California Institute for Regenerative Medicine

FORT LEE, NJ, USA I March 06, 2025 I Luxa Biotechnology LLC (Luxa), a clinical-stage biotechnology company developing a novel adult retinal pigment epithelial stem cell (RPESC) therapy for dry age-related macular degeneration (AMD), today announced clinical data from its Phase 1/2a clinical trial evaluating RPESC-RPE-4W, a proprietary retinal pigment epithelium (RPE) cell therapy for patients with dry age-related macular degeneration (dry AMD). The data were presented today at the 77th Annual Wills Eye Conference in Philadelphia, Pennsylvania. Findings from six patients in the first low-dose cohort of the ongoing first-in-human trial were presented ( NCT04627428 ). Each patient received a 50,000-cell suspension of RPESC-RPE-4W implanted under the macula. Post-implant clinical outcomes were assessed over 12 months for the worse-seeing Group I and over 3 months for the better-seeing Group II. No serious adverse events (SAEs) related to the investigational therapy were observed. Best Corrected Visual Acuity (BCVA) improvements were substantial, with Group I experiencing an average gain of +21.67 ETDRS letters at 12 months and Group II showing a +3.3-letter improvement at 3 months. These gains are particularly meaningful given that even a 5- to 10-letter improvement can translate into a real-world functional benefit for patients, such as improved reading ability, better recognition of faces, and enhanced mobility. The patients with the most severe baseline vision impairment and the greatest clinical need achieved the most significant vision restoration, reinforcing the potential of this therapeutic approach to address the substantial unmet need in advanced dry AMD. Meanwhile, individuals who began with better baseline vision saw smaller, yet still clinically relevant, improvements that counteracted the natural decline expected in dry AMD progression. This reversal of disease trajectory underscores the potential for preserving vision in earlier-stage patients, possibly delaying or even preventing progression to severe visual impairment. “These findings are encouraging, as they demonstrate not only meaningful vision improvements in patients with the greatest need but also the potential to alter the trajectory of dry AMD in those with better baseline vision,” said Dr. Jeffrey Stern, Chief Medical Officer of Luxa. “The substantial BCVA gains, particularly in worse-seeing patients, highlight the promise of our approach in restoring vision, while the modest improvements in better-seeing individuals suggest a beneficial impact in slowing disease progression. This reinforces our commitment to advancing innovative treatments that address the urgent unmet needs in dry AMD.” Last month, Luxa announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) Designation for RPESC-RPE-4W in the treatment of dry AMD. This designation will further facilitate the development and regulatory progress of the novel adult stem cell therapy. “We are thrilled with these promising clinical outcomes, which mark a significant step forward in our progress towards addressing a major unmet need in dry AMD,” said Dr. Keith Dionne, CEO of Luxa. “This achievement underscores our commitment to reversing vision loss for millions of adults affected by dry AMD and geographic atrophy. We look forward to advancing our Phase 1/2a trial and evaluating higher dose cohorts.” About Dry AMD Age-related macular degeneration (AMD) is the leading cause of adult-onset blindness in the Western world, affecting approximately 20 million Americans and 200 million people globally, with over 80% of cases classified as the dry form. Dry AMD causes diminished vision accompanied by loss of central retinal pigmented epithelium (RPE) cells in individuals aged 55 and older. There are no approved vision-improving treatments for dry AMD. About Luxa Biotechnology Luxa Biotechnology is a joint venture of the Korean technology company Y2 Solution and the Neural Stem Cell Institute (NSCI) in Albany, New York. Luxa licensed RPESC-RPE-4W from NSCI and maintains a robust research program at NSCI to advance RPESC-RPE-4W as an effective, commercially viable therapy to restore the lost vision of AMD patients. Luxa acknowledges the support and critical contributions of the clinical team at the University of Michigan Kellogg Eye Center, the manufacturing team at the Cedars Sinai Biomanufacturing Center, the California Institute for Regenerative Medicine and the U.S. National Institutes of Health and the National Eye Institute. For more information, visit our website and follow us on LinkedIn . SOURCE: Luxa Biotechnology

LONDON, UK & CHICAGO, IL, USA I March 05, 2025 I Anova Enterprises, Inc. (Anova), a technology-enabled CRO dedicated to accelerating promising treatments, has announced enrollment of the first two patients in the highly anticipated study of DB107 for the treatment of brain tumors. The Phase 1/2a clinical trial is a multicenter, open-label study designed to confirm whether treatment DB107, when added to standard-of-care (SOC), provides clinical benefit to patients with newly diagnosed high-grade gliomas (HGG) when compared to historical performance. The study is funded with a California Institute for Regenerative Medicine (CIRM) grant to advance the development of DB107 in patients with newly diagnosed high-grade glioma. Led by Noriyuki Kasahara, MD, PhD, Principal Investigator, Brain Tumor Research Center (BTRC) at University of California, San Francisco (UCSF), the study is also enrolling at the University of Southern California (USC) and the University of California at San Diego UCSD, and will enroll 70 participants over the coming months. “Enrolling our first patients is an important milestone in our mission to advance treatment options for patients with high- grade glioma,” said Nicholas Butowski, MD, Neuro-Oncologist and Lead Investigator at UCSF. “We are optimistic this trial will provide valuable insights into how we can better treat patients and improve survival in newly diagnosed patients.” High- grade gliomas remain one of the most challenging cancers to treat, with limited treatment options and a high degree of complexity. Treatment for the disease has not changed in 20 years. DB107 is an investigational gene therapy being developed by Denovo Biopharma for the treatment of HGG, including glioblastoma multiforme (GBM). It comprises two components: DB107-RRV (vocimagene amiretrorepvec), a retroviral replicating vector designed to selectively infect and change cancer cells, and DB107-FC, an extended-release oral formulation of 5-fluorocytosine (5-FC), which is converted into a chemotherapeutic agent within the tumor. This combination aims to eradicate tumor cells while stimulating a robust and durable anti-cancer immune response with minimal toxicity. Chris Beardmore said, “This is revolutionary science where the treatment infects cancer cells and genetically changes them to make chemotherapy inside of the infected cells. This is known to reduce systemic toxicity commonly seen with systemic treatments. It also has a chance to infect cells that cannot be removed by surgery and other interventions to improve outcomes in newly diagnosed brain tumor patients.” The clinical trial is currently recruiting eligible patients who meet the study criteria at UCSF, USC and UCSD. Individuals interested in participating or learning more about the trial can contact the study team at info.us@anovaevidence.com . To find out more, contact info.us@anovaevidence.com . About Denovo’s RRV Platform and DB107 DB107 is an innovative approach utilizing a proprietary gene therapy platform, recombinant retroviral vector (RRV) bearing cytosine deaminase, combined with a prodrug of 5-FU (5-FC), to selectively infect and kill cancer cells while stimulating a robust and durable anti-cancer immune response against a tumor with minimal toxicity. DB107 has been tested clinically in solid tumors including recurrent high-grade GBM and colorectal cancer, most recently in a randomized 403-patient Phase 3 trial. DB107 received Orphan Drug Designation in GBM from the FDA and EMA, and Fast Track Designation from the FDA. About Anova Anova Enterprises, Inc. (Anova) is technology enabled concierge research organization committed to accelerating clinical development for start-up biopharmaceutical companies utilizing the company’s proprietary technology platform (AnovaOS™). For more information, please visit www.anovaevidence.com . About Denovo Biopharma Denovo Biopharma LLC (Denovo) is a clinical‑stage biopharmaceutical company that uses a novel biomarker discovery platform including whole genome sequencing (WGS) and artificial intelligence (AI) to find new genetic biomarkers predictive of drug efficacy, and then uses these biomarkers to execute efficient clinical trials in targeted patient populations to increase the probability of success. Denovo has eight late‑stage drugs in its pipeline addressing major unmet medical needs in oncology and central nervous system diseases; most of the pipeline are first‑in‑class drugs with global rights. Denovo recently announced a major breakthrough in treatment‑resistant depression (TRD) with its DGM4™ biomarker‑guided DB104 drug (liafensine, a potential first‑in‑class triple reuptake inhibitor for TRD). The global Phase 2b clinical trial of DB104 in DGM4‑positive patients with TRD demonstrated strikingly positive results, with highly significant improvements in symptoms of depression seen in DGM4‑positive patients. Visit www.denovobiopharma.com for additional information. About the California Institute for Regenerative Medicine (CIRM) The California Institute for Regenerative Medicine (CIRM) is a funding agency established by Californians to accelerate regenerative medicine research to deliver treatments for patients with unmet medical needs. Established in 2004 through the passage of Proposition 71, CIRM was initially funded with $3 billion from the state of California to support ongoing research, and in 2020, was funded again with another $5.5 billion through Proposition 14 to continue the Agency’s important work. CIRM has provided billions in funding to support stem cell, genetic research, and development programs in its portfolio. Through the Agency’s research, infrastructure, and education programs, CIRM aims to transform the field of regenerative medicine, stimulate economic growth, and improve the lives of diverse communities throughout the state. For more information go to cirm.ca.gov . SOURCE: Anova Enterprises

WOODBRIDGE, Conn.--( BUSINESS WIRE )--Cure Rare Disease (CRD) announced it has been awarded a $5.69 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of an antisense oligonucleotide therapy for spinocerebellar ataxia type 3 (SCA3), a neurodegenerative disorder with no current treatment. Cure Rare Disease launched its SCA3 program in 2021 to develop an antisense oligonucleotide (ASO) therapy, with initial funding support provided by Gregory Klassen, a patient living with SCA3. In 2022 and 2023, in collaboration with Leiden University Medical Center and the Cure Rare Disease team, several in vivo studies were conducted in a disease mouse model, demonstrating functional improvement. A clinical candidate was identified through non-GLP toxicology studies conducted with Charles River Labs. In 2024, a Type B pre-IND meeting with the Food and Drug Administration (FDA) provided regulatory guidance ahead of manufacturing scale-up and clinical trial design. With CIRM funding support, Cure Rare Disease will complete manufacturing scale-up, conduct IND-enabling toxicology studies and submit an investigational new drug (IND) application to initiate early clinical trials. The trials will be led by Principal Investigator Dr. Susan Perlman at UCLA. "This program is a powerful example of what can be achieved when patients, organizations, researchers and governing bodies come together with a shared mission. From Greg Klassen’s initial support to our collaborations with leading scientists and clinicians, industry partners and regulatory guidance from the FDA, the development of a therapy for SCA3 has been a truly unified effort,” said Cure Rare Disease CEO and founder Richard Horgan. “We are incredibly appreciative of CIRM’s support in helping us take this program into the clinic, proving that rare disease treatments can be brought to patients through our model of collective dedication and innovation." About SCA3 Spinocerebellar ataxia type 3 (SCA3) is a rare neuromuscular disease with a prevalence of one to five in 100,000 people. It is the most common form of spinocerebellar ataxia. The disease is caused by mutations in the ATXN3 gene, which codes for the enzyme ataxin-3. Ataxin-3 is found throughout the body and helps destroy and remove damaged proteins. SCA3 results from a mutation that causes a toxic accumulation of trinucleotide repeats. About Cure Rare Disease (CRD) Cure Rare Disease (CRD) is a nonprofit biotechnology company focused on developing genetic medicines for rare and ultra-rare diseases. CRD is committed to accelerating the development of treatments for individuals with rare diseases through a patient-centric approach to research and development. The company's programs include gene therapy and antisense oligonucleotide (ASO) treatments for a range of rare diseases. For more information, visit www.cureraredisease.org . About the California Institute for Regenerative Medicine (CIRM) The California Institute for Regenerative Medicine (CIRM) is a funding agency established by Californians to accelerate regenerative medicine research and deliver treatments for patients with unmet medical needs. Established in 2004 through the passage of Proposition 71, CIRM was initially funded with $3 billion from the state of California to support ongoing research. In 2020, Proposition 14 provided an additional $5.5 billion to continue the agency’s work. CIRM has funded billions in stem cell and genetic research, as well as development programs. Through its research, infrastructure and education programs, CIRM aims to transform regenerative medicine, stimulate economic growth and improve the lives of diverse communities throughout the state. For more information, visit www.cirm.ca.gov .