Affinia Therapeutics and DCM Foundation Announce Partnership to Increase Awareness About BAG3 Dilated Cardiomyopathy and Critical Need for Genetic Testing to Help Save Lives
Affinia Therapeutics ('Affinia'), an innovative gene therapy company with a pipeline of first-in-class and/or best-in-class adeno-associated virus (AAV) gene therapies for devastating cardiovascular and neurological diseases, and the DCM Foundation together with the Genetic Cardiomyopathy Awareness Consortium (GCAC), today announced they have joined forces to raise awareness about BAG3 dilated cardiomyopathy (DCM), with the goal of promoting early diagnosis and the critical need for genetic testing.
BAG3 DCM is a devastating monogenic heart disease affecting more than 70,000 patients in the U.S., Europe, and U.K. regions alone. The BAG3 gene, or Bcl2-associated athanogene 3, encodes for a protein that is critical to the normal structure and function of heart cells. Patients with BAG3 DCM have a mutation in the BAG3 gene and a deficiency in functional BAG3 protein, resulting in early onset heart failure that progresses rapidly. Despite current standard of care, almost 25% of patients require a heart transplant. According to a study published in Circulation, the journal of the American Heart Association, close to 50% of cardiomyopathy has some type of genetic basis, such as BAG3 DCM, yet only a fraction of diagnosed cardiomyopathy patients get genetic testing.
This new-found partnership between Affinia and the DCM Foundation and GCAC aims to educate about BAG3 DCM and the critical need for genetic testing, and champions the needs and voices of people living with this devastating heart disease through initiatives including:
'The DCM Foundation and GCAC are very grateful for this partnership with Affinia,' said Greg Ruf, Founder and Executive Director, the DCM Foundation. 'By getting more cardiomyopathy patients tested, we can potentially save and improve lives and help advance cardiomyopathy research and therapies. Through this collaboration, we will collectively unite our strengths and work together in the hope of making a real difference for those living with this devastating disease.'
Hideo Makimura, M.D., Ph.D., Chief Medical Officer of Affinia, commented, 'BAG3 DCM is a devastating heart disease with a known genetic cause. Unfortunately, only a fraction of patients affected with BAG3 DCM and other genetic cardiomyopathies are tested, which is putting lives at risk. We are committed to working together with the DCM Foundation and GCAC to increase disease awareness and the role genetics plays in cardiomyopathy, which we believe will ultimately lead to better outcomes for people living with BAG3 DCM.'
'Our partnership with the DCM Foundation and GCAC is an exciting milestone as we advance our lead program, AFTX-201 for BAG3 DCM, toward an Investigational New Drug submission and clinical trial initiation which are aligned with Affinia's purpose to make a lasting positive impact in the lives of people affected by devastating rare and prevalent diseases where the genetic cause is understood,' said Rick Modi, Affinia's Chief Executive Officer.
About Affinia Therapeutics
Affinia Therapeutics is pioneering a shift to a new class of rationally designed gene therapies that treat rare and prevalent diseases. Affinia Therapeutics' pipeline of first-in-class or best-in-class product candidates in cardiovascular and neurological diseases leverages its proprietary next-generation capsids, payloads, or manufacturing approaches and have shown efficacy, safety, and differentiation in relevant animal models. For more information, visit https://www.affiniatx.com.
About DCM Foundation
Founded in 2018, the DCM Foundation's mission is to provide hope and support to DCM patients and families with dilated cardiomyopathy through education, research and advocacy. Our mission is being executed through three foundational pillars: information and education, patient and family support, and understanding the need for genetic testing. In 2023, DCMF created the Genetic Cardiomyopathy Awareness Consortium, comprised of 11 patient group members, to address the extreme lack of knowledge about genetics and genetic testing in the cardiomyopathy patient and medical community. For more information, visit www.dcmfoundation.org and www.geneticcardiomyopathy.org.
View source version on businesswire.com:https://www.businesswire.com/news/home/20250603460821/en/
CONTACT: Media contacts:For Affinia
Kathy Vincent
[email protected]
310-403-8951For DCM Foundation
Buddy Butler
[email protected]
304-615-9884
KEYWORD: MASSACHUSETTS OHIO UNITED STATES NORTH AMERICA
INDUSTRY KEYWORD: RESEARCH NEUROLOGY GENETICS CARDIOLOGY PHILANTHROPY BIOTECHNOLOGY PHARMACEUTICAL HEALTH FOUNDATION SCIENCE
SOURCE: Affinia Therapeutics
Copyright Business Wire 2025.
PUB: 06/03/2025 07:45 AM/DISC: 06/03/2025 07:44 AM
http://www.businesswire.com/news/home/20250603460821/en
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Business Wire
16 hours ago
- Business Wire
OPM Announces Its 2025 Half-Year Financial Results and Provides an Update on Its Clinical Developments and Financial Position
DIJON, France--(BUSINESS WIRE)--Regulatory News: Oncodesign Precision Medicine (OPM) (ISIN: FR001400CM63; Mnemonic: ALOPM), a biopharmaceutical company specializing in precision medicine for the treatment of resistant and metastatic cancers, announces its financial results for the first half of 2025, as approved by the Board of Directors on July 31, 2025. Philippe GENNE, Chairman and CEO of Oncodesign Precision Medicine, comments: "To date, it remains impossible to raise funds on Euronext for a biotechnology company that needs to conduct clinical proof-of-concept trials. As a result, our hopes for revenue and therefore cash availability rest primarily on discussions with potential buyers of our assets. The various cost-cutting measures we have taken since the beginning of this year are enabling us to continue these discussions over time, and the last quarter will be crucial in this regard." Karine LIGNEL, Managing Director and COO of Oncodesign Precision Medicine adds: "In a very difficult financial environment, we have taken management decisions that have enabled us to significantly reduce our expenses and thus extend our cash flow horizon as much as possible. We have also set up an equity financing line to deal with market uncertainties. Nevertheless, the situation remains complicated, and we are making every effort to secure a partnership for one of our assets." Key financial data A cost reduction program was implemented in the first half of 2025: Negotiations were held with the banks and BPI that granted the loan, resulting in a six-month deferral of principal repayments starting in April 2025 Expenses were reduced through The layoff of five employees in early 2025 A 50% reduction in the compensation of non-salaried corporate officers A freeze on salaries and bonus payments for the 2024 fiscal year A reduction in spending on non-priority programs. In terms of cash flow, rather than from an accounting perspective, this results in a reduction in payroll costs of €242k in the first half of 2025 and a projected reduction of €440k in the second half of this year. The reduction in expenditure is particularly significant for the item 'subcontracting and fees,' which will fall from €2,900k in the first half of 2024 to €1,773k in the first half of 2025. R&D expenses amounted to €1.74 million in the first half of 2025, compared with €3.61 million in the same period last year, as the company focused its efforts on the clinical development of OPM-101. Reintegration, in close collaboration with Servier, of all OPM-201 data and product stocks The most significant financial item in the first half of 2025 is the reintegration, as provided for in the partnership agreement, of the stock of OPM-201 GMP material produced by Servier. As this reintegration is being carried out without financial competing, OPM has called upon an external expert to determine fair value for these stocks. This expert carried out three different valuations to arrive at this fair value. A summary of these valuations is shown in the table below: The expert concluded that the fair value of these inventories falls within a range of €13 million to €16.2 million. OPM used the lower end of this range. OPM's revenue for the first half of 2025 was €0.1 million, the same as in the first half of 2024. Other income mainly consists of subsidies amounting to €0.3 million and €0.3 million in provision reversals. OPM's financial result was (€0.26) million, compared with (€0.1) million in the same period last year, mainly due to interest on outstanding loans and financial income from cash investments. OPM recorded €0.4 million in R&D tax credits in the first half of 2025, down 52% compared to last year. The amount of R&D tax credits was reduced by the amount of public aid, the R&D expenditure base decreased, and the rules for collecting R&D tax credits changed (exclusion of technology watch patents, etc.). OPM's half-year net income thus stands at €9,846k, due to the integration of OPM-201 inventories at an estimated value of €13m. Cash position of €2.5m at June 30, 2025 As of June 30, 2025, OPM had cash reserves of €2.5 million, before receiving the Research Tax Credit, compared to €9.6 million (including the Research Tax Credit) as of June 30, 2024. The Research Tax Credit, amounting to €1.1 million, was received in July 2025. OPM has access to a maximum €5 million equity financing facility, which it has not yet used. The Company is in discussions with financial and pharmaceutical partners with the goal of signing a partnership agreement or raising funds. 2025 perspectives OPM has submitted its Phase 1b/2a protocol for the REVERT study evaluating OPM-101 in patients with advanced melanoma resistant to anti-PD1 and is awaiting a response from Swiss ethics institutions. The first patient is expected to be enrolled by the end of 2025. Work on radioligands is continuing as part of the COMETE project. Partnership prospects or transfer mainly focus on the two most advanced molecules to date (OPM-101 and OPM-201). OPM's goal is to license or transfer OPM-101 at the end of Phase 2a to a pharmaceutical partner who will complete its development and commercialization. While we do not anticipate licensing or sales before the end of this phase in 2027, we are considering the possibility of a pharmaceutical partner taking a licensing option with the payment of an exclusivity fee, giving them first refusal on the results. For OPM-201, the objective is to find a partnership similar to the one we found with Servier in 2019 or a buyer. Availability of the 2025 half-year financial report The 2025 half-year financial report will be available on the company's website, within the legal deadline, before October 31, 2025. About Oncodesign Precision Medicine (OPM) Oncodesign Precision Medicine (OPM), founded in 2022, is a biopharmaceutical company specializing in precision medicine, dedicated to discovering treatments for resistant and metastatic cancers. OPM currently has two kinase inhibitors in clinical phase: OPM-101, intended for the treatment of chronic immuno-inflammatory digestive diseases and immuno-oncology, has demonstrated a significant therapeutic margin and absence of toxicity in its phase I healthy volunteers, with the start of phase 1b/2a in Oncology scheduled for early 2025. OPM-201, initially licensed to Servier and intended for the treatment of Parkinson's disease, completed its phase I trial in healthy volunteers at the end of 2024, and was reintegrated into OPM's portfolio. Both molecules come from the Nanocyclix ® technology platform, which enables the design and selection of small, highly effective and selective macrocyclic kinase inhibitors. We now have 12,000 molecules in our library and will be using AI to accelerate the discovery of drug candidates while reducing the cost of this phase. OPM's other two technology platforms are: OncoSNIPER, for the selection of therapeutic targets using artificial intelligence, on which we have a partnership with Servier for the search for targets in pancreatic cancer, PROMETHE ® for the design and selection of radiolabeled biological molecules for systemic radiotherapy, on which we are currently discussing partnerships with vectorization manufacturers. OPM, co-founded by Philippe Genne, Jan Hoflack and Karine Lignel, is based in Dijon, at the heart of the university and hospital cluster, and employs 14 people. More info at: Forward-looking statements This document contains forward-looking statements and estimates with respect to the financial condition, results of operations, strategy, plans and future performance of the Company and the market in which it operates. Some of these statements, forecasts and estimates can be identified by the use of words such as, without limitation, "believes", "anticipates", "expects", "projects", "plans", "seeks", "estimates", "may", "will" and "continue" and other similar expressions. They include all matters that are not historical facts. Such statements, forecasts and estimates are based on various assumptions and assessments of known and unknown risks, uncertainties and other factors, which were believed to be reasonable when they were made but which may not prove to be correct. Actual events are difficult to predict and may depend on factors beyond the Company's control. Consequently, the actual results, financial conditions, performance or achievements of the Company, or industry results, may differ materially from future results, performance or achievements as expressed or implied by such statements, forecasts and estimates. Given these uncertainties, no representation is made as to the accuracy or fairness of such forward-looking statements, forecasts and estimates. Furthermore, the forward-looking statements, forecasts and estimates speak only as of the date of publication of this document. The Company disclaims any obligation to update these forward-looking statements, forecasts or estimates to reflect any change in the Company's expectations with regard thereto, or any change in events, conditions or circumstances on which these statements, forecasts or estimates are based, except as required by French law.
Yahoo
24-07-2025
- Yahoo
DATA Communications Management Corp. to Announce Second Quarter 2025 Results on August 6, 2025
BRAMPTON, Ontario, July 24, 2025--(BUSINESS WIRE)--DATA Communications Management Corp. (TSX: DCM; OTCQX: DCMDF) ("DCM" or the "Company"), a leading Canadian provider of print and digital solutions that help simplify complex marketing communications and workflow, will announce its Second Quarter 2025 results the evening of Wednesday, August 6, 2025. The Company will host a conference call and webcast on Thursday, August 7, 2025 at 9:00 a.m. EST. Richard Kellam, President and CEO of DCM, and James Lorimer, CFO, will present the results followed by a live Q&A. Register for the webcast prior to the start of the event: Microsoft Virtual Events Powered by Teams All attendees must register for the webinar prior to the call. Please complete the phone field in the form at the above link (prior to the start of the event) if you wish to dial in. The Company's full results will be posted on its Investor Relations page and on SEDAR+. A video message from Mr. Kellam will also be posted on the Company's website. About DATA Communications Management Corp. DCM is a leading Canadian tech-enabled provider of print and digital solutions that help simplify complex marketing communications and operations workflow. DCM serves over 2,500 clients including 70 of the 100 largest Canadian corporations and leading government agencies. Our core strength lies in delivering individualized services to our clients that simplify their communications, including customized printing, highly personalized marketing communications, campaign management, digital signage, and digital asset management. From omnichannel marketing campaigns to large-scale print and digital workflows, our goal is to make complex tasks surprisingly simple, allowing our clients to focus on what they do best. Additional information relating to DATA Communications Management Corp. is available on and in the disclosure documents filed by DATA Communications Management Corp. on SEDAR+ at View source version on Contacts For further information, contactMr. Richard KellamPresident and Chief Executive OfficerDATA Communications Management (905) 791-3151Mr. James E. LorimerChief Financial OfficerDATA Communications Management (905) 791-3151ir@
Business Wire
24-07-2025
- Business Wire
Rocket Pharmaceuticals Announces Strategic Corporate Reorganization and Pipeline Prioritization of Cardiovascular Programs
BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced a strategic corporate reorganization and pipeline prioritization aimed at maximizing near-term value, extending operational runway into the second quarter of 2027, and positioning the company for sustained long-term growth. The restructuring and reprioritization initiative focuses Rocket's resources on the adeno-associated virus (AAV) cardiovascular platform, comprised of clinical programs in Danon disease, PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM), and BAG3-associated dilated cardiomyopathy (BAG3-DCM), as well as submitting the completed responses to the CRL for KRESLADI™ (formerly RP-L201) for severe leukocyte adhesion deficiency-I (LAD-I). The transition reflects the company's focus on its late-stage AAV pipeline, a strategy previously announced in January 2025. The company will implement a reduction in workforce of approximately 30%, which, along with other planned cost-saving initiatives, is expected to reduce Rocket's 12-month operating expenses by nearly 25%. Rocket anticipates that its existing cash resources, excluding any potential proceeds from a Priority Review Voucher that may be granted upon FDA approval of KRESLADI™, will fund operations into the second quarter of 2027. 'We are grateful for the remarkable contributions of our entire team and the significant progress we've made in advancing genetic therapies for rare diseases,' said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. 'Our prioritization reflects a commitment to the programs with the most compelling near-term opportunities for patients and to setting a foundation for Rocket's long-term growth and success. Together, our cardiovascular programs have demonstrated strong potential for impact in areas of high unmet need in substantial patient populations.' Rocket is advancing its leadership in cardiovascular gene therapy by strategically leveraging its AAV platform to address monogenic cardiomyopathies with high unmet need. With a portfolio spanning Danon disease, PKP2-ACM, and BAG3-DCM, Rocket is uniquely positioned to develop one-time, potentially curative treatments for distinct molecular subtypes of heart failure. Collectively, these programs target the major genetically defined causes of hypertrophic, arrhythmogenic, and dilated cardiomyopathies which represent a significant portion of inherited heart disease and impact approximately more than 100,000 patients in the U.S. and EU. Based on business and strategic priorities and as part of its realignment, Rocket anticipates delays associated with the Fanconi Anemia (FA; RP-L102) and Pyruvate Kinase Deficiency (PKD; RP-L301) programs. FDA approval of RP-L102 is no longer anticipated in 2026. These adjustments reflect a disciplined approach to capital allocation and a strategic reprioritization of assets. Additional information is expected to be provided with second quarter earnings updates as the company continues to evaluate strategic options. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket's innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives. Rocket's adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage clinical program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, and an early-stage clinical program for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death. Rocket has also received IND clearance for its AAV-based gene therapy for BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. Rocket's lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. For more information about Rocket, please visit and follow us on LinkedIn, YouTube, and X. Rocket Cautionary Statement Regarding Forward-Looking Statements This press release contains forward-looking statements concerning Rocket's future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as 'could,' 'believe,' 'expect,' 'anticipate,' 'intend,' 'plan,' 'will give,' 'estimate,' 'seek,' 'will,' 'may,' 'suggest' or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket's ability to realize the intended benefits of the restructuring plan and reduction in workforce, expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket's ongoing and planned clinical trials, the expected timing and outcome of Rocket's regulatory interactions and planned submissions, including the timing and outcome of the FDA's review of the additional CMC information that Rocket will provide in response to the FDA's request, the safety, effectiveness and timing of pre-clinical studies and clinical trials, Rocket's ability to establish key collaborations and vendor relationships for its product candidates, Rocket's ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates, Rocket's ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies, Rocket's ability to transition to a commercial stage pharmaceutical company, and Rocket's expectation that its cash, cash equivalents and investments will be sufficient to fund its operations into the second quarter of 2027. There can be no assurance that the restructuring plan or the planned reduction in workforce will have the intended effect on the Company's operational results and strategic decisions, that any anticipated charges and any anticipated cost savings associated with the restructuring plan or the reduction in workforce will achieve their intended benefits. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket's competitors' activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket's ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, the integration of new executive team members and the effectiveness of the newly configured corporate leadership team, Rocket's ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket's ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled 'Risk Factors' in Rocket's Annual Report on Form 10-K for the year ended December 31, 2024, filed February 27, 2025 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
