Q2 2025 Update

Glafabra's Progress Towards Repeating Fabry Trials in USA

Glafabra attending BIO International Convention - +20,000 attendees: pharma, VC and support services contractors

Health of Business

Company Synopsis:

  • Glafabra is a clinical stage company bringing a cell-based gene therapy to patients with enzyme deficiency disorders.

  • Providing patients a freedom of lifestyle with one dose lasting years, not weeks.

  • We are currently partnering with various investors to bring to market a set of gene therapies addressing enzyme deficiency in Fabry, Pompe and Gaucher diseases.

  • Our lead asset is for Fabry disease and is clinically demonstrated to be Safe, Effective and Durable (PMID 39794302).

Glafabra is meticulously tracking its progress through a series of Objectives and Key Results (OKRs). Our primary goal, PO1, is to raise $2,000,000 by December 31, 2025. Key results include finalizing our IP strategy by August 1, with specific action plans to submit and option in key intellectual property by June 30. Another key result is for the "Big Checks" exceeding $500,000, where we aim to achieve one term sheet by October 1. This result is supported by the activity of pitching at events like Bullpen, BIO, Healthcare Shares and Kinect Capital. Another key result is revamping our data room by July 1 and generating 50 new LinkedIn introductions. On "Small Checks" key results (under $500,000), we are targeting 100 commitments on Wefunder by year-end, which will require dedicated marketing efforts and direct outreach for initial commitments.

Investor Asks

To achieve our fundraising goals, we are actively seeking introductions to various investor groups. So any introductions that can be made are quite welcome! We are wanting to connect with early stage investors who want to partner in bringing cell-based gene therapies to patients with enzyme deficiency disorders.

At BIO2025 we had good VC and Strategic meetings and are excited to see where those relationship can grow. Specifically, we're targeting major players in the cell and gene therapy (CTG) space such as Novo Holdings, Deerfield, RA Capital, ARCH, Casdin, Alexandria, NEA, and Orbimed. So again, any introductions to individuals at these firms would be highly appreciated. We're also keen to connect with any individuals interested in angel investing in the CTG space.

Investor Shoutouts

We extend a sincere thank you to our committed investors. Founder/investors Jeff Medin and Chris Hopkins have begun strategic planning for a potential trial in Japan and strengthening ties with key Japanese pharma companies, Kyowa Kirin, Kaken, and Teijin. Ronan Foley has committed to $10,000 under the pre-seed note funding terms. Among our advisors, Aneal Khan has also committed to $10,000 under the same terms and is planning a poster presentation at a meeting in Japan this fall, further aligning with our strategic geographic interests. These early commitments are vital to our momentum.

People Updates

Glafabra is excited to announce key additions to our leadership team. Elizabeth Wagner has joined as acting Chief Operations Officer, bringing over 18 years of life science executive experience, including leading Constant Therapeutics to three Phase 2 assets. Her expertise in navigating CROs, labs, and CDMOs will be instrumental in advancing our pipeline.

We also welcome Krista Casazza as acting Director of Regulatory Science. Krista previously served as Scientific Director for the Critical Path Institute's CPLD pre-Consortia, leading efforts to develop regulatory-grade biomarkers for lysosomal storage diseases. Her deep understanding of regulatory pathways will be crucial for our IND filing.

Monthly Highlights

A significant highlight for May was initiating the pursuit of Orphan Drug Designation (ODD) with the FDA for our Fabry asset. This critical milestone, led by Elizabeth Wagner with support from Krista Casazza and Dwayne Barber, unlocks valuable incentives like tax credits, fee waivers, and market exclusivity. Furthermore, our CEO, Chris Hopkins, was accepted into the Innosphere virtual incubator program, providing valuable structure and coaching for our business planning and investor presentations. Chris successfully pitched at the Bullpen group at BIO International Convention, leading to follow-on meetings. Our "Strategic deck" was also well-received in partnering meetings.

Monthly Lowlights

Our progress in collecting "Small Checks" for pre-seed funding has been slower than anticipated. Despite setting up a Wefunder campaign, it has not yet gained self-sustaining momentum. To overcome this, Chris has conducted interviews with individuals from companies that successfully raised funds, revealing a common need for a dedicated marketing team specializing in Wefunder campaign promotion. Securing the necessary resources to engage such a firm is a current challenge and a high priority to accelerate our early fundraising efforts.

What's on Deck

Looking ahead, our near-term targets (within 6 months) include securing Orphan Drug Designation (ODD) for Fabry and preparing our pre-IND submission for an FDA meeting. For our far targets (beyond 6 months), our primary objective is to obtain an open IND on Fabry, enabling US-based clinical trials. Concurrently, we plan to advance our additional indications, Pompe and Gaucher, towards IND readiness. These steps are crucial for translating our innovative science into clinical impact.

Word-of-Mouth / Press Roundup

Glafabra recently secured positive press through a paid PR campaign announcing our Letter of Intent with the University of Utah Health as our Fabry clinical site. This significant partnership was highlighted across multiple platforms, including Pulse2.com and Advo.io, and shared prominently on LinkedIn by the University of Utah Health. Additionally, we generated soft PR on social channels regarding the valuable addition of Elizabeth Wagner to our team, further enhancing our professional visibility and credibility within the industry.