Participated · Equity · Jun 2021

Neurona Therapeutics is developing allogeneic, off-the-shelf regenerative neural cell therapy products intended to provide long-term targeted repair of the nervous system following a single administration. Its lead candidate, NRTX-1001, comprises GABAergic interneurons and is being evaluated in two ongoing open-label multicenter Phase 1/2 trials for drug-resistant unilateral and bilateral mesial temporal lobe epilepsy (NCT05135091 and NCT06422923). The company presented updated Phase 1/2 data showing in a five-subject lower-dose cohort a median seizure reduction of 75% at six months, with four of five subjects reporting >50% reduction and two subjects reporting durable >95% reduction at 16 and 21 months; NRTX-1001 has been well tolerated and neuropsychological testing suggests absence of neurocognitive impairment. The FDA granted NRTX-1001 Regenerative Medicine Advanced Therapy (RMAT) designation in June 2024 based on these positive clinical data. Neurona plans to expand indications to neocortical focal epilepsy and other CNS disorders. Financially, the company has received multiple grants from the California Institute for Regenerative Medicine (CIRM), with cumulative CIRM commitment of $18.5M across five grants, and completed a $120M private financing in February 2024 co-led by Viking Global Investors and Cormorant Asset Management. Neurona Therapeutics is a San Francisco–based clinical-stage biotherapeutics company focused on developing regenerative, allogeneic, off-the-shelf cell therapy candidates with single-dose curative potential. Its candidates are designed to provide long-term repair of dysfunctional neural networks across multiple neurological indications. The company's lead investigational candidate, NRTX-1001, is being evaluated in an ongoing open-label, single-arm Phase I/II clinical trial for treatment of drug-resistant mesial temporal lobe epilepsy (MTLE). NRTX-1001 also has potential application in Alzheimer’s disease and other disorders of the nervous system. Led by CEO Cory R. Nicholas, Ph.D., Neurona intends to use newly raised capital to advance its wholly-owned pipeline of off-the-shelf cell therapies. The company recently raised $120M and announced that Raymond Kelleher, M.D., Ph.D., of Cormorant will be joining its board of directors. Neurona Therapeutics develops neural cell therapies intended to provide long-term repair of dysfunctional neural networks in chronic neurological disorders. Its lead candidate, NRTX-1001, is a neuronal cell therapeutic derived from human pluripotent stem cells comprising GABA-secreting interneurons designed as a one-time administration to provide local long-term inhibition. The company is advancing wholly-owned, off-the-shelf neuronal, glial, and gene-edited cell therapy candidates across multiple indications, initially targeting temporal lobe (focal) epilepsy. Neurona plans a Phase 1/2a clinical study for NRTX-1001 planned to launch later this year. The company completed a $41.5M financing that will be used to advance the pipeline; total proceeds raised to date are $135 million. Neurona describes its platform as based on a novel neural cell lineage with single-dose curative potential. Neurona Therapeutics is a pre-clinical biotechnology company focused on the transplantation of selected neurons to treat intractable neurological diseases. The company develops regenerative cell-based therapeutics composed of unique cell compositions that can be precisely targeted and integrated into damaged neural circuits. Its approach emphasizes activity-dependent regulation once transplanted into host circuitry. Neurona was founded by neuroscientists and stem cell researchers from the University of California, San Francisco, including Arturo Alvarez-Buylla, Arnold Kriegstein, John Rubenstein and Cory Nicholas. The company is led by interim CEO Tim Kutzkey and launched with a $23.5M Series A financing. Neurona is based in South San Francisco, CA and remains in the pre-clinical stage.

Participated · Equity · Sep 2020

ESCAPE Bio is a clinical-stage, privately held biopharmaceutical company developing novel, precisely targeted therapeutics for genetically defined neurodegenerative diseases. Its lead programs include ESB1609, an orally administered, brain-penetrant S1P5 receptor agonist in a randomized, double-blind, placebo-controlled Phase 1 multiple ascending dose study in healthy volunteers; ESB5070, a mutant-selective LRRK2 G2019S kinase inhibitor in IND-enabling toxicology studies; and a pharmacologic structure corrector for ApoE4 Alzheimer's patients in discovery. ESB1609 has shown preclinical activity normalizing brain ceramide and sphingosine phosphate levels and promoting clearance of aggregation-prone proteins across multiple models. ESB5070 is being developed specifically for patients carrying the LRRK2 G2019S variant, which occurs in 1–3% of Parkinson’s disease patients. The company announced the closing of a $73 million financing led by Wellington Management Company LLP to advance its clinical programs. Proceeds will be used to accelerate two programs into patients who lack disease-modifying treatments. E-Scape Bio develops small-molecule drugs that target inherited genetic drivers of neurodegenerative disorders and correct dysfunctional proteins at the root of disease. Its lead program targets the Apolipoprotein E4 (ApoE4) protein structure, a major genetic risk factor for Alzheimer's disease. The company's therapeutic programs are designed to restore normal function disrupted by inherited genetic mutations, with programs aimed at Alzheimer's disease and Parkinson's disease. E-Scape Bio was co-founded by Robert Mahley, M.D., Ph.D., and Yadong Huang, M.D., Ph.D., based on their research at the Gladstone Institutes linking ApoE4 to disease pathogenesis. Leon Chen, Ph.D., is cited as the interim chief executive officer. The company says it will apply resources to rapidly build its pipeline and advance toward the clinic.

Participated · Series B · Jun 2020

C4 Therapeutics pioneers a class of small-molecule drugs that selectively destroy disease-causing proteins via targeted protein degradation using the cell's innate machinery. The company emphasizes advantages of degraders over traditional inhibitors, including potential for higher potency, reduced resistance, and greater selectivity. C4T has invested heavily in its TORPEDO™ platform, which combines a chemistry engine, proprietary assays, high-throughput screening, and predictive modeling to accelerate discovery and design of drug candidates. The company plans to use proceeds to advance multiple degrader candidates to human proof-of-concept, filing its first IND by the end of this year and targeting four drug candidates in the clinic by the end of 2022. C4T recently closed a $170 million financing (a $150 million Series B and $20 million venture debt) to support these plans. The company says the round was oversubscribed and attracted top-tier life-science investors. C4 Therapeutics is developing a new class of targeted protein degradation (TPD) therapeutics built on its proprietary Degronimid™ platform. The Degronimid technology, pioneered in the Bradner Lab at Dana-Farber and described in Science, uses small-molecule adapters to recruit the ubiquitin/proteasome system and rapidly degrade disease-causing proteins. The platform is positioned to target previously undruggable proteins and to reduce the potential for drug resistance. C4 has executed a worldwide-exclusive license with Dana-Farber for Degronimid applications and plans to advance multiple proprietary and partnered drug programs. The company announced a $73 million Series A financing to fund its early development and build its team. C4 will be located at Mass Innovation Labs in Kendall Square, Cambridge, and has appointed Jason Fisherman, M.D., as CEO to lead clinical and business development.

Participated · Series B · Sep 2019

Passage Bio is a genetic medicines company focused on developing AAV-delivered therapeutics to treat rare monogenic CNS diseases. The company works with the University of Pennsylvania’s Gene Therapy Program and Orphan Disease Center under research, collaboration and license agreements. Passage Bio has a development portfolio of five product candidates and has licensed two additional indications from Penn, with options to license up to seven more. The company planned to initiate clinical trials for lead programs in GM1 gangliosidosis and frontotemporal dementia in the first half of 2020, and a Krabbe disease program in the second half of 2020. Passage Bio is based in Philadelphia and conducts clinical development, regulatory strategy and commercialization while Penn’s GTP conducts IND‑enabling preclinical work. Financially, the company announced a $110.0 million Series B and, since launch, has raised $225.5 million in total. Passage Bio is developing a portfolio of AAV-delivered therapeutics targeting rare monogenic CNS diseases, with initial lead programs in GM1 gangliosidosis and frontotemporal dementia. The company has a research, collaboration and license agreement with the University of Pennsylvania’s Gene Therapy Program and Orphan Disease Center; Penn will perform IND-enabling preclinical work while Passage Bio is responsible for clinical development, regulatory, manufacturing and commercialization. Passage Bio plans a portfolio of five therapeutic candidates and holds an option to fund preclinical development of up to seven additional Penn programs and license resulting intellectual property. The company completed a $115.5 million Series A financing to advance its pipeline and build its team. Leadership and scientific expertise include co-founders James M. Wilson (scientific advisor), Stephen Squinto (interim CEO) and Tachi Yamada (chairman), and a Board with partners from key investors. The ODC will support natural history studies, KOL engagement and patient advocacy outreach as programs move toward the clinic.

Participated · Series B · Aug 2019

Oncorus develops next-generation oncolytic virus therapies built on a proprietary oncolytic herpes simplex virus (oHSV) platform and a synthetic oncolytic virus platform. Its lead candidate, ONCR-177, is an intratumorally administered oHSV clinical candidate armed with multiple transgenes and designed to be fully replication-competent while selectively attenuated in normal tissues. The company is also advancing a synthetic platform and novel delivery methodology intended to enable repeat intravenous (systemic) administration for indications where intratumoral dosing is not feasible, such as lung cancer. Oncorus plans to use proceeds from its recently completed financing to advance ONCR-177 into the clinic in early 2020 and to progress its portfolio for systemic administration. The company emphasizes proprietary safety strategies including micro-RNA attenuation and inactivation of neuronal retrograde transport to prevent replication in healthy tissues. Oncorus is headquartered in Cambridge, Mass. Oncorus is a Cambridge, MA–based immuno-oncology company focused on an oncolytic herpes simplex virus (oHSV) platform. Its lead candidate is in preclinical development for glioblastoma multiforme (GBM). The company plans to advance oncolytic viral constructs through preclinical development and into clinical trials, expand and improve its technology platform, and accelerate development of pipeline programs across other cancer types. Management is led by CEO and CSO Mitchell H. Finer, Ph.D., and President and CBO Cyrus D. Mozayeni, M.D. Oncorus licensed patent rights from the University of Pittsburgh based on work by Joseph Glorioso III, Ph.D., and Paola Grandi, who will join its Scientific Advisory Board. Proceeds from the Series A financing are being used to advance its immunotherapy platform and support R&D and pipeline progression.