Cone Dystrophy

SAN DIEGO, April 4, 2025 /PRNewswire/ -- Experts from NYU Langone's Department of Neurology present their latest clinical findings and research discoveries at the American Academy of Neurology (AAN) 2025 Annual Meeting, April 5 to 9, in San Diego. Among the topics presented: optimal blood pressure management in acute treatment in hemorrhage stroke a review of clinical outcomes following lecanemab therapy for mild cognitive impairment and mild dementia due to Alzheimer's disease an overview on the management of optic neuritis and the need for improved diagnostics a presentation on how social determinants of health early in life increase pediatric MS onset "Our faculty continue to push the boundaries of discovery in neurological science, and the presentations at this year's AAN annual meeting are another testament to that," said Steven L. Galetta, MD, the Philip K. Moskowitz, MD, Professor and Chair of Neurology in the Department of Neurology at NYU Grossman School of Medicine. "We are proud to be part of an institution that invests in pushing forward the field of neurology and uncovering new ways to improve the lives of our patients." U.S. News & World Report's "Best Hospitals" ranks NYU Langone the No. 1 hospital in the country for neurology and neurosurgery; its Department of Neurology includes 18 divisions and centers. At this year's meeting, there are nearly 70 presentations by Department of Neurology faculty—below are some of the more notable. Plenary Session: Blood Pressure Targets in ICH: Should We Go Low? Blood pressure management is a critical part of intracerebral hemorrhage (ICH) stroke management, although the optimal target is still unknown. Kara R. Melmed, MD, clinical associate professor in the Department of Neurology and the Department of Neurosurgery at NYU Grossman School of Medicine, will debate the optimal target for blood pressure management following acute ICH. The most recent ICH guidelines suggest that acute blood pressure lowering below 150 mm HG is safe, but lowering to under 130 mm HG is potentially harmful. Dr. Melmed will present the most recent studies and data to support these guidelines. She will also discuss the benefits of minimizing blood pressure variability and most important, early intervention. As neurologists say, "Time is brain," and this is as true for ICH as it is for acute ischemic stroke (AIS). Dr. Melmed says more attention needs to be given to acute ICH management. In a recent study analyzing over 8,700 stroke patients from 11 major U.S. hospitals, patients with ICH received critical treatments much later than those with AIS, despite ICH having a higher risk of death. "These delays in ICH treatment are unacceptable given the potential impact on survival," said lead author Dr. Melmed. "Our findings highlight the urgent need to improve emergency care for hemorrhagic stroke patients." Saturday, April 5, 9:55 to 10:35 am PT, San Diego Convention Center | Ballroom 20. PL1 - Controversies in Neurology Plenary Session. Platform: Early Clinical Outcomes following Lecanemab Therapy for Mild Cognitive Impairment and Mild Dementia due to Alzheimer's Disease A recent study at NYU Langone Health evaluated early patient outcomes following treatment with Leqembi (lecanemab), an FDA-approved drug for mild cognitive impairment and early-stage Alzheimer's disease. A team of five neurologists carefully reviewed each patient before starting treatment to ensure they met strict criteria, including a confirmed Alzheimer's diagnosis and a low risk of brain hemorrhage. Patients underwent MRI scans to monitor side effects. After nine months of treatment, cognitive decline was generally slow and consistent with findings from the CLARITY AD trial. The clinical dementia rating (CDR) score remained stable, with a slight increase from 0.73 at baseline to 0.85 at nine months. However, the Mini-Mental State Exam (MMSE) scores declined more noticeably, dropping from 24.13 at baseline to 21.65 (p=0.003). Some patients experienced side effects, including brain swelling in as many as four patients and small brain bleeds in as many as 11 patients, particularly among those with the ApoE4 genetic variant. Eight patients stopped treatment, but no deaths were linked to the drug. "Our findings suggest that cognitive decline remains relatively slow in the first nine months of Leqembi therapy," said lead author Philip Kuball, MD, a resident in the Department of Neurology, who will present on the work during AAN. "Establishing a review committee helped ensure a standardized approach to patient selection." Tuesday, April 8, 1:12 to 1:24 pm PT, San Diego Convention Center | 3. S23 - Innovations in Dementia Treatment. Seminar: Management of Optic Neuritis Steven L. Galetta, MD, a neuro-ophthalmologist and the Philip K. Moskowitz, MD, Professor and Chair of Neurology, Department of Neurology at NYU Grossman School of Medicine, provides an in-depth look at optic neuritis, a condition that causes sudden vision loss due to inflammation of the optic nerve. Affecting individuals between ages 20 and 50, optic neuritis is often associated with multiple sclerosis (MS) and other neurological conditions. Key symptoms include eye pain, blurred vision, and color vision impairment. Most patients (95%) recover to 20/40 acuity, but at least half experience some lasting deficits in contrast sensitivity, color vision, or visual field. MRI is essential for diagnosis, particularly to assess the risk of developing MS. Patients with brain lesions on MRI have a significantly higher likelihood of progressing to MS. New MS diagnostic criteria will add the optic nerve as a fifth topographical site to the more traditional MS sites involving the spinal cord, periventricular area, cortical/juxtacortical, and infratentorial areas. Furthermore, optic nerve involvement may be determined by clinical criteria, inter-eye differences on optical coherence tomography (e.g., at least 4 microns in the ganglion cell/inner plexiform layer, or GCIPL), inter-eye differences when evaluating the visual system from the retina to the primary visual cortex, or optic nerve changes on MRI. Current treatment of MS associated optic neuritis includes intravenous steroids, which can speed up recovery by a few weeks but do not change long-term vision outcomes. But newer therapies are needed. As Dr Galetta noted, "While most patients regain relatively good high contrast letter acuity, we still need better treatments to prevent long-term complications including low contrast letter acuity impairment. We also should separate optic neuritis associated with myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) and neuromyelitis optica (NMO) because these patients may require different regimens including a prolonged taper of oral corticosteroids typically over months." Further research is needed to determine whether treatment within the first several days of symptom onset of MOGAD and NMO related optic neuritis can improve visual outcomes for these patients. Tuesday, April 8, 1:10 to 1:30 pm PT, San Diego Convention Center | 30C-E. C146 - Neuro-ophthalmology 2: Optic Neuritis, Visual Fields, and Anisocoria. Seminar: Treatment of Pediatric Multiple Sclerosis in the Current Era Lauren B. Krupp, MD, the Nancy Glickenhaus Pier Professor of Pediatric Neuropsychiatry in the Department of Neurology at NYU Grossman School of Medicine and director of NYU Langone's Multiple Sclerosis Comprehensive Care Center, provides an overview of pediatric MS from an ecological and psychosocial perspective. She notes that environmental factors both increase the risk of developing MS as well as increase the likelihood of having MRI indicators of more severe MS. Factors associated with an increased risk include crowding with early exposure to the Epstein-Barr Virus, residence in neighborhoods high in pollution and poor air quality, exposure to secondhand smoking, neglect and abuse, mothers with increased mental health care utilization rates, and socioeconomic hardship. Dr. Krupp and colleagues noted that among 138 individuals for whom MS began before age 18, poverty indicators and growing up in low-resourced neighborhoods were associated with more severe MRI findings of MS that were not explained by delays in accessing treatment. "Nearly 90% of our brain's development happens before age 5, so environmental and socioeconomic adversities early in life can have lasting effects related to how the nervous system responds to the inflammatory processes associated with MS," said Dr. Krupp. These findings underscore the importance of early diagnosis and optimal treatment approaches in improving long-term outcomes for pediatric MS patients, including not only medications but healthy diet, regular exercise, and the vigilance of providers to monitor with digital screening clinical outcomes prevent the new MS lesions that could lead to slower cognitive processing speed. Tuesday, April 8, 7:50 to 8:35 am PT, San Diego Convention Center | 6A. C135 - Treatment of Pediatric Multiple Sclerosis in the Current Era. About NYU Langone Health NYU Langone Health is a fully integrated health system that consistently achieves the best patient outcomes through a rigorous focus on quality that has resulted in some of the lowest mortality rates in the nation. Vizient Inc. has ranked NYU Langone No. 1 out of 115 comprehensive academic medical centers across the nation for three years in a row, and U.S. News & World Report recently placed nine of its clinical specialties among the top five in the nation. NYU Langone offers a comprehensive range of medical services with one high standard of care across seven inpatient locations, its Perlmutter Cancer Center, and more than 320 outpatient locations in the New York area and Florida. With $14.2 billion in revenue this year, the system also includes two tuition-free medical schools, in Manhattan and on Long Island, and a vast research enterprise. Media Inquiries Colin DeVries Phone: 212-404-3588 [email protected] SOURCE NYU Grossman School of Medicine and NYU Langone Health WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

MENLO PARK, Calif., March 17, 2025 (GLOBE NEWSWIRE) -- PacBio (NASDAQ: PACB), a leading provider of high-quality, highly accurate sequencing platforms, today announced a newly published study in Nature Communications unveiling a powerful new method for analyzing some of the most complex regions of the human genome. Led by researchers from PacBio, GeneDx, and a global consortium of genomics experts, the study utilizes Paraphase, an informatics tool that, when paired with HiFi long-read sequencing, allows for high-precision variant detection and copy number analysis in 316 previously inaccessible segmental duplication regions, including 9 challenging medically-relevant genes. Segmental duplications (SDs) are highly similar, duplicated regions of the genome that have posed persistent challenges for genetic analysis. These regions contain hundreds of genes critical to human health—including those implicated in spinal muscular atrophy (SMN1/SMN2), congenital adrenal hyperplasia (CYP21A2), and red-green color blindness (OPN1LW/OPN1MW)—but their high sequence similarity makes accurate mapping and variant detection nearly impossible with short-read sequencing. Paraphase, combined with HiFi sequencing, overcomes these challenges by phasing haplotypes across paralogous gene families, providing a more complete and accurate view of genetic variation. This is enabled by the length and accuracy of reads from HiFi sequencing. Study Reveals Previously Inaccessible Regions of the Genome By applying Paraphase to 160 long (>10 kb) segmental duplication regions spanning 316 genes, the researchers revealed new insights into genetic variation across five ancestral populations. Among the key findings: Newly Identified De Novo Variants in SDs in Parent-Offspring Trios: Analysis of 36 trios uncovered 7 previously undetected de novo single nucleotide variants (SNVs) and 4 de novo gene conversion events, two of which were non-allelic—a level of detail not possible with traditional sequencing approaches.Copy Number Variability Across Populations: The study profiled the copy number distributions of paralog groups across populations, showing high copy number variability in many gene families in SDs. It also provided a new approach for identifying false duplications in the reference genome.Gene Conversion Drives Sequence Similarity between Genes and Paralogs: The team identified 23 paralog groups with strikingly low genetic diversity between genes and paralogs, indicating that frequent gene conversion and/or unequal crossing-over may have played a role in preserving highly similar gene copies over time. “For decades, sequencing technologies have struggled to provide reliable data on paralogous genes—some of the most medically relevant but hardest to analyze regions of the genome,” said Dr. Michael A. Eberle, Vice President of Bioinformatics at PacBio and senior author of the study. “With Paraphase and HiFi sequencing, we now have a scalable way to accurately genotype SD-encoded genes across diverse populations, filling in long-standing gaps in genomic research and improving our ability to identify disease-linked variants.” The study also highlights how Paraphase can disentangle medically important gene families that have long required specialized, multi-step assays like MLPA and Sanger sequencing. For example, in the CYP21A2/CYP21A1P region—where mutations cause congenital adrenal hyperplasia—the researchers characterized a previously overlooked duplication allele carrying both a functional CYP21A2 copy and a CYP21A2(Q319X) pseudogene copy, which could have led to misclassification in standard tests. “This study demonstrates that when we use HiFi sequencing we see a much richer and more complex picture of genetic variation,” said Dr. Xiao Chen, lead author of the study and principal scientist at PacBio. “Paraphase enables the precise resolution of genetic regions that have been largely inaccessible until now, providing new opportunities for disease research, population genetics, and potentially even clinical testing.” “Long-read genome sequencing offers the ability to detect variants that are difficult to identify using other testing methods, particularly in regions with highly similar sequence,” said Dr. Paul Kruszka, MD, FACMG, Chief Medical Officer at GeneDx. “This work may enhance variant detection, resolve complex genomic regions, and provide more answers for patients and families, so we are encouraged by the prospect of the data.” The full study, “Genome-wide profiling of highly similar paralogous genes using HiFi sequencing,” is now available in Nature Communications. About PacBio PacBio (NASDAQ: PACB) is a premier life science technology company that is designing, developing and manufacturing advanced sequencing solutions to help scientists and clinical researchers resolve genetically complex problems. Our products and technologies stem from two highly differentiated core technologies focused on accuracy, quality and completeness which include our HiFi long-read sequencing and our SBB® short-read sequencing technologies. Our products address solutions across a broad set of research applications including human germline sequencing, plant and animal sciences, infectious disease and microbiology, and oncology. For more information, please visit www.pacb.com and follow @PacBio. PacBio products are provided for Research Use Only. Not for use in diagnostic procedures. Forward Looking Statements This press release may contain “forward-looking statements” within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the U.S. Private Securities Litigation Reform Act of 1995. All statements other than statements of historical fact are forward-looking statements, including statements relating to the uses, coverage, advantages, and benefits or expected benefits of using, PacBio products or technologies, including in connection with providing a scalable way to accurately genotype SD-encoded genes across diverse populations, fill in long-standing gaps in genomic research, and improve the ability to identify disease-linked variants; enabling precise resolution of genetic regions that were previously largely inaccessible; providing new opportunities for disease research, population genetics, and potential clinical testing; potentially detecting or enhancing the detection of variants difficult to identify using other methods, resolving complex genomic regions, and providing more answers for patients and families; and other future events. You should not place undue reliance on forward-looking statements because they are subject to assumptions, risks, and uncertainties and could cause actual outcomes and results to differ materially from currently anticipated results, including, the difficulty of generating discoveries in complicated areas of biology; potential performance, quality and regulatory issues; and third-party claims alleging infringement of patents and proprietary rights or seeking to invalidate PacBio's patents or proprietary rights. Additional factors that could materially affect actual results can be found in PacBio's most recent filings with the Securities and Exchange Commission, including PacBio's most recent reports on Forms 8-K, 10-K, and 10-Q, and include those listed under the caption "Risk Factors." These forward-looking statements are based on current expectations and speak only as of the date hereof; except as required by law, PacBio disclaims any obligation to revise or update these forward-looking statements to reflect events or circumstances in the future, even if new information becomes available. Contacts Investors and Media:Todd Friedmanir@pacificbiosciences.com Media:ir@pacificbiosciences.com

6-month efficacy and safety data from ongoing Phase 1/2 trial of OPGx-LCA5 to be presentedFARMINGTON HILLS, Mich., Dec. 03, 2024 (GLOBE NEWSWIRE) -- Opus Genetics, Inc. (Nasdaq: IRD), a clinical-stage ophthalmic biotechnology company developing gene therapies for the treatment of inherited retinal diseases (IRDs) and other ophthalmic disorders, today announced that it will host a virtual key opinion leader (KOL) event on Wednesday, December 11, 2024 at 4:00 PM ET. To register, click here. The event will feature Jean Bennett, MD, PhD, and Tomas Aleman, MD, from the University of Pennsylvania, along with Christine Kay, MD, and Arshad Khanani, MD, MA, FASRS, who will discuss detailed, patient level 6-month efficacy and safety data on OPGx-LCA5, an adeno-associated virus (AAV)-based gene therapy, in Phase 1/2 development for LCA5-associated inherited retinal disease (IRD). The event will focus on the OPGx-LCA5 clinical data and will dive into the key efficacy assessments in detail. The presenters will also highlight the current unmet need with respect to LCA5-associated inherited retinal disease, next steps in the development program, and the potential of Opus Genetics’ therapeutic approach. A live question and answer session will follow the formal presentations. Jean Bennett, MD, PhD is the F.M. Kirby Emeritus Professor of Ophthalmology at the Perelman School of Medicine and continues to serve as director of the Center for Advanced Retinal and Ocular Therapeutics (CAROT) at the University of Pennsylvania. In addition to Dr. Bennett’s positions at the University of Pennsylvania, she has been an Investigator at the Center for Cellular and Molecular Therapeutics at The Children’s Hospital of Philadelphia (CHOP) for more than a decade. She also co-founded life science companies Spark Therapeutics (acquired by Roche), GenSight Biologics and Limelight Bio. Dr. Bennett received her PhD in Zoology and Cell Biology from the University of California, Berkeley, and obtained her MD from Harvard University. She also completed postdoctoral fellowships in Radiobiology and Environmental Health at the University of California, San Francisco, Human Genetics at the Yale School of Medicine and Development Genetics at the Johns Hopkins University School of Medicine. She received her BS in Biology from Yale University. Tomas Aleman, MD is the Irene Heinz-Given and John LaPorte Research Professor at the Perelman School of Medicine, University of Pennsylvania. He is an Attending Physician in the Department of Ophthalmology at the Hospital of the University of Pennsylvania. He is part of the Retinal Degeneration Center at Scheie Eye Institute and the Center for Advanced Retinal & Ocular Therapeutics (CAROT), also at the University of Pennsylvania. Dr. Aleman has extensive expertise in the study of hereditary retinal degenerations. Most of the studies in which he has been investigator have aimed to lead patients with incurable retinal degenerations into clinical trials as well as in the detailed characterization of patients with these conditions. Dr. Aleman received an M.D. with High Honors from Sup de Cien Med de la Habana and completed a fellowship at the Scheie Eye Institute. Christine N. Kay, MD is a board-certified ophthalmologist and vitreoretinal surgeon and has been conducting clinical research since 2012. Her focus and passion are inherited retinal diseases, including but not limited to Stargardt disease, Retinitis Pigmentosa, and Achromatopsia. Dr. Kay is the Director of Research at Vitreoretinal Associates in Gainesville, Florida. She and her research team aim to improve the lives of people diagnosed with these and other diseases, as well as prevent blindness. Currently, the clinical trial team is conducting over 20 different studies targeting a multitude of retinal diseases. She was previously an Assistant Professor at the University of Florida and is currently Affiliate faculty at the University of South Florida. Dr. Kay is a member of the Macula Society, Retina Society, and American Society of Retina Specialists. Arshad M. Khanani, MD, MA, FASRS is the Managing Partner, Director of Clinical Research, and Director of Fellowship at Sierra Eye Associates, as well as a Clinical Professor at the University of Nevada, Reno School of Medicine. Dr. Khanani founded the clinical research department at Sierra Eye Associates, which has since become one of the nation's leading centers for clinical research. He has served as the principal investigator in over 120 clinical trials and has been a top enroller for several Phase 1-3 trials. He is also at the forefront of several collaborative studies examining real-world outcomes for newly approved treatments. Additionally, Dr. Khanani has been the first to perform surgical procedures in various clinical trials focused on sustained drug delivery and gene therapy. His extensive body of work includes numerous publications in highly regarded journals such as The Lancet, Ophthalmology, and JAMA Ophthalmology. Dr. Khanani is a lead principal investigator for several ongoing clinical trials and contributes to national and international clinical trial steering committees and scientific advisory boards. These efforts are aimed at developing new treatment options for patients with retinal diseases. A sought-after speaker, he is frequently invited to present at major national and international meetings. In 2021, Dr. Khanani founded the Clinical Trials at the Summit meeting to foster discussion on clinical trial design and data. Dr. Khanani is a distinguished member of both the Macula Society and the Retina Society, and he has earned various honors throughout his career. In 2019, he was named Healthcare Heroes Physician of the Year by Nevada Business Magazine for his dedication to ophthalmology. In 2023, he was chosen to deliver the prestigious Ernst Bodenheimer Memorial Lecture at the Wilmer Eye Institute, Johns Hopkins University. Dr. Khanani was also the recipient of the American Society of Retina Specialists (ASRS) Presidents’ Young Investigator Award in 2021 and the ASRS Presidential Award in 2024. About OPGx-LCA5 OPGx-LCA5 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5-associated inherited retinal disease is an early-onset severe inherited retinal dystrophy. Studies in patients with this mutation have reported evidence for the dissociation of retinal architecture and visual function in this disease, suggesting an opportunity for therapeutic intervention through gene augmentation. OPGx-LCA5 uses an adeno-associated virus 8 (AAV8) vector to precisely deliver a functional LCA5 gene to the outer retina. OPGx-LCA5 is currently being evaluated in a Phase 1/2 clinical trial at the University of Pennsylvania designed to evaluate its safety and preliminary efficacy in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. New 6-month efficacy and safety data on OPGx-LCA5 for treatment of LCA5-associated inherited retinal disease will be presented at a virtual KOL event on December 11, 2024. About Opus Genetics Opus Genetics is a clinical-stage ophthalmic biotechnology company developing gene therapies to treat patients with inherited retinal diseases (IRDs) and other treatments for ophthalmic disorders. The pipeline includes adeno-associated virus (AAV)-based gene therapies that address mutations in genes that cause different forms of bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa. The company’s most advanced gene therapy program is designed to address mutations in the LCA5 gene, which encodes the lebercilin protein and is currently being evaluated in a Phase 1/2 open-label, dose-escalation trial. BEST1 gene therapy is designed to address mutations in the BEST1 gene, which is associated with retinal degeneration; a Phase 1/2 study is expected to be initiated in 2025. The pipeline also includes Phentolamine Ophthalmic Solution 0.75%, a non-selective alpha-1 and alpha-2 adrenergic antagonist to reduce pupil size, and APX3330, a novel small-molecule inhibitor of Ref-1 to slow the progression of non-proliferative diabetic retinopathy. Phentolamine Ophthalmic Solution 0.75% is currently being evaluated in Phase 3 trials for presbyopia and dim (mesopic) light vision disturbances. For more information, please visit www.opusgtx.com. Forward Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements concerning expectations regarding data from and future enrollment for our clinical trials and the effectiveness of our pipeline of indications and gene therapies. These forward-looking statements relate to us, our business prospects and our results of operations and are subject to certain risks and uncertainties posed by many factors and events that could cause our actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the heading “Risk Factors” included in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 and in our other filings with the U.S. Securities and Exchange Commission (the “SEC”). Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this press release. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “aim,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise. These forward-looking statements are based upon our current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including, without limitation: Our ability to successfully integrate the business of former Opus Genetics Inc. and manage our expanded combined product pipeline;Our ability to develop and obtain regulatory approval for newly acquired gene therapies to treat inherited retinal diseases;Our ability to obtain and maintain orphan drug designation or rare pediatric disease designation for our current and future product candidates;The success and timing of regulatory submissions and pre-clinical and clinical trials, including enrollment and data readouts;Regulatory requirements or developments;Changes to or unanticipated events in connection with clinical trial designs and regulatory pathways;Delays or difficulties in the enrollment of patients in clinical trials;Substantial competition;Rapid technological change;Our development of sales and marketing infrastructure;Future revenue losses and profitability;Changes in capital resource requirements;Risks related to our inability to obtain sufficient additional capital to continue to advance our product candidates and our preclinical programs;Domestic and worldwide legislative, regulatory, political and economic developments;Our dependency on key personnel;Changes in market opportunities and acceptance;Reliance on third parties to conduct our clinical trials and supply and manufacture drug supplies;Future, potential product liability and securities litigation;System failures, unplanned events, or cyber incidents;The substantial number of shares subject to potential issuance associated with our equity line of credit arrangement;Risks that our licensing or partnership arrangements may not facilitate the commercialization or market acceptance of our product candidates;Future fluctuations in the market price of our common stock;The success and timing of commercialization of any of our product candidates;Obtaining and maintaining our intellectual property rights; andThe success of mergers and acquisitions. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive. Readers are urged to carefully review and consider the various disclosures made by us in this report and in our other reports filed with the SEC that advise interested parties of the risks and factors that may affect our business. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Contacts CorporateInvestor RelationsNirav JhaveriCFOir@opusgtx.comCorey Davis, Ph.D.LifeSci Advisorscdavis@lifesciadvisors.com