Rinderpest

Basel, Switzerland, June 22, 2023 --In recognition of their pioneering work on restoring vision to blind patients using optogenetic therapy, Botond Roska and José-Alain Sahel were today awarded the International Prize for Translational Neuroscience by the Gertrud Reemtsma Foundation, managed by the Max Planck Society. The €60,000 annual prize rewards biomedical scientists and clinicians who make exceptional contributions toward the understanding of neurobiology and neurological diseases. The prize was awarded on June 22, 2023, in Hamburg, Germany. “It is with great appreciation that we receive this positive recognition from the neuroscience community,” said Roska, Director of the Institute of Molecular and Clinical Ophthalmology Basel (IOB). “For us, to be listed among the prior award recipients --all of them are major names in the field-- is a great honor.” Optogenetics is a way of creating light-sensitive cells using genes derived from algae. Roska, who has been Professor at the Faculty of Medicine since 2010 and Professor at the Faculty of Natural Sciences of the University of Basel since 2019, was one of the first to apply this technique to restore vision in animal models. After his investigations suggested promising opportunities for treating degenerative retinal diseases, Roska teamed with Sahel, who is currently chair and Distinguished Professor of the Department of Ophthalmology at the University of Pittsburgh School of Medicine and director of the UPMC Vision Institute and also the chair of the Scientific Advisory Board of IOB. In an unprecedented feat, they used optogenetic methods to partially restore vision in patients blinded by retinitis pigmentosa (RP). This is the first demonstration of optogenetics in humans and a milestone in the treatment of blinding conditions that affect millions of people worldwide. The two scientists met in 2001 while Roska was studying for a Ph.D. in cell and molecular biology in Berkeley, US. He had come to Strasbourg, France, to spend a month at Louis Pasteur University, where Sahel was then a laboratory director. Late one night, Roska succeeded in recording the activity of some retinal cells, and his boisterous reaction prompted Sahel --who was upstairs and still working-- to investigate the commotion. “Suddenly, Dr. Sahel was there at the door, asking ‘What is going on here?’” Roska says. “So, I showed him my recordings, and we spent hours discussing the research.” Thus, began a long and complementary collaboration. A basic scientist (who also has an MD), Roska directs lab studies with optogenetic therapies, while Sahel --an accomplished clinician and an expert in basic and translational research-- spearheads development and access to patients for clinical trials. By equipping diseased photoreceptors in the eye with light-sensing proteins, the pair hopes to reactivate the cells and restore their functionality. “When we started, people thought we were crazy to put algae proteins into the eyes of blind patients,” Roska says. But to Roska, retinal diseases were low-hanging fruit for optogenetics. The methods provide a mutation-independent approach for restoring visual function, and unlike the rest of the brain --which is covered by bone-- the eyes provide easy access to an optical system. Moreover, if the gene therapy treatments don’t work, “the patients are already blind, so you haven’t harmed them further,” Roska says. In their preliminary research, Roska and Sahel successfully resensitized photoreceptor circuits in blind lab mice, which soon exhibited behavioral changes in light versus dark environments. Then in 2021, the two scientists and their co-authors from the Paris Vision Institute, founded by Sahel, reported stunning results from an early-stage clinical trial with blind RP patients. Though the trial was interrupted by the COVID pandemic, one patient did complete the full study protocol. Within months, he could detect and move various objects placed on a table in front of him. The resulting paper in Nature Medicine has been downloaded more than 150,000 times. “This was the first proof-of-concept for optogenetics in any human disease,” says Roska, adding that newly-emerging data from the trial show some treated patients are achieving even better responses. “After many years of working together, winning this award comes as a happy surprise,” Sahel says. “Dr. Roska and I share a common enthusiasm and keen interest in science, and our collaboration continually produces new ideas. Optogenetics has a huge future in ophthalmology, and our long-term goal is to make it even better for patients.” About IOB At the Institute of Molecular and Clinical Ophthalmology Basel (IOB), basic researchers and clinicians work hand in hand to advance the understanding of vision and its diseases, and to develop new therapies for vision loss. IOB started its operations in 2018. The institute is constituted as a foundation, granting academic freedom to its scientists. Founding partners are the University Hospital Basel, the University of Basel and Novartis. The Canton of Basel-Stadt has granted the institute substantial financial support. www.iob.ch Follow us on Social Media Twitter @IOB_chLinkedIn Institute of Molecular and Clinical Ophthalmology Basel (IOB) YouTube IOB Basel Switzerland IOB Media Contact:lavinia.brunold@iob.ch Attachment J-A. Sahel (left)_B.Roska (right)

Phase 1/2 SLO-RP trial completed target enrollment of 48 patients diagnosed with RP associated with USH Interim efficacy data anticipated by year end 2023 FORT WORTH, Texas, May 31, 2023 (GLOBE NEWSWIRE) -- Nacuity Pharmaceuticals, Inc., a clinical stage biopharmaceutical company developing treatments for retinitis pigmentosa, cataracts and other diseases caused by oxidative stress, today announced that target enrollment of 48 patients has been reached in the SLO-RP Phase 1/2 clinical trial of NPI-001 tablets in patients with retinitis pigmentosa (RP) associated with Usher syndrome (USH). “We are pleased to reach our target enrollment, an important step toward our goal to realize the potential of NPI-001 for patients affected by RP,” said Halden Conner, Chairman, CEO and Co-Founder of Nacuity Pharmaceuticals. “We extend our gratitude to the principal investigators, Usher patient advocacy organizations and clinical sites who supported our recruitment efforts, working through challenges including the early days of a global pandemic. We are especially appreciative of the patients who have made this work possible. We look forward to sharing results from an interim analysis including efficacy data by year end 2023.” The SLO-RP trial is a randomized, placebo-controlled, multicenter, double-masked, dose-escalation trial designed to assess the clinical safety, tolerability and efficacy of NPI-001 tablets versus placebo in patients with RP associated with USH. The trial enrolled 48 male and female patients, ages 18 years and older, at its four trial sites in Australia. Patients will be followed for two years in the core study to assess efficacy. The protocol has been amended to allow continued treatment beyond two years for interested participants while the trial is ongoing. To date, all eligible participants have elected to continue on treatment. “The incredible compliance and enthusiasm of participants in the trial is a promising demonstration of the potential of NPI-001 for the treatment of RP,” said Jami Kern, Ph.D., Senior Vice President and Chief Clinical Officer of Nacuity. “We hope to confirm and build upon the encouraging preclinical and Phase 1 results to generate a robust package of data supporting the promise of an oral treatment option for patients with RP, while validating the potential for broader applications in other diseases caused by oxidative stress.” For more information, visit clinicaltrials.gov (NCT04355689). About NPI-001 NPI-001 is a proprietary, GMP-grade formulation of N-acetylcysteine amide (NACA) tablets being developed to target oxidative stress associated with blinding eye diseases, such as retinitis pigmentosa. Preclinical studies indicate that oral NPI-001 protects photoreceptor cones in the eye from oxidative damage by boosting glutathione, the body’s most powerful endogenous antioxidant, to stop chemically aggressive oxygen molecules from damaging eye tissue. NPI-001 and related small molecules have been shown to be potent antioxidants in several preclinical models that may lead to additional indications. NPI-001 has been granted orphan drug designation, which provides seven years of U.S. FDA regulatory exclusivity for the product upon regulatory approval. About Nacuity PharmaceuticalsNacuity Pharmaceuticals is a clinical-stage leader in innovative treatments for oxidative stress. The company’s powerful, targeted therapies aim to stop oxidative tissue damage, a driver of blinding eye diseases and a broad spectrum of serious chronic conditions. Nacuity has three highly differentiated clinical programs ongoing in retinitis pigmentosa, cataracts and cystinosis with the potential to be first-of-a-kind therapies and gateways to wider applications. Based in Fort Worth, TX, the company has extensive managerial and scientific domain expertise as well as backing from Foundation Fighting Blindness (https://www.fightingblindness.org/) and its venture arm RD Fund (https://www.retinaldegenerationfund.org). For more information, please visit www.nacuity.com. Media Contact: Julia Clements6 Degrees PR267.626.1085jclements@6degreespr.com

CTx-PDE6b demonstrated clinically meaningful benefit in visual functions at the highest dose and exhibits a good safety profile Positive results from the study to date support preparations for a registrational trial for CTx-PDE6b in PDE6b Retinitis Pigmentosa Regulatory approval received to expand the current study to include younger patients with earlier stages of disease who are expected to benefit most from Coave’s gene therapy to preserve visual function Paris, France, May 31, 2023 - Coave Therapeutics (‘Coave’), a clinical-stage biotechnology company focused on developing life-changing gene therapies for CNS (Central Nervous System) and eye diseases, today announces the positive 12-month results from its Phase I/II trial evaluating the safety and efficacy of its innovative gene therapy, CTx-PDE6b, for retinitis pigmentosa (RP) caused by bi-allelic mutations in the PDE6B gene (PDE6b RP). These positive data support Coave’s preparations for a registrational trial with CTx-PDE6b in this indication. In addition, Coave has received regulatory approval to expand the ongoing trial to include a new cohort of six younger patients (aged 13-25 years) with earlier stages of disease to further explore the clinical benefit of CTx-PDE6b in a broader patient population. The first patient is expected to receive treatment by July 2023. To date, CTx-PDE6b has been administered to 17 patients aged 18 years and older presenting an advanced form of PDE6b RP using two ascending doses. The treatment was administered in the more affected eye while the other eye served as an untreated control. Following the 12-month study period, both doses were well tolerated (n=17). A subgroup of patients (n=6) with less advanced disease who received the higher dose showed positive efficacy results, consistently measured across all five clinical endpoints (BCVA - Best Corrected Visual Acuity, Visual Field, Microperimetry, Full Field Sensitivity Test and Mobility test) and the retinal anatomical evaluation by OCT (Optical Coherence Tomography). In this sub-group, using microperimetry, a significant favorable progression of sensitivity of the four central loci of the retina was observed in the treated eyes compared to the untreated eyes. Furthermore, the full-field stimulation test in blue light assessing rod function shows an improvement of the light perception threshold in favor of the treated eyes, which is considered clinically meaningful. These data support a positive effect of CTx-PDE6b on visual functions and provide a strong rationale to pursue the product’s clinical development with the preparation of a registrational trial. The Phase I/II study design and data will be presented at upcoming conferences. “We are highly encouraged by the safety and efficacy data observed so far in patients who have been treated with CTx-PDE6b. This therapy has shown a good safety pro promising benefit in visual functions within the treated eye after a 12-month follow-up period,” said Rodolphe Clerval, Chief Executive Officer. “These initial findings provide strong support for expanding this Phase I/II study to include patients with less advanced disease while we begin our preparations for a registrational trial with CTx-PDE6b.” “PDE6B retinitis pigmentosa is a progressive and irreversible inherited degenerative disease that leads to significant visual impairment and blindness. These initial safety and promising efficacy results are of great medical interest and could represent a significant step towards providing a specific treatment for patients with this devastating disease,” commented Professor Michel Weber, MD, PhD, Professor at Nantes University Hospital (France) and Guylène Le Meur, MD, PhD, MCU-PH, Associate Professor and Head of Ophthalmology at Nantes University Hospital. Coave is responsible for the global development of CTx-PDE6b and retains commercial rights to the product in the US, Japan, South Korea, China and other territories outside Europe. In Europe and certain other countries, CTx-PDE6b is being co-developed by Coave and Théa Open Innovation (‘TOI’) under a license and development agreement with exclusive rights granted to TOI to commercialize CTx-PDE6b in these territories. xxx About CTx-PDE6b CTx-PDE6b is a clinical stage AAV5-based gene therapy for the treatment of retinitis pigmentosa (RP) caused by bi-allelic mutations of the PDE6b gene (PDE6b RP). RP is a type of inherited retinal dystrophy that leads to blindness by midlife and is characterized by a progressive loss of photoreceptors. There are currently no approved treatments for PDE6b RP. CTx-PDE6b is designed to deliver a full-length non-mutated copy of the functional human PDE6b gene into the subretinal space where it rapidly induces robust transgene expression and synthesis of functional PDE6b protein in photoreceptive rod cells. By providing these cells with the functional missing protein, CTx-PDE6b is expected to stabilize or halt retinal degeneration in PDE6b-deficient patients. About Clinical Trial NCT03328130 CTx-PDE6b (AAV2/5-hPDE6B or HORA-PDE6b) is being evaluated in a Phase I/II, monocentric, open-label, dose-ranging clinical trial to assess its safety and efficacy in patients with PDE6b retinitis pigmentosa (RP). Seventeen patients aged 18 years and above across three cohorts and two doses have completed the 12-month study period. The study will now enroll a further cohort of six patients aged 13-25 years with earlier stage disease. About Coave Therapeutics At Coave Therapeutics, we are leading the transition of genetic medicine from rare to prevalent conditions, in neurodegenerative and eye diseases. The Company’s proprietary AAV-Ligand Conjugates platform (‘ALIGATER’) introduces chemical modifications onto AAV capsids, overcoming the limitations of current vectors on efficacy, safety, and manufacturability. With low doses and optimized routes of administration, our conjugated AAV vectors have demonstrated markedly improved transduction and biodistribution in the central nervous system and the eye across different species. Our diverse pipeline of novel genetic medicines can potentially transform the lives of people afflicted by rare and prevalent neurodegenerative and ocular diseases – including genetically and non-genetically defined indications. Headquartered in Paris, France, Coave Therapeutics is backed by leading international life sciences investors. For more information about the science, pipeline, and people, please visit and follow us on LinkedIn. CONTACTS Coave Therapeutics Rodolphe Clerval, CEO contact@coavetx.com MEDiSTRAVA Consulting Sylvie Berrebi, Eleanor Perkin, Mark Swallow PhD coavetx@medistrava.com Tel: +44 (0)203 928 6900