Core Insights - Harness Therapeutics has nominated HRN001 as its lead drug candidate for Huntington's disease, aiming for first-in-human studies in 2027 [1][5] - The company has formed a Clinical Advisory Board (CAB) to provide strategic guidance for the clinical development of HRN001 [6][8] - Dr. Andy Billinton, the Chief Scientific Officer, will present preclinical data at the CHDI Foundation Huntington's Disease Therapeutics Conference [7] Company Overview - Harness Therapeutics is a biotechnology company focused on addressing previously undruggable targets to transform treatments for neurodegenerative diseases [20] - The company utilizes its proprietary MISBA® platform technology to enable precise upregulation of target protein levels, which is crucial for developing disease-modifying therapies [20][21] - The company is based in Cambridge, UK, and has attracted leading life science investors, including Takeda Ventures and SV Health Investors' Dementia Discovery Fund [22] Product Development - HRN001 is an antisense oligonucleotide designed to upregulate FAN1, a key DNA repair nuclease, which has shown promise in slowing somatic expansion in Huntington's disease models [4][5] - Preclinical development of HRN001 will continue throughout 2026, with the goal of supporting clinical entry in 2027 [5] - The MISBA® platform is also being explored for other triplet repeat disorders and a broader pipeline of neurodegenerative diseases [5] Clinical Advisory Board - The CAB consists of leading experts in Huntington's disease, including Dr. Irina Antonijevic, Dr. Anne Rosser, and Dr. Jeffrey Long, among others [9][10] - The CAB will provide strategic guidance on clinical development, trial design, and translational strategy as the program advances [6][10] Scientific Rationale - Huntington's disease is caused by the expansion of CAG repeats in the huntingtin gene, with ongoing somatic expansion recognized as a key driver of disease progression [2][3] - FAN1 has emerged as a compelling target for suppressing somatic expansion, demonstrating a strong genetic association with disease onset [3][10]

Company Overview - CAMP4 is focused on using antisense oligonucleotides to selectively increase gene expression, positioning itself as a product company with a powerful platform [3]. Key Program - The flagship program of CAMP4 targets SYNGAP1-related disorders, a genetic haploinsufficiency CNS disorder with no approved treatments or disease-modifying therapies currently in development [4]. - The lead program for SYNGAP is CMP-002, which aims to be the first to enter clinical trials with a disease-modifying approach [4]. Market Potential - There are tens of thousands of patients suffering from SYNGAP around the world, indicating a significant unmet medical need [4].

Camp4 Therapeutics FY Conference Summary Company Overview - **Company**: Camp4 Therapeutics (NasdaqGM:CAMP) - **Industry**: Biotechnology, focusing on genetic disorders and CNS diseases - **Key Product**: CMP-002, an antisense oligonucleotide targeting SYNGAP1-related disorders Core Insights and Arguments 1. **Flagship Program**: Camp4 is developing CMP-002 for SYNGAP1-related disorders, a genetic haploinsufficiency CNS disorder with no approved treatments. This program aims to be the first disease-modifying therapy for SYNGAP patients, with tens of thousands affected globally [2][3][34]. 2. **Platform Technology**: The company utilizes a proprietary WRAP platform to identify and drug regulatory RNAs, allowing for selective increases in protein expression. This platform is designed to address various haploinsufficient diseases, including developmental epileptic encephalopathies (DEEs) [4][5][6]. 3. **Preclinical Data**: Strong preclinical data supports the efficacy of CMP-002, showing potential for significant disease modification. The program is currently completing GLP-tox studies, with plans to enter clinical trials later in the year [3][8][27]. 4. **Market Opportunity**: Camp4 estimates at least 20,000 patients in the U.S. and EU5 with SYNGAP1, with only about 25% diagnosed. Increased awareness and diagnostic capabilities are expected to drive this number higher [15][17]. 5. **Collaborations**: The company has formed a partnership with GSK to explore additional targets in CNS and kidney diseases, providing non-dilutive capital and expanding the application of its platform [9][60][61]. Important but Overlooked Content 1. **Patient-Centric Approach**: The presentation emphasized the impact of SYNGAP1 on patients and their families, highlighting the emotional and physical challenges faced by caregivers [10][11][12]. 2. **Regulatory Strategy**: Camp4 is engaging with regulatory agencies to facilitate a smooth transition into clinical trials, with a focus on rapid enrollment and diverse endpoints to demonstrate efficacy [28][30][50]. 3. **Financial Stability**: The company is funded through the end of 2027, with additional financing available upon achieving regulatory milestones, ensuring a stable runway for development [61]. Future Directions - **Clinical Trials**: Plans to initiate a global phase one-two study for CMP-002, focusing on various endpoints including seizures, sleep, motor function, and cognitive development [28][29][30]. - **Expansion of Pipeline**: Camp4 aims to leverage its platform to develop additional programs targeting other haploinsufficient diseases, with expectations to announce new initiatives later in the year [34]. - **Long-Term Vision**: The company aspires to become a leader in the field, drawing parallels to successful companies like Alnylam, while maintaining a strong focus on patient needs and innovative therapies [34].