Led · Equity · May 2025

Armatus Bio is developing next-generation vectorized RNAi therapeutics targeting genetically driven neuromuscular diseases, including facioscapulohumeral muscular dystrophy (FSHD). Its lead candidate, ARM-201, is an AAV-delivered microRNA therapy designed to silence DUX4, the toxic protein implicated in FSHD, with the aim of arresting muscle degeneration and reducing inflammation and oxidative stress. ARM-201 uses the AAV-SLB101 capsid licensed from Solid Biosciences, which showed enhanced skeletal and cardiac muscle transduction and decreased liver exposure in preclinical studies and was well tolerated with robust transduction in a Duchenne clinical trial. Armatus has completed a pre-IND meeting with the FDA and is positioning ARM-201 for IND-enabling work and regulatory submission. The company is privately held, late preclinical, based in Columbus, Ohio, and led by a team experienced in drug development and delivery. In addition to ARM-201, Armatus is building a pipeline of precision medicines aimed at other genetic neuromuscular disorders and is partnered with experts in vector biology, genomics, and neurology. Armatus Bio is advancing ARM-101, a vectorized RNAi gene therapy candidate intended to normalize PMP22 in Schwann cells for CMT type 1A. Its development studies are designed to answer critical questions about how to effectively deliver genetic medicines to the right parts of the body and to generate evidence to enable human trials. The company is collaborating with gene therapy experts to build a pipeline of novel therapeutic candidates aimed at overcoming limitations of current approaches. Armatus says the data from ARM-101 studies are intended to support progression toward human trials and potentially provide benefit to people living with CMT1A. The announcement highlights the unmet need: there are currently no FDA-approved therapies for any type of Charcot‑Marie‑Tooth disease. CMTA’s participation signals patient‑community philanthropic support for Armatus’s early development work.

Participated · Series B · Mar 2025

Epicrispr Biotechnologies develops programmable epigenetic medicines designed to durably regulate gene expression without altering DNA sequence, leveraging its proprietary GEMS platform. Its lead program, EPI-321, is an investigational epigenetic therapy for facioscapulohumeral muscular dystrophy (FSHD) delivered in a single AAV vector intended to target muscle following intravenous administration. EPI-321 is in a Phase 1/2 trial with enrollment completed and interim results showing statistically significant increases in whole-body lean muscle volume, favorable biomarker changes consistent with DUX4 suppression, and a manageable safety profile. The company is advancing additional gene-modulating programs across multiple therapeutic areas and plans to expand its manufacturing capabilities. Epicrispr is based in San Francisco and is positioning to move EPI-321 into pivotal studies as it develops its next-generation programmable epigenetic medicines.

Led · Series B · Oct 2022

Vita Therapeutics develops iPSC-based cellular therapeutics aimed at repairing and replacing defective muscle cells and creating cancer immunotherapies. Its lead programs include VTA-100 for LGMD2A (in IND-enabling studies), VTA-120 for FSHD, and VTA-300, a CAR-based cancer immunotherapy. VTA-100 and VTA-120 are designed as autologous treatments combining gene correction with iPSC technology; the company intends to pursue a dual strategy beginning with autologous cells and later moving to a universal hypoimmunogenic cell line. Vita is working with partners including PanCella, the Wyss Institute, and Johns Hopkins University to advance its programs. The company aims to file Investigational New Drug applications for VTA-100 within the next 18 months. Financially, Vita completed a $31 million Series B and has raised $66 million since inception. Vita Therapeutics is a cell engineering company that uses induced pluripotent stem cell (iPSC) technology and genetic engineering to develop autologous and universal hypoimmunogenic cellular therapies for muscular dystrophies. Its lead program, VTA-100, combines gene correction with iPSC-derived muscle stem cells and is undergoing IND-enabling studies for limb-girdle muscular dystrophy (LGMD) 2A/R1. A second program, VTA-200, is a genetically engineered hypoimmunogenic iPSC-derived treatment intended to address multiple muscular dystrophies, and the company is also developing VTA-300 (cell type undisclosed). Vita plans to complete IND-enabling studies for VTA-100, submit an IND to the FDA, manufacture cells for clinical evaluation, and recruit patients for its first trial. The company follows a dual manufacturing strategy—starting autologously before moving to a universal hypoimmunogenic cell line—and was founded in 2019 out of Johns Hopkins University and the Kennedy Krieger Institute. Vita is a Cambrian Biopharma affiliate and is based in Baltimore, Maryland. The oversubscribed $32 million Series A will fund completion of IND-enabling work, cell manufacturing, patient recruitment, and further development of VTA-200 and VTA-300.

Led · Equity · Sep 2022

Altay Therapeutics develops small-molecule drugs that target disease-causing transcription factors to treat chronic illnesses and cancers. The company uses a proprietary, integrated platform combining biochemical assays, next-generation computational biology, and chemoproteomics to discover novel inhibitors. Using that platform Altay discovered three first-in-class inhibitors, including one that targets DUX4, a key driver of facioscapulohumeral muscular dystrophy (FSHD). Altay was co-founded in 2019 by Dr. Ali Ozes and Dr. Osman Ozes and was accepted into Y Combinator. The company's stated vision is to be a leader in targeting disease-causing transcription factors and to extend patient lifespan via safer, more specific therapies. A US$3 million investment from SOLVE FSHD is intended to support identification and advancement of a DUX4 inhibitor toward clinical trials.