Capsida Biotherapeutics

We were thrilled to present on our progress at #CGMesa24 earlier this week. We shared new preclinical data from our gene therapy program for Parkinson’s associated with GBA mutations, showing groundbreaking GCase expression and activity in non-human primates. We are also excited to continue to advance our IND enabling studies for genetic epilepsy caused by STXBP1 mutations. Both programs are well on track for IND filing in the first half of next year. #GeneTherapy #CapsidaBiotherapeutics 

We’re excited to join other #cellandgenetherapy sector leaders at the Alliance for Regenerative Medicine Cell & Gene Meeting on the Mesa. Please join our CEO @Peter Anastasiou today at 3.45pm local time in the FLW Ballroom G. Recordings of the content from the meeting will be available for registrants via the website: https://lnkd.in/eiUpMy4 #CGMesa24 #GeneTherapy

#NEWS: At #SfN2024 we presented new preclinical data on CAP-003, our investigational gene therapy for Parkinson’s disease associated with GBA mutations (PD-GBA). Highlights: 🔎 Across doses and brain regions, including the substantia nigra, levels of GCase activity were increased by up to ~250% compared to untreated animals following CAP-003 IV infusion. Increases of 30% are expected to normalize GCase activity in patients with PD-GBA. 🔎 CAP-003 demonstrated brainwide RNA expression greater than 200-fold higher than IV-delivered AAV9, with significant detargeting of liver and DRGs. We are on track to file an IND in the first half of 2025. We will also present these data as part of a corporate presentation at the Cell & Gene Meeting on the Mesa in Phoenix, Arizona, on Tuesday, October 8, 2024, at 3:45 PM local time. Read the press release for full details: https://lnkd.in/gGP84sr4 #GeneTherapy #Biotech #Healthcare #SfN24 #CGMesa24

We look forward to presenting at the Society for Neuroscience (SfN) meeting in Chicago. Join us at our poster to learn more about our unprecedented, late breaking, preclinical data from our wholly owned gene therapy program for Parkinson’s Disease associated with GBA mutations. We are on track to file our IND in 1H 2025. Poster title: LBA003.86 / LBA84 - Systemic AAV gene therapy with CNS-targeted engineered capsids achieves significant GCase activity increases in the primate brain to support the potential treatment of PD-GBA Sunday, October 6, 2024, 2-3 PM ET MCP Hall A #SfN2024

The Cell & Gene Meeting on the Mesa is right around the corner! Organized by the Alliance for Regenerative Medicine, the event will host industry experts, including our CEO Peter Anastasiou His presentation will be on October 8 at 3:45pm local time.  We’re thrilled to be part of one of our sector’s most important conferences aimed at advancing cell and gene therapy research and development. For a glimpse of our progress so far, watch below 👇 #CGMesa24  

Today, we’re joining our industry peers in NYC for the Chardan 8th Annual #GeneticMedicine Conference. In addition to giving a corporate presentation (at 4pm ET), our CEO, Peter Anastasiou, will also join a panel discussion (at 9.30am ET) about engineering the adeno-associated virus (AAV) and the promise of next gen AAV’s. To learn about our breakthrough AAV engineered capsids to develop targeted intravenously delivered gene therapies for the central nervous system, we welcome you to read our corporate presentation. Just click below 👇 #ChardonGMConf

This week, we laced up our sneakers to participate in a 5km walk in support of Move to Cure STXBP1 Disorders. The theme of the event is "Shine a Light on STXBP1," and we are proud to show our solidarity with those affected by this neurodevelopmental disorder. Speaking about the walk, our CEO Peter Anastasiou said: "At Capsida, our hearts are with the patients and families living with STXBP1 disorders. We are committed to being a steadfast partner in the journey to better understand and address the unique challenges faced by this community. Through initiatives like the Move to Cure STXBP1 walk, we strive to amplify their voices, drive progress, and ultimately, improve the quality of life for all those impacted by this condition." Let's walk together to show our solidarity, spread hope, and shine a light on STXBP1. You can also #MoveToCureSTXBP1 and #ShineALightOnSTXBP1 until Tuesday, December 3, 2024. Details at: https://lnkd.in/gbGk-Fv9 #Move2cureSTXBP1 #CapsidaTeamWalk

We are excited to join the Epilepsy Foundation 2024 Epilepsy Pipeline Conference. This event brings together leaders in the field of epilepsy treatment, therapeutic innovation, and product development to preview new therapies, explore future advancements, and foster collaboration.  Speaking at the event this morning at 9am ET, our Chief Medical Officer, Swati Tole, MD MS, will present on our preclinical intravenously delivered gene therapy with first-in-class potential to treat genetic epilepsy and developmental encephalopathy due to STXBP1 mutations, a neurodevelopmental condition estimated to affect one in 30,000 children born each year globally. Learn more about the pipeline conference at: https://lnkd.in/geAERpWd

To mark STXBP1 awareness this September, we wanted to introduce the powerful story of the Zink family. Their son Reece is living with epilepsy and developmental encephalopathy due to a mutation in the gene for STXBP1, which results in a rare neurological disorder. Justin, Kristin, and Reece have faced numerous challenges since he began to show symptoms of STXBP1 and received the diagnosis. Throughout all of it, their resilience, love, and unwavering commitment to each other are truly inspiring. We will share more details about the Zink's journey, the impact of living with genetic epilepsy and developmental encephalopathy due to STXBP1 mutations, and the importance of information, community support and hope in navigating a rare diagnosis. Together, let's raise awareness and understanding for STXBP1 during this month and beyond. #STXBP1Awareness 

We’re at the Mass General Brigham World Medical Innovation Forum in Partnership with Bank of America and looking forward to our CEO Peter Anastasiou speaking on a panel of gene therapy leaders to discuss the future of viral vector manufacturing and the delivery of genetic medicines.   Peter will talk about our progress in developing intravenously delivered gene therapies based on engineered adeno-associated virus (AAV) capsids, starting with diseases for neurological conditions with high unmet need.   We have two programs on track to enter the clinic in the first half of 2025: one for a genetic form of epilepsy and the other for a genetic form of Parkinson’s Disease.   Click the link for details of the panel: https://lnkd.in/gGePt_SZ   #WMIF2024 #Biotech #GeneTherapy