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Genezen and Galibra Neuroscience partner to advance Gene Therapy for SSADH deficiency toward first-in-human trial. Partnership will supply material for IND-enabling non-clinical studies and a Phase 1 trial of Galibra's AAV gene therapy for a rare pediatric neurometabolic disorder, which restored survival in 100% of treated mice in preclinical studies. 14-Oct-2026 at 8:05 AM EDT, by Genezen Newswise - INDIANAPOLIS, Oct. 14, 2026 - Genezen, a gene therapy contract development and manufacturing organization (CDMO), and Galibra Neuroscience, a biotechnology company developing transformative therapies for rare neurological disorders, today announced a manufacturing partnership to advance Galibra's GAL005 program, an AAV-based gene therapy for succinic semialdehyde dehydrogenase (SSADH) deficiency. Genezen will produce material for IND-enabling toxicology and biodistribution studies and manufacture cGMP clinical material for a planned Phase 1 trial. SSADH deficiency is a rare, inherited disorder of GABA metabolism caused by mutations in the ALDH5A1 gene which encodes the SSADH enzyme. Children with the condition experience developmental delay, impaired speech, low muscle tone, behavioral challenges, and about half have seizures. There are no approved therapies that address the underlying cause of this disease. After their son Sam was diagnosed in 1998, Brad and Carolyn Hoffman founded the SSADH Association, which has raised millions of dollars for research. The SSADH Association has supported a natural history study and patient-derived stem cell lines at Boston Children's Hospital, as well as the preclinical gene therapy research of Drs. Alexander Rotenberg and Henry Lee, who went on to co-found Galibra Neuroscience. Galibra's one-time gene therapy is designed to restore a working copy of the ALDH5A1 gene using CapX, a novel capsid developed by Apertura Gene Therapy. This second-generation, intravenously (IV) administered AAV capsid is targeted to bind human transferrin receptor 1 (hTfR1) that crosses the blood-brain barrier to deliver its payload to the central nervous system. The Galibra program has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). Galibra's preclinical data are highly encouraging with normalization of brain chemistry and disease symptom reversal in SSADH knockout mice treated with their product. "When Sam was diagnosed, my wife Carolyn and I decided we were going to do something about it, and we have been searching for a solution for nearly three decades," said Brad Hoffman, Director at Galibra Neuroscience and Co-Founder of the SSADH Association. "We have always believed gene therapy was the answer, and the animal data are remarkable. This partnership brings us one step closer to the clinic and to the treatment our community has been waiting for." Genezen will support this program at its Indianapolis facility. Galibra selected Genezen following an evaluation of manufacturing partners, citing the team's AAV expertise, genuine engagement with the program, and strong recommendations from the rare disease community, as well as its potential as a long-term partner across Galibra's broader pipeline beyond rare diseases. "Brad, Carolyn, and the SSADH Association have spent nearly 30 years building the foundation for this moment, and we are honored and humbled that Galibra has trusted us to help carry it into the clinic," said Susan D'Costa, Chief Technical and Commercial Officer at Genezen. "For a community that has waited this long, manufacturing should never be the step that slows a therapy down. Our team will apply our platform AAV process and analytical methods to build a robust, well-characterized process from the start, giving Galibra a strong quality foundation for its IND and Phase 1 manufacturing. We look forward to being a long-term partner as Galibra's pipeline grows." About Genezen Genezen is a best-in-class gene therapy CDMO with over 12 years of experience and state-of-the-art viral vector manufacturing facilities. From concept to commercial scale, Genezen partners with innovators to deliver life-saving gene and cell therapies worldwide. Learn more at genezen.com or follow Genezen on LinkedIn. About Galibra Neuroscience Galibra Neuroscience, founded by Drs. Lee and Rotenberg at Boston Children's Hospital, is a biotechnology company that is developing transformative therapies for rare neurologic disorders. Through scientific innovation and close collaboration with patient communities, Galibra advances programs that address the underlying biology of devastating genetic diseases. For more information, visit www.galibraneuroscience.com. Media contact. Type of article. Section.

Genezen and InterAct Therapeutics partner to advance AAV8-based gene therapy for liver-dominant metastatic Breast Cancer. Oct 05, 2026, 08:00 ET INDIANAPOLIS, Oct. 5, 2026 /PRNewswire/ - Genezen, a gene therapy contract development and manufacturing organization (CDMO), and InterAct Therapeutics, a gene therapy innovation organization with a proprietary platform that pinpoints cancer targets and powers anti-metastasis therapies to save lives, today announced a strategic manufacturing partnership to advance the clinical development of a therapy for breast cancer liver metastases (BCLM). InterAct Therapeutics' lead program, IAT-S2, is an AAV8-based therapy targeting hepatocytes to modulate the liver microenvironment and suppress metastatic outgrowth. Metastatic disease remains a leading cause of mortality in advanced solid tumors, yet few therapies specifically target the complex interactions between tumor cells and the metastatic microenvironment. IAT-S2 emerged from research in the lab of Dr. Isaac Chan, MD, PhD of UT Southwestern Medical Center, whose team was investigating how metastatic breast cancer cells interact with healthy liver cells to establish and grow within the liver. Dr. Chan then co-founded InterAct Therapeutics to move the program forward. Rather than targeting cancer cells alone, InterAct Therapeutics' approach is to alter the biology of the metastatic environment to eliminate metastatic growth. Liver metastases are common among patients with metastatic breast cancer, with median survival of just two to three years following diagnosis. InterAct Therapeutics is developing a one-time, disease-modifying gene therapy designed to address the tumor-liver microenvironment, for which there are currently no specifically approved therapies. InterAct Therapeutics selected AAV8 as the delivery vector for IAT-S2 based in part on its ability to efficiently target hepatocytes, enabling a liver-directed approach to the treatment of metastatic disease. In preclinical studies, InterAct Therapeutics observed near elimination of the liver metastases across five models of liver metastasis in mice. Following positive FDA INTERACT feedback, InterAct Therapeutics is now preparing for IND-enabling studies with manufacturing support from Genezen's experienced viral vector team at its state-of-the-art facility in Indianapolis. Genezen will provide its platform AAV process development, scale-up, and GMP manufacturing expertise to support toxicology studies and the production of clinical material for a Phase 1 trial. The collaboration will support InterAct Therapeutics as it advances IAT-S2 toward IND submission and first-in-human clinical development. "As a physician, I am concerned when metastatic breast cancer spreads to the liver, as these are among the patients I worry about the most. As a scientist, I've dedicated my work to understanding why these metastases are so difficult to treat and finding a fundamentally different way to approach them," said Dr. Isaac Chan, MD, PhD, Chief Scientific Officer and Co-founder of InterAct Therapeutics. "This partnership is an important step toward translating a discovery that began in our lab into a potential therapy for patients. Announcing this partnership during Breast Cancer Awareness Month is especially meaningful as we move beyond awareness toward the progress patients are waiting for." "Genezen was built to help innovators translate promising science into therapies that can make a meaningful difference for patients," said Steve Favaloro, Chairman and Chief Executive Officer of Genezen. "We are proud to bring our deep AAV development and manufacturing expertise to this partnership and work alongside InterAct Therapeutics to help move this program forward and make an impact in the oncology space. Collaborations like this embody what drives our team every day: scientific excellence, urgency for patients and a solutions-driven commitment to our partners as we work together to bring the next generation of therapies to life." InterAct Therapeutics selected Genezen following an evaluation of manufacturing partners, citing the company's AAV experience, reputation, reliability and ability to support the program's critical development timeline. The partnership builds on Genezen's deep technical experience in AAV development and manufacturing, including its experience manufacturing a commercially approved AAV gene therapy, and positions the companies to scale manufacturing as IAT-S2 progresses through clinical development. About Genezen Genezen is a best-in-class gene therapy CDMO with over 12 years of experience and state-of-the-art viral vector manufacturing facilities. From concept to commercial scale, Genezen partners with innovators to deliver life-saving gene and cell therapies worldwide. Learn more at genezen.com or follow Genezen on LinkedIn. About InterAct Therapeutics InterAct Therapeutics is a biotechnology company focused on developing first-in-class therapies that target the biology of metastatic disease by modulating organ-specific microenvironments. Learn more at interacttx.com or follow InterAct on LinkedIn. SOURCE Genezen