Participated · Seed · Jan 2023

Myosana has built a muscle-specific non-viral gene delivery platform designed to target muscle tissue with large gene capacity, enable repeat dosing, and avoid immune responses associated with viral vectors. The company’s lead therapeutic approach is delivery of full-length dystrophin as a potential therapy for Duchenne muscular dystrophy. Management says the platform could be applied across a broad range of neuromuscular and cardiac diseases to increase longevity and improve quality of life. Myosana plans to identify its first development candidate for Duchenne by 2025 and management has cited an 18–24 month timeline for candidate nomination tied to platform optimization. The company recently completed a seed financing of over $5 million to support those development activities. Myosana’s leadership includes cofounders Stan Froehner, Ph.D., and Nick Whitehead, Ph.D., and newly appointed CEO Dr. Matthew Lumley, a physician-scientist with experience in rare disease clinical development. Myosana Therapeutics is developing a pioneering non-viral gene delivery platform that specifically targets skeletal and cardiac muscle cells to deliver full-length genes, including dystrophin, regardless of gene size. The platform is intended to overcome limitations of AAV vectors such as micro-dystrophin delivery and immunogenicity. The company’s initial focus is on disease-modifying therapeutics for Duchenne muscular dystrophy, with potential applicability to other neuromuscular genetic diseases. Myosana will use the seed funding to advance development of its non-viral full-length dystrophin gene therapy. The company is a University of Washington spinout led by founders Stan Froehner and Nick Whitehead. As part of the transaction, CureDuchenne’s Debra Miller will join Myosana’s board.

Led · Equity · Jan 2023

PathMaker Neurosystems develops a proprietary neuromodulation platform, MyoRegulator®, designed to suppress motor neuron hyperexcitability for disorders including ALS and post-stroke spasticity. The MyoRegulator® device delivers simultaneous multi-site direct current stimulation to modulate circuits linked to motor neuron hyperexcitability. MyoRegulator® has been investigated in two completed clinical trials for post-stroke spasticity and one first-in-human trial for ALS, and has received FDA Breakthrough Device designation. From its Boston headquarters, PathMaker is collaborating with leading institutions to run clinical studies and advance its product toward market. The company plans to launch an expanded pilot ALS study (the CALM study) using the MyoRegulator® device, with enrollment expected to begin in late 2024 at Spaulding Rehabilitation Hospital and Beth Israel Deaconess Medical Center. The company recently received targeted government funding to support this trial. PathMaker Neurosystems is a near-commercial neuromodulation company developing breakthrough non-invasive systems to treat serious neurological disorders such as stroke and ALS. Its lead product, MyoRegulator®, is a first-in-class investigational device based on the company's DoubleStim® technology intended to suppress hyperexcitable spinal motor neurons and treat muscle spasticity without drugs or surgery. MyoRegulator® has been designated by the U.S. Food and Drug Administration as a "breakthrough medical device" and is limited to investigational use. PathMaker is conducting a U.S. multi-center pivotal trial in post-stroke spasticity and plans to launch its first clinical trial in ALS this year. The company has offices in Boston and Paris and is collaborating with external institutions to accelerate commercialization. Financially, PathMaker received a $200,000 START Stage 2 award from MassVentures, following a $100,000 START Stage 1 award in 2022 and a $4.9M NIH NINDS CREATE Devices grant that underpinned the award. PathMaker Neurosystems is a near-commercial stage neuromodulation company developing non-invasive systems to treat serious neurological disorders such as stroke and amyotrophic lateral sclerosis (ALS). Its MyoRegulator® platform is based on proprietary technology to suppress hyperexcitable spinal motor neurons and the MyoRegulator® ALS device specifically targets motor neuron hyperexcitability in ALS. MyoRegulator® has been designated by the U.S. Food and Drug Administration as a "breakthrough medical device," and the devices are investigational and limited to investigational use. The company plans an ALS early feasibility study using its non-invasive technology and is collaborating with academic and clinical partners to accelerate clinical application and market entry. PathMaker has offices in Boston and Paris and highlights clinical translation to address high unmet medical need. Recent financial support cited includes a $600,000 MDA Venture Philanthropy investment, a $250,000 CERF Prize in ALS, and a $371,000 NIH grant awarded in 2021 to apply the technology to ALS. PathMaker Neurosystems is a near-commercial stage neuromodulation company developing non-invasive systems to treat people with serious neurological disorders such as stroke and ALS. Its lead product, MyoRegulator, is a first-in-class, non-invasive neuromodulation device designed to treat muscle spasticity using PathMaker’s proprietary DoubleStim® technology. MyoRegulator has completed an EU pivotal trial, is undergoing a US multi-center pivotal trial, and has been designated by the U.S. FDA as a “breakthrough medical device.” PathMaker maintains offices in Boston and Paris and is collaborating with world‑class institutions to bring its products to market. Financially, the company was awarded a $4.9M grant from the NINDS CREATE Devices program and a $100,000 Stage I START award from MassVentures to support development and transfer-to-manufacturing as it approaches commercial launch. The MyoRegulator device remains investigational and is limited by federal law to investigational use only. PathMaker Neurosystems is a clinical-stage bioelectronic medicine company developing non-invasive systems to treat chronic neuromotor conditions such as spasticity, paralysis and muscle weakness. Its lead platform, MyoAmplifier, integrates magnetic and electrical stimulation and is based on proprietary TripleStim™ technology protected by issued U.S. and foreign patents. TripleStim provides simultaneous non-invasive stimulation at cortical, spinal and peripheral sites associated with targeted muscles to improve motor function. MyoAmplifier is an investigational device and is limited by United States law to investigational use only. The company has offices in Boston and Paris and collaborates with research institutions to accelerate product development and commercialization. PathMaker is applying MyoAmplifier to pediatric cerebral palsy and other neuromotor indications where there is high unmet need.

Led · Equity · Jul 2014

Akashi Therapeutics is a clinical-stage biopharmaceutical company developing treatments for Duchenne muscular dystrophy (DMD) and other rare pediatric diseases. Its lead candidate, HT-100 (delayed-release halofuginone), is an orally available small molecule designed to reduce fibrosis and inflammation and promote healthy muscle fiber regeneration in DMD patients. HT-100 has been granted orphan designation in the U.S. and E.U. and fast track designation in the U.S., and is being evaluated in a phase 1b/2a multi-center clinical program to assess safety, tolerability, exploratory biomarkers, and efficacy endpoints. Akashi was founded by two patient organizations, Charley’s Fund and the Nash Avery Foundation, and is managed by drug development veterans. The company follows a cooperative funding model backed largely by patient advocacy organizations and has raised a cumulative total of more than $2.5 million from 25 patient groups, including a recent collective $1.5 million round. Akashi says it continues to treat patients in the ongoing trial and anticipates reporting additional progress in the months to come.