Participated · Series D · Jan 2023

Palvella Therapeutics focuses on developing and commercializing novel therapies for serious, rare genetic skin diseases that currently lack FDA-approved treatments. The company advances a broad pipeline built on its patented QTORIN platform, initially targeting lifelong rare skin disorders. Palvella works with patient advocacy organizations and patient registries to design accelerated development programs to expedite patient access to targeted therapies. Its lead product candidate is QTORIN 3.9% rapamycin anhydrous gel, being developed for pachyonychia congenita, microcystic lymphatic malformations, and prevention of basal cell carcinomas in Gorlin syndrome. QTORIN rapamycin has received FDA Fast Track Designation for all three indications. The company recently completed a Series D financing to support advancement of QTORIN rapamycin across these indications. Palvella Therapeutics is a Wayne, PA–based rare disease biopharmaceutical company focused on developing and commercializing pathogenetically targeted therapies for serious genetic diseases with no approved treatments. Its lead program, PTX-022 (QTORIN™ 3.9% rapamycin anhydrous gel), is in a Phase 2/3 pivotal study for pachyonychia congenita (PC), a rare, chronically debilitating and lifelong genetic disease estimated to affect more than 9,000 individuals in the U.S. The company partners with patient advocacy organizations and patient registries to design fit-for-purpose, accelerated clinical development programs aimed at expediting targeted therapies to patients lacking approved options. Palvella intends to use the proceeds from the financing to advance PTX-022 in adults with PC. Wes Kaupinen serves as President and CEO. In conjunction with the funding, Scott Morenstein of CAM Capital and Cory Freedland of Samsara BioCapital joined Palvella’s Board of Directors. Palvella Therapeutics is a Wayne, PA-based rare disease biopharmaceutical company focused on developing and commercializing pathogenetically targeted therapies for debilitating, rare genetic diseases with no approved treatments. The company is led by Wes Kaupinen, president and chief executive officer. Its lead program, PTX-022 (QTORIN™ rapamycin formulation), is entering Phase 2/3 development for pachyonychia congenita (PC). PC is a rare, chronically debilitating lifelong monogenic disease in which mutations in keratin genes lead to dysregulated keratinocyte proliferation, increased skin fragility and impaired plantar skin barrier function. Palvella will continue to retain responsibility for all clinical development, regulatory, manufacturing, marketing and other commercialization activities worldwide. The company has secured external development funding to advance PTX-022.

Participated · Series B · Apr 2021

Jaguar Gene Therapy is advancing an initial pipeline of three AAV-based, preclinical gene therapy programs targeting Type 1 galactosemia (JAG101), SHANK3-related neurodevelopmental disorders including Phelan-McDermid syndrome (JAG201), and Type 1 diabetes (JAG301). The company emphasizes first-in-class CMC (Chemistry, Manufacturing and Controls) capabilities and commercially scalable manufacturing processes for AAV therapeutics. Jaguar says it will use the proceeds from the new investments, together with existing cash resources, to expand CMC capabilities and to advance IND-enabling activities across its preclinical pipeline. The team highlights prior experience that includes overseeing development and launch of one of the first FDA‑approved gene therapies. Jaguar positions JAG301 as a cure-oriented program aiming to restore endogenous insulin production via PAX4-mediated transdifferentiation of alpha cells. The company continues to evaluate opportunities to expand its investigational gene therapy pipeline. Jaguar Gene Therapy, based in Lake Forest, Ill., develops AAV9-based gene therapies for patients with severe genetic diseases. Led by former AveXis leadership, the company is advancing a pre-clinical pipeline including JAG101 for galactosemia, JAG201 for a genetic cause of autism spectrum disorder, and JAG301 for Type 1 diabetes. JAG101 targets galactosemia, a condition affecting an estimated 4,500 patients in the U.S. in its most severe form and an additional ~17,000 with less severe disease; current standard of care is dietary and often insufficient. JAG201 targets an estimated 30,000 U.S. patients with a genetically caused autism spectrum disorder for which no treatments exist, while JAG301 is evaluating proof-of-concept data to define clinically relevant newly diagnosed Type 1 diabetes patients. A majority-owned subsidiary, Axovia Therapeutics, is advancing AXV101 for BBS1, a subset of Bardet-Biedl syndrome. Jaguar emphasizes development, manufacturing and commercialization capabilities and intends to use recent financing and existing cash to advance its initial pre-clinical programs.

Participated · Series D · Jul 2020

Encoded Therapeutics is a South Francisco, Calif.-based precision gene therapy company developing precision gene therapies for a broad range of severe genetic disorders. Led by co-founder and chief executive officer Kartik Ramamoorthi, Ph.D., the company is advancing its lead asset, ETX101, for the treatment of SCN1A+ Dravet Syndrome. Encoded leverages a discovery engine that combines biological and computational approaches to identify and screen human DNA regulatory elements at high throughput. ETX101 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation by the U.S. FDA for SCN1A+ Dravet Syndrome. The company intends to use proceeds from the Series D to conduct clinical trial activities, including a natural history study and first-in-human trials for ETX101, and to progress its pipeline of gene therapies for additional pediatric CNS disorders. The article reports the company raised $135M in Series D financing. Encoded Therapeutics develops precision gene therapies that aim for cell-type selectivity, potency, and the ability to modulate expression of endogenous genes. The company leverages a proprietary platform and initially uses clinically‑validated adeno-associated viral (AAV) vectors. It is focused on four core research areas: neurocircuitry disorders, liver and metabolic disease, neurodegeneration and cardiovascular disease. Encoded’s lead program targets Dravet syndrome, a severe genetic disorder characterized by uncontrolled seizures, ataxia, developmental delays and elevated risk of SUDEP. The company raised $104M in a Series C and intends to use the proceeds to advance its Dravet program, push its preclinical pipeline and expand platform-enabled programs for severe genetic disorders. Encoded was incubated by Illumina Accelerator, seeded by Venrock and ARCH Venture Partners, and is led by co-founder and CEO Kartik Ramamoorthi, Ph.D.

Led · Seed · Apr 2020

Taysha Gene Therapies develops intrathecally delivered AAV gene-replacement therapies targeting rare monogenic central nervous system (CNS) diseases. Its pipeline includes TSHA-102 (a self-complementary AAV9 therapy for Rett syndrome using the miRARE auto-regulatory element) and TSHA-120 (an AAV9 program for giant axonal neuropathy, in Phase 1/2). TSHA-102 is described as the first-and-only gene therapy in clinical development for Rett syndrome; both programs have received multiple Orphan Drug designations. Taysha has partnered with the UT Southwestern Gene Therapy Program and emphasizes a fully integrated AAV platform to translate treatments from bench to bedside. The company is publicly listed (Nasdaq: TSHA) and announced a strategic equity investment from Astellas to support advancement of its clinical-stage programs. Taysha’s stated mission is to eradicate monogenic CNS disease by rapidly developing curative medicines. Taysha Gene Therapies develops gene therapies. The company announced it entered into a loan and security agreement with Silicon Valley Bank. The agreement provides Taysha with up to $100 million of borrowing capacity. The financing is described as a non-dilutive term loan. The facility is structured as debt under a term loan instrument to bolster the company's liquidity. Taysha Gene Therapies is a patient-centric company developing AAV-based gene therapies to treat monogenic CNS diseases in both rare and larger patient populations. It was founded in partnership with the UT Southwestern Gene Therapy Program and has assembled a pipeline of 17 product candidates with exclusive options to acquire four additional programs. Lead programs include TSHA-101 for GM2 Gangliosidosis, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder and TSHA-104 for SURF1 deficiency, with TSHA-101 expected to enter clinical studies later this year. Taysha expects to file INDs for these four candidates by the end of 2021. The company plans to use financing proceeds to advance initial cohorts into the clinic, accelerate IND submissions and build a commercially scalable GMP manufacturing facility. Taysha emphasizes rapid translation of preclinical programs into the clinic to develop potentially curative therapies for CNS disease. Taysha Gene Therapies was launched to eradicate severe and life-threatening monogenic CNS diseases by advancing a pipeline of AAV gene therapies. The company currently has 15 AAV programs with options to an additional four programs; TGTX-101, a gene replacement for GM2-Gangliosidosis, is expected to enter clinical development later this year. Taysha plans to file four IND applications by the end of 2021, including indications for SURF1 deficiency, SLC6A1 haploinsufficiency and Rett syndrome. It has a strategic partnership with the UT Southwestern Gene Therapy Program, which will conduct discovery, preclinical research, IND-enabling studies, clinical GMP manufacturing and natural history studies. Taysha will lead clinical development, regulatory strategy, commercial manufacturing and commercialization. The company is developing a novel AAV capsid platform using machine learning, DNA shuffling and directed evolution, as well as an AAV redosing platform that targets the vagus nerve. Taysha launched with $30 million in seed financing and brings together former AveXis investors and executives alongside UT Southwestern expertise.