Erythromelalgia

Charles River will help bring NT-Z001 to the clinic by leveraging its off-the-shelf plasmid products, custom plasmid capabilities and adeno-associated virus (AAV) production. After charting multiple personalized medicine expansions in recent years, contract manufacturer Charles River Laboratories is back with another gene therapy pact—this time centered on an adeno-associated virus 9 (AAV9) candidate meant to serve as a non-opioid alternative for rare disease patients living with chronic pain. Charles River on Thursday announced a new accord with epigenetic gene therapy outfit Navega Therapeutics as part of the CDMO’s Cell and Gene Therapy Accelerator Program. The deal will grant Navega access to Charles River’s contract development and manufacturing capabilities and advisory services to produce the AAV gene therapy candidate NT-Z001 for phase 1 clinical trials, the companies said in a press release. Navega is positioning its gene therapy candidate to help treat chronic pain associated with rare diseases like small fiber neuropathy and primary erythromelalgia. The drug leverages Navega’s artificial intelligence-enabled zinc-finger gene-editing platform, the company explained. NT-Z001 specifically addresses a gain-of-function mutation in the Nav 1.7 gene, which is linked to inherited erythromelalgia, small fiber neuropathy and other chronic, debilitating pain disorders. Aside from pain tied to rare diseases, Navega figures its gene therapy could eventually address other pain indications, including neuropathic and inflammatory pain. Charles River, for its part, will help bring NT-Z001 to the clinic by leveraging its off-the-shelf plasmid products, custom plasmid capabilities and AAV production. Over the years, Charles River says it has significantly enhanced its cell and gene portfolio through a mix of acquisitions, capacity expansions and manufacturing platform launches, including eXpDNA for plasmids, nAAVigation for AAV and Lentivation for lentiviral vectors. Back in November 2022, the company debuted the expansion of its cell therapy manufacturing facility in Memphis, Tennessee. The plant reveal came just weeks after the CDMO opened a separate plasmid production facility in the U.K. Under the expansion, Charles River added nine processing suites to its 16 clean rooms at the Memphis site. The additions were designed to handle high-volume production—plus options for dual production lines—for late-stage clinical and commercial cell therapies, the company said at the time. Charles River also recently acquired a site in Rockville, Maryland, through its $350-million buyout of gene therapy CDMO Vigene Biosciences back in 2021. The site largely helps with the production of viral vectors, which have experienced supply constraints among advanced cell and gene therapies for several years. Elsewhere on the personalized medicines front, Charles River last year linked up with South Korea’s Rznomics to develop and crank out viral vectors for its partner’s experimental liver cancer gene therapy RZ-001. RZ-001 marks the first ribozyme-based RNA reprogramming approach to receive FDA authorization for evaluation in humans, the companies said last January.

DUBLIN, March 6, 2024 /PRNewswire/ -- The "Chemotherapy Induced Peripheral Neuropathy Treatment Global Market Report 2024" has been added to ResearchAndMarkets.com's offering. The chemotherapy induced peripheral neuropathy treatment market size has grown strongly in recent years, from $1.54 billion in 2023 to $1.65 billion in 2024 at a compound annual growth rate (CAGR) of 7.5%. The growth observed in the historical period can be attributed to advancements in chemotherapy, expansion in cancer treatment options, limited alternatives for treatment, a rise in cancer incidences, and progress in neurological research. The market size is expected to see strong growth in the next few years, reaching $2.25 billion in 2028 at a compound annual growth rate (CAGR) of 8.0%. The anticipated growth in the forecast period can be ascribed to the development of targeted therapies, the emergence of neuroprotective agents, the implementation of integrated care pathways, adoption of patient-centered care models, and regulatory support. Key trends in the forecast period encompass innovations in drug formulations, the integration of non-pharmacological interventions, the introduction of neuroplasticity-based therapies, enhanced support for long-term survivorship, and the incorporation of cognitive behavioral therapies. The growth trajectory of the chemotherapy-induced peripheral neuropathy treatment market is anticipated to be driven by the increasing prevalence of cancer. Cancer, characterized by the unregulated proliferation and dissemination of abnormal cells within the body, often necessitates chemotherapy treatment. However, one of the potential side effects of certain chemotherapy drugs is chemotherapy-induced peripheral neuropathy (CIPN). The rising incidence of cancer cases globally is expected to contribute significantly to the expansion of the chemotherapy-induced peripheral neuropathy treatment market. For instance, a report by the American Cancer Society in January 2023 revealed an increase in newly diagnosed cases of liver cancer in females in the United States, rising from 12,660 cases in 2022 to 13,230 cases in 2023. This escalation in cancer cases acts as a catalyst propelling the growth of the chemotherapy-induced peripheral neuropathy treatment market. The growth of the chemotherapy-induced peripheral neuropathy treatment market is being fueled by an upsurge in healthcare spending. Healthcare spending, denoting the total financial resources allocated to the healthcare sector within a specific region or country, plays a pivotal role in supporting treatments for chemotherapy-induced peripheral neuropathy (CIPN). This allocation aims to mitigate and manage the adverse neurological effects induced by certain cancer treatments, ensuring the well-being of patients undergoing and recovering from chemotherapy. For instance, data from the American Medical Association in March 2023 indicated a 2.7% increase in health spending in the United States in 2021, reaching $4.3 trillion or $12,914 per capita. This surge in healthcare expenditure serves as a driving force behind the growth of the chemotherapy-induced peripheral neuropathy treatment market moving forward. Product innovation emerges as a prominent trend in the chemotherapy-induced peripheral neuropathy treatment market, with major market players prioritizing the development of novel products to enhance efficacy and consolidate their market position. Notably, in March 2023, AlgoTx, a clinical-stage company specializing in complex pain management, announced the FDA's approval of their Investigational New Drug Application (IND) for ATX01, a Phase 2 first-in-class candidate designed for individuals with erythromelalgia. The ATX01 for the Pain of Chemotherapy (ACT) study targeting chemotherapy-induced peripheral neuropathy (CIPN) has commenced randomized patient enrollment. Both programs have secured fast-track designation from the FDA, signifying their potential significance in addressing unmet medical needs. Technological advancements, particularly the incorporation of quell technology, are a focal point for major companies in the chemotherapy-induced peripheral neuropathy treatment market as they endeavor to fortify their market standing. The primary drug categories for CIPN treatment include nerve protective therapy, anti-inflammatory therapy, neurotransmitter-based therapy, antioxidants, and others. Nerve protective therapy aims to preserve nerve health and function, crucial in managing chemotherapy-induced peripheral neuropathy for symptom relief. Treatment modalities encompass medications, therapies, and other interventions, with both branded and generic drugs available. These CIPN treatment drugs are distributed through hospital pharmacies, retail outlets, online platforms, and other channels, catering to end-users such as hospitals, research institutes, and specialty clinics. This report focuses on chemotherapy induced peripheral neuropathy treatment market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond. The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market's historic and forecast market growth by geography. Report Scope Markets Covered: By Drug Class: Nerve Protective Therapy; Anti-Inflammatory Therapy; Neurotransmitter Based Therapy; Antioxidant; Other Drug Classes By Treatment: Medication; Therapy; Other Treatments By Drug Type: Branded; Generic By Distribution Channel: Hospital Pharmacy; Retail Pharmacy; Online Pharmacies; Other Distribution Channels By End-user: Hospitals; Research Institutes; Specialty Clinics Countries: Australia; Brazil; China; France; Germany; India; Indonesia; Japan; Russia; South Korea; UK; USA; Canada; Italy; Spain Regions: Asia-Pacific; Western Europe; Eastern Europe; North America; South America; Middle East; Africa Time Series: Five years historic and ten years forecast Data: Ratios of market size and growth to related markets, GDP proportions, expenditure per capita Data Segmentation: Country and regional historic and forecast data, market share of competitors, market segments Companies Profiled Sanofi Bristol Myers Squibb Abbott Laboratories Takeda Pharmaceutical Company Amgen Inc. Asahi Kasei Pharma Corporation Toray Industries Vertex Pharmaceuticals Inc. Alexion Pharmaceuticals Inc. BioMerieux SA Jazz Pharmaceuticals Inc. WEX Pharmaceuticals Inc. Quidel Corporation Laboratories Dr. Esteve S.A. (ESTEVE) Genentech Inc. MediciNova Inc. ChromaDex Corp WinSanTor Inc. Solasia Pharma K.K. NeuroMetrix Inc. Nemus Bioscience Inc. Aphios Corporation Kineta Inc. DARA BioSciences Inc. Aptinyx Inc. Apexian Pharmaceuticals Inc. Krenitsky Pharmaceuticals Inc. For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 Logo: SOURCE Research and Markets

Patent and CA8* gene therapy technology licensed by Adolore BioTherapeutics from the University of Miami Company advancing development of rdHSV-CA8* gene therapy for the treatment of chronic osteoarthritis (OA) knee pain toward an IND DELRAY BEACH, FL / ACCESSWIRE / March 4, 2024 / Adolore BioTherapeutics ("Adolore" or the "Company"), a biotechnology company focused on developing breakthrough opioid-free gene therapy treatments for chronic pain, today announced the U.S. Patent and Trademark Office (USPTO) has issued patent No. 11,911,450 covering its innovative CA8* (*Carbonic anhydrase-8-like analgesic peptides, CA8 variants) gene therapies, using a state-of-the-art replication-defective disease-free HSV vector (rdHSV-CA8*). This issued patent and patent family, as well as the underlying CA8*-related technology were licensed in 2023 by Adolore BioTherapeutics from the University of Miami, Miami, FL. "The issuance of this patent establishes a proprietary position for Adolore's highly innovative approach for treating chronic pain," commented Roelof Rongen, CEO of Adolore. "We continue to believe in the potential of this platform and building the intellectual property protection for our CA8* gene therapies is a key first-step as we progress toward clinical testing and commercialization of these novel and potentially transformational therapies." "This important patent covers a range of CA8 variants, both as novel composition of matter as well as their novel uses, and includes technology, which holds the potential to minimize unwanted effects of avoiding biodistribution of our novel gene therapies to unaffected healthy tissues," said Dr. Roy Levitt, Inventor and Founder/Chairman of Adolore. "There is a significant and growing need to provide an alternative to the currently available and widely used oral opioid pain treatments that can adversely impact the entire body. These oral opioid-based treatments are systemic, short-acting and complicated by dependence, tolerance, abuse, addiction, death and diversion. We continue to develop our innovative approach to potentially address the significant unmet need for safe and effective locally acting pain therapies to replace opioids; and our preclinical data strongly support progression toward the clinics with continued development." There are currently very few safe, efficacious non-opioid analgesic treatments for chronic pain on the market, leaving a large and very urgent unmet medical need. Leveraging its innovative CA8* gene therapy, Adolore is currently advancing two preclinical development programs, ADB-101 for the treatment of genetically-determined life-long recurrent severe neuropathic pain due to erythromelalgia an orphan disease where there are no approved therapies; and ADB-102 for the treatment of patients with chronic pain caused by knee OA. Based on compelling data generated to date, the Company is progressing these programs toward an IND filing and first-in-human clinical studies. The Company's development program for the treatment of chronic pain in knee osteoarthritis is supported by a UG3/UH3 grant awarded to the University of Miami by NIH/NINDS HEAL program to support all formal pre-clinical GLP/GMP/GCP development work through a first-in-human study in patients expected to commence in 2026. About Carbonic Anhydrase-8 (CA8*) Gene Therapy CA8* (*Carbonic anhydrase-8 like analgesic peptides, CA8 variants) gene therapies are a novel class of neuronal calcium channel inhibitors that activate Kv7 voltage-gated potassium channels that are long-acting when administered locally. Oral therapeutics that activate Kv7 voltage-gated potassium channels demonstrated proven analgesic efficacy before they were removed from the market due to severe adverse events related to systemic exposure. CA8* gene therapy provides for versatile dosing regimens and routes of administration, including intra-articular, intra-neuronal (nerve block) and intradermal injection. This non-opioid CA8* mechanism-of-action addresses neuropathic, inflammatory, and nociceptive pain, which apply to a broad range of chronic pain indications, including osteoarthritis pain, diabetic and other forms of peripheral neuropathy, post-herpetic neuralgia, lower back pain, and cancer pain, as well as rare pain conditions such as erythromelalgia, an orphan drug disease. Using a replication-defective HSV vector enables disease-free localized delivery to the peripheral somatosensory nervous system with an excellent safety profile. HSV vectors are known for their stability and prolonged gene-expression, providing an excellent basis for long-term treatment. About Adolore BioTherapeutics, Inc. Adolore BioTherapeutics, Inc., is a biotechnology company focused on developing novel therapies for the treatment of chronic pain and other pain and nervous system conditions or disorders. Our best-in-class programs are long-acting, locally acting gene-therapies that are opioid-free Disease Modifying Anti-Pain therapies (DMAPs) for the treatment of chronic pain. The Company's two current CA8* gene therapy programs are in preclinical development for treatment of patients suffering from erythromelalgia, a life-long heritable chronic pain condition representing an orphan drug disease with no approved therapy, and chronic osteoarthritis knee pain, affecting a large number of patients that is often treated with opioids due to the lack of good alternatives, thus contributing to the ongoing opioid crisis. For more information, visit adolore.com. Forward Looking Statements To the extent this announcement contains information and statements that are not historical, they are considered forward-looking statements within the meaning of the federal securities laws. You can identify forward-looking statements by the use of the words "believe," "expect," "anticipate," "intend," "estimate," "project," "will," "should," "may," "plan," "intend," "assume" and other expressions which predict or indicate future events and trends and which do not relate to historical matters. You should not rely on forward-looking statements, because they involve known and unknown risks, uncertainties and other factors, some of which are beyond the control of the Company. These risks and uncertainties include, but are not limited to, those associated with drug development. These risks, uncertainties and other factors may cause the actual results, performance or achievements of the Company to be materially different from the anticipated future results, performance or achievements expressed or implied by the forward-looking statements. Investor Relations Contact JTC Team, LLC Jenene Thomas 833-475-8247 adolore@jtcir.com SOURCE: Adolore Biotherapeutics, Inc. View the original press release on accesswire.com