, industry leaders say, is cost. A 2019 study estimated that orphan drug trials cost researchers $166 million per approved drug. Prices for drugs that hit the market in 2023 were 35% higher than those commercialized in 2022, a change that a Reuters analysis attributed to rare disease therapies that aren’t “big sellers” because of their limited target population. Pharmaceutical companies maintain that restricting drug prices will harm innovation, especially for rare diseases, but health economists argue it is possible to both lower prices and foster innovation by investing in companies and research. On Rare Disease Day, BioSpace looks at some of these investments, as well as the status of the treatment pipeline and what is required to bring more treatments to the market. Industry and Government Investment There has been considerable recent investment in rare disease treatments—from small molecule drugs to antibodies and cell and gene therapies. In July 2023, Biogen acquired Reata Pharmaceuticals and its small molecule Friedreich’s ataxia drug Skyclarys for $7.3 billion. And according to the Alliance for Regenerative Medicine (ARM), around $11.7 billion was invested in cell and gene therapies worldwide in 2023, and five gene therapies were approved for rare disorders last year. Other industry players include Chiesi Ventures, which specifically invests in companies focused on rare diseases, and Deerfield Management, which in 2023, partnered with Invitae to develop genetic treatments for rare diseases. Many early-stage investments in research and development come from the U.S. government. The Orphan Drug Act, passed by Congress in 1983, incentivizes companies to develop treatments for rare diseases using tax credits and grants. State governments can play an important role as well. The California Institute for Regenerative Medicine (CIRM) funds research, clinical trials and drug development for a variety of rare conditions such as ADA-SCID, a very rare condition where patients lack virtually all immune protection from infectious bacteria, viruses and fungi. CIRM is a member of the Bespoke Gene Therapy Consortium (BGTC), a public-private partnership including the NIH, FDA, patient advocacy groups (PAG), venture funds and pharmaceutical companies. The consortium aims to streamline the regulatory process for adeno-associated virus (AAV) vector–based therapies and has acquired $97 million in commitments since it launched in 2021. CIRM’s involvement has allowed the BGTC to address more rare diseases than it had originally planned, said P.J. Brooks, consortium co-chair and deputy director of the Division of Rare Diseases Research Innovation at the NIH’s National Center for Advancing Translational Sciences. “[CIRM has] really stepped up and we’ve been able to develop a partnership with them that I think is quite novel because they are also a major funder of research,” Brooks told BioSpace. Earlier this month, CIRM granted $4 million to Elpida Therapeutics, another BGTC member, to develop an AAV therapy for Charcot-Marie-Tooth disease type 4J, a rare, progressive neurologic disorder involving muscle weakness. CMT4J is one of eight rare diseases the consortium is prioritizing for clinical trials. Elpida is “optimistic” it will be able to launch a clinical trial within the next year, CEO Terry Pirovolakis told BioSpace in an email. The company has registered a clinical trial to study the natural history of CMT4J. Patient Groups Drive R&D, Investment Patient advocate groups (PAG) also play a key role in supporting research for rare diseases. In addition to being experts on their conditions, they may also raise funds for studies and trials. In addition to reviewing research proposals and helping partners find patients for clinical trials, the National Organization for Rare Diseases (NORD) and its member organizations help raise funds for researchers, Pamela Gavin, the organization’s chief operating officer, recently told BioSpace. PAGs helped bring the first targeted therapies for progeria and Friedreich’s ataxia to the market. Pirovolakis founded Elpida after his son was diagnosed with spastic paraplegia 50, a progressive neurological disorder that is also part of the BGTC portfolio. The company’s SPG50 drug Melpida is currently in clinical trials. “Since treating my son, between CureSPG50 and Elpida Therapeutics, we've been able to treat three more children,” Pirovolakis noted. It is “undeniably a challenging period” for rare disease drug developers, he added, especially for those targeting conditions that affect children. Although biotech investment in rare diseases increased between 2020 and 2021, investment decreased overall in 2022. Additionally, while children make up half of rare disease patients in the U.S., only a third of orphan drug indications approved between 2010 and 2018 targeted pediatric patients. Brooks estimated that in the future, “entities that are different from what we think of as pharmaceutical companies” will be more involved in the commercialization of these drugs. He said novel business models that take advantage of the “platform capacity” of gene editing and gene therapy, for example, could be what ultimately brings forward drugs for “very, very rare diseases.” A Healthy Pipeline Several biopharma companies are on the case. In 2021, the Rare Disease Company Coalition launched to advocate for policies that will help bring more treatments to market. There are more than 700 rare disease treatments in the pipeline, according to the Pharmaceutical Research and Manufacturers of America (PhRMA). Large companies such as Takeda, Sanofi and Genethon are among the leaders, with each company possessing at least 10 investigational drugs. And there were 1,329 ongoing cell and gene therapy trials involving rare diseases as of December 2023, according to data collected by ARM, with about 44% of those trials in Phase I. The same data indicate that later-stage trials are less likely to be sponsored by non-industry entities. Brooks noted that one of the challenges in making rare disease treatments more accessible is actually getting them to market. “There’s a big difference between [research] and then actually filing the license that will allow you to sell the drug,” he said. “There’s a whole . . . step in the regulatory process there that is a much bigger step than I was aware of, and taking that next step takes a lot of money.” Pirovolakis noted that while there has been an increase in families pursuing treatments, there has been a decrease in companies focusing on these diseases and a greater number of programs being discontinued. “Currently, rare diseases affecting pediatric patients are severely underfunded, including in the realm of biotech investment,” he said. “To drive progress in treating these diseases, essential support from governments, philanthropists and the biotech investment community is urgently needed.” Nadia Bey is a freelance reporter in North Carolina. Her work and contact information can be found at nadiabey.com.

Scientists have designed the first synthetic protein-based motor which harnesses biological reactions to fuel and propel itself. 'Imagine if a Roomba could be powered only by the dirt it picks up,' says one of the authors of the study. The motor uses the digestive enzyme trypsin to cut the peptides and convert them into the energy it needs to propel itself. An SFU-led collaboration has designed the first synthetic protein-based motor which harnesses biological reactions to fuel and propel itself. "Imagine if a Roomba could be powered only by the dirt it picks up," says SFU Physics professor Nancy Forde, one of the authors of the study. The team's paper, led by SFU Physics PhD graduate Chapin Korosec and published today in Nature Communications, describes a protein-based molecular motor called "The Lawnmower," which has been designed to cut a lawn of peptide "grass." The motor uses the digestive enzyme trypsin to cut the peptides and convert them into the energy it needs to propel itself. The researchers at SFU and in Lund, Sweden demonstrated that the Lawnmower is capable of self-guided motion and can be directed in specific directions using a specially designed track, an important step towards their implementation in a variety of settings. The team's findings build on decades of research on the role and function of molecular motors in organisms. As the researchers explain, all living systems, from humans to plants to bacteria, are kept alive by protein-based molecular motors. These motors convert chemical energy from one form into another to do useful work such as facilitating cell division, delivering cargo, swimming towards food or light, and maintaining healthy tissues. The Lawnmower is the first artificial motor device created with proteins from nature. As Forde explains, these experiments help researchers test our understanding of how molecular motors work in nature. "If the rules that we've learned from studying nature's molecular motors are correct and sufficient, then we should be able to build motors out of different protein parts and have them work in expected ways," she says. In the future molecular motors may have important applications in medicine and biocomputing. In the human body, motor proteins are especially important for transporting cargo within neurons. Knowing how these molecular machines work may be key to understanding and treating motoneuron diseases such as multiple sclerosis and spastic paraplegia. Molecular machines designed to mimic biological processes may also help healthcare providers deliver more targeted treatment for diseases. "Influenza is thought to work as a molecular motor to infiltrate the area around cells in order to infect them," Forde says. "Maybe synthetic motors could use the same approach, but rather than infecting cells, they could be engineered to deliver drug payloads to specifically target diseased cells." "We are inspired by the Nobel-prize-winning physicist, Richard Feynman, who famously wrote 'What I cannot create, I do not understand.' Our team's work aims to test our understanding of the fundamental operational principles of molecular machines by trying to create them from scratch."

NEW YORK, Jan. 15, 2024 /PRNewswire/ -- Medical Exoskeleton Market Insights - Continue Reading Technavio has announced its latest market research report titled Global Medical Exoskeleton Market 2023-2027 Vendors: 15+, Including Bionik Laboratories Corp., B-Temia, Cyberdyne Inc., DIH Group, Ekso Bionics Holdings Inc., Exoatlet Global SA, Focal Meditech, GOGOA Mobility Robots SL, Honda Motor Co. Ltd, Marsi Bionics SL, Myomo Inc., Ottobock SE and Co. KGaA, P and S Mechanics Co. Ltd., Parker Hannifin Corp., Rehab-Robotics Co. Ltd., ReWalk Robotics Ltd., Rex Bionics Ltd., Sarcos Corp., Wandercraft, Wearable Robotics Srl, among others Coverage: Parent market analysis; key drivers, major trends, and challenges; customer and vendor landscape; vendor product insights and recent developments; key vendors; and market positioning of vendors Segments: Technology (Powered exoskeletons and Passive exoskeletons), Mobility Type (Mobile exoskeleton and Stationary exoskeleton), and Geography (North America, Europe, Asia, and Rest of World (ROW)) To understand more about the information services market, request a Free sample report The medical exoskeleton market size is estimated to grow by USD 974.84 million from 2022 to 2027 at a CAGR of 36.43% according to Technavio. Medical Exoskeleton Market – Market Dynamics Major Drivers - Increasing usage of medical exoskeletons in rehabilitation is a key factor driving market growth. By increasing the standing and walking time of patients with muscular impairment, mechanical exoskeletons are used in addition to restoration of movement but also for improved exercise. In patients with paraplegia and paralysis, it is used mainly as a rehabilitation device to help them stand up when they lose movement due to reduced function of their brains resulting from the loss of erythropoietic functions or an increased risk for osteoporosis, pressure ulcers, and blood clots. Segmentation Analysis The Mobile Exoskeleton segment is significant during the forecast period. Mobile exoskeletons, specialized for homecare, assist spinal cord injury patients by enabling standing, walking, and balance support. Utilizing advanced sensors and controllers, they offer tailored assistance but pose higher complexity and costs compared to stationary exoskeletons. Drivers, and challenges have an impact on market dynamics and can impact businesses. Find some insights from a free sample report!The medical exoskeleton market report provides critical information and factual data, with a qualitative and quantitative study of the market based on market drivers and limitations as well as future prospects. Powered exoskeletons are revolutionizing rehabilitation robotics, aiding in SCI and stroke rehabilitation through lower and upper body exoskeletons. These devices facilitate gait training and support for musculoskeletal disorders, merging bionic technology with neurorehabilitation. Prosthetics integration and wearable sensors enhance functionality, while orthotics support physical therapy across demographics, from the geriatric population to pediatric needs. Customizable interfaces, powered by AI and machine learning, adapt to individual biomechanics, benefiting from advances in battery technology and 3D printing. Adherence to FDA regulations and inclusion in healthcare insurance coverage are crucial for wider adoption. Tele-rehabilitation expands access, while R&D drives patient mobility analysis, benefiting military, veteran, and civilian applications. Assistive technology's growth, intertwined with HCI, raises ethical considerations. Market trends forecast significant growth in the medical exoskeleton sector, propelled by these technological advancements. Why Buy? Add credibility to strategy Analyzes competitor's offerings Get a holistic view of the market Grow your profit margin with Technavio- Buy the Report What are the key data covered in this Medical Exoskeleton Market report? CAGR of the market during the forecast period Detailed information on factors that will drive the growth of the Medical Exoskeleton Market between 2023 and 2027 Precise estimation of the size of the Medical Exoskeleton Market size and its contribution to the market in focus on the parent market Accurate predictions about upcoming trends and changes in consumer behavior Growth of the Medical Exoskeleton Market industry across North America, Europe, Asia, and Rest of World (ROW) A thorough analysis of the market's competitive landscape and detailed information about vendors Comprehensive analysis of factors that will challenge the growth of Medical Exoskeleton Market vendors Gain instant access to 17,000+ market research reports. Technavio's SUBSCRIPTION platform Related Reports: The global exoskeleton market size is estimated to grow by USD 1,851.23 million at a CAGR of 35.98% between 2022 and 2027. The Wearable Robots and Exoskeletons Market size is estimated to grow at a CAGR of 33.11% between 2022 and 2027 and the size of the market is forecast to increase by USD 4,718.14 million. ToC: Executive Summary Market Landscape Market Sizing Historic Market Sizes Five Forces Analysis Market Segmentation by MobilityType Market Segmentation by Technology Market Segmentation by Geography Customer Landscape Geographic Landscape Drivers, Challenges, & Trends Company Landscape Company Analysis Appendix About US Technavio is a leading global technology research and advisory company. Their research and analysis focus on emerging market trends and provide actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavio's report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavio's comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios. Contact Technavio Research Jesse Maida Media & Marketing Executive US: +1 844 364 1100 UK: +44 203 893 3200 Email: [email protected] Website: SOURCE Technavio