Participated · Series A · Apr 2020

ROME Therapeutics leverages a proprietary data science platform to identify functionally active repeat elements within the dark genome and translate those findings into therapeutics. Its lead program is an inhibitor of LINE-1 reverse transcriptase (RT) intended to block pathogenic inflammation in autoimmune diseases, including lupus. ROME plans to advance the LINE-1 RT inhibitor into early clinical trials, including Phase 1 studies to evaluate safety, dosing, and proof of mechanism. The company is progressing several additional first-in-class programs across autoimmune disease, cancer, and neurodegeneration informed by the same platform. ROME intends to use recent funds to advance its pipeline and further develop its platform, which also supports clinical trial design and patient selection. The company is based in Boston, Massachusetts. ROME Therapeutics has built a repeatomics data-science platform to analyze the repeatome (roughly 60% of the human genome) and identify druggable targets. The company has identified multiple repeatome-derived targets and is advancing several internal discovery programs. Its core focus is developing novel therapeutics for cancer and autoimmune diseases by leveraging insights from previously understudied repetitive genomic sequences. ROME says it is moving lead programs toward the clinic while expanding its pipeline and enhancing its repeatomics platform. The company launched in April 2020 and is based in Cambridge, Mass. Financially, ROME recently closed a Series B financing that strengthens its capitalization for near-term clinical and discovery activities. ROME Therapeutics develops novel therapies for cancer and autoimmune diseases by harnessing the repeatome, the roughly 60% of the human genome made of repetitive sequences. The company has identified several promising drug targets and launched multiple discovery programs based on insights that viral-like repeat elements are activated in times of stress and may drive disease. ROME draws on expertise in oncology, virology, immunology and machine learning, and its founding intellectual property comes from Massachusetts General Hospital and the Icahn School of Medicine at Mount Sinai. The company was incubated at GV and launched in April 2020. ROME announced $50 million in Series A funding from GV, ARCH Venture Partners and Partners Innovation Fund to advance its discovery programs. It has assembled a leadership team and scientific advisory board with deep drug discovery and translational experience to move its repeatome-based programs forward.

Led · Series A · Apr 2020

SwanBio Therapeutics is a gene therapy company advancing adeno-associated virus (AAV)-based therapies for devastating, inherited neurological conditions, with an initial focus on spinal cord-related disorders. Its lead program, SBT101, is the first clinical-stage AAV-based candidate for adrenomyeloneuropathy (AMN), designed to compensate for the disease-causing ABCD1 mutation. The company plans to dose patients in a Phase 1/2 trial of SBT101 by the end of the year; the program has an FDA-cleared IND and has received Orphan Drug and Fast Track designations. In preclinical studies, SBT101 showed dose-dependent improvement of disease markers and functional benefit in AMN mouse models and was well tolerated in non-human primates at six months. SwanBio is building platform capabilities in delivery and biodistribution, cassette design, and novel capsid development to advance a broader intrathecally delivered pipeline targeting both central and peripheral nervous systems. Financially, the company has raised significant funding to support its R&D expansion, including the recent Series B. SwanBio Therapeutics develops adeno-associated virus (AAV)-based gene therapies aimed at treating AMN and other neurological diseases. The company completed a $52m expanded Series A financing to advance its pipeline. Syncona Ltd. and Partners Innovation Fund contributed $20m as part of the additional commitment to the round. SwanBio plans to use the funds to progress understanding and development of AAV gene therapies, advance manufacturing and development capabilities, and execute a hiring plan to meet pipeline demands. Led by CEO and Director Tom Anderson, the company focuses on the development and commercialization of AAV-based gene therapies for neurological diseases with significant unmet medical need. SwanBio is based in Bala Cynwyd, Pennsylvania, and also has operations in Philadelphia, Pennsylvania and Cambridge, Massachusetts.

Participated · Series A · Apr 2020

Affinia Therapeutics engineers novel adeno-associated virus (AAV) capsids to create gene therapies that more selectively target heart and skeletal muscle while reducing liver exposure. Its lead candidate, AFTX-201, employs a proprietary cardiotropic capsid to deliver a fully human BAG3 transgene for the treatment of BAG3 dilated cardiomyopathy (DCM), a life-threatening inherited heart disease affecting more than 70,000 patients across the U.S., Europe and the U.K. Preclinical studies showed that AFTX-201 restored cardiac function in animal models where conventional capsids failed at the same dose. The company plans to submit an IND for AFTX-201 in Q4 2025 and, pending FDA acceptance, begin a Phase 1/2 UPBEAT trial in Q1 2026. Affinia’s generative-AI discovery platform is also being applied to build a broader pipeline of gene therapies for additional devastating cardiovascular and neurological indications. The newly announced $40 million Series C financing extends the company’s runway to support these clinical and pipeline milestones.

Participated · Series B · Mar 2020

Akouos develops precision gene therapies for sensorineural hearing loss using an adeno-associated viral (AAV) vector-based platform. Its lead program, AK-OTOF, targets hearing loss caused by mutations in the otoferlin (OTOF) gene. The company plans to advance AK-OTOF to first-in-human clinical studies subject to IND filing and acceptance. Proceeds from its recent financing will also support establishing in-house GMP manufacturing at new offices in the Boston Seaport and accelerating multiple pipeline programs with well-defined mechanisms and gene targets. Akouos intends to expand its teams across research, clinical development, and manufacturing. The company has strategic partnerships with Massachusetts Eye and Ear and Lonza, Inc. Akouos is a precision genetic medicine company focused on developing targeted adeno-associated viral (AAV)-based gene therapies for sensorineural hearing loss, with an initial emphasis on monogenic forms that cause profound deafness. The company leverages a proprietary AAV gene therapy platform and has strategic partnerships with Massachusetts Eye and Ear and Lonza. Proceeds from a recent $50 million Series A will support advancement of its lead program to first-in-human clinical studies and accelerate multiple pipeline programs addressing other validated gene targets. Akouos has assembled clinical and scientific leadership, expanded its board, and formed a scientific advisory board composed of world-leading experts in gene therapy and hearing loss. The company states its approach aims to deliver one-time precision therapies to treat underlying genetic causes of hearing disorders. Akouos develops gene therapies aimed at restoring and preserving hearing and balance using in silico-designed Anc-AAV viral vectors. The company entered strategic license agreements with Lonza and Massachusetts Eye and Ear to obtain exclusive rights to the Anc-AAV gene therapy platform and broad sublicensing rights. Anc-AAVs were first developed in the laboratory of Dr. Luk H. Vandenberghe and are intended for application across hearing and balance disorders. Akouos was launched in 2017 by Dr. Manny Simons, who serves as CEO; its scientific founders and advisors include Drs. Michael McKenna, William Sewell, Richard Smith and Luk Vandenberghe. In conjunction with closing a $7.5M seed financing, the licensing and partnership framework is designed to accelerate AAV gene therapy development for hearing and balance conditions. Dr. Vandenberghe is a listed inventor on Anc-AAV intellectual property licensed to Lonza and Akouos and receives royalties.

Participated · Series A · Dec 2019

Triplet Therapeutics harnesses human genetic insights to develop treatments for repeat expansion disorders by targeting the DNA damage response pathway. The company is advancing antisense oligonucleotide (ASO) and small interfering RNA (siRNA) candidates designed to precisely knock down DDR components that drive repeat expansion. Its approach targets disease drivers upstream of current therapies with the goal of halting onset and progression across multiple disorders, including Huntington’s disease, myotonic dystrophy and spinocerebellar ataxias. Triplet has assembled a senior management team and a Scientific Advisory Board of leading investigators in the field. The company will use recent financing to progress its first development candidates into IND-enabling studies and to advance natural history studies to inform its clinical plans. Triplet was founded in 2018 and is headquartered in Cambridge, Mass.