Invest in Glafabra

Value Proposition

Glafabra is bringing Live-cel - a living therapy to patients of genetic disorders for long lasting relief and stop disease progression in lysosomal storage disorders (LSDs).

Market Problem

Standard of care is challenging for LSDs - tedious clinical visit every other week for 4 hrs long protein infusions of enzyme replacement therapy (ERT).

Patients need a better solution.

A one time treatment that will last for years, and perhaps... the rest of their life!

Glafabra is introducing: Live-cel - A One-time Treatment for a Lifetime of Relief.

Market Solution

Glafabra's Live-cel is a living therapy that set patients on track to live a normal life. The therapy has already been demonstrated in patients. In a Canadian clinical trial, our Live-cel living therapy approach was found to be safe, effective and capable of lasting more than 5 years after one infusion procedure.

The Live-cel procedure is conceptually simple and occurs in three steps:

1. Harvest the patient's own cells,
2. Modify these cells to express the missing protein,
3. Return the cells back into the patient as the therapy.

In the trials, the team observed at least a 2x drop in toxic metabolites for all 5 patients treated with our Live-cel living therapy - an effect that, for most patients, is lasting longer than the 5 years so far measured.

Product Portfolio

Glafabra’s clinical plan is to create Live-cel living therapies for three type of metabolic disorders:

• Fabry (Clinical Stage): deficiency in alpha-galactosidase A (GLA).
• Pompe: deficiency in acid alpha-glucosidase (GAA).
• Gaucher:  deficiency in glucocerebrosidase (GBA1).

There are 1.3 M for Fabry, Pompe and Gaucher around the world and only 2% currently served with the standard-of-care ERT treatment. However, Sanofi is currently netting nearly $3 B per year from treatments for Fabry, Gaucher, and Pompe.

Competitive Landscape

Glafabra's approach is superior over our competitors. 

• Long Duration. Compared to existing ERT therapy, we can achieve an effect that lasts for years, not weeks. The clinical team’s latest published study is a 5 year follow up (PMID 39794302) and it shows the reduction in the lyso-Gb3 toxic metabolite was maintained in 4 out of 5 patients. As a result, the duration of our Live-cel therapy appears to be at least 5 years. However, it remains to be seen how long the therapy effect will persist. Indeed, many of the patients in the clinical trial are on track for a 10 year or more durability.
• Repeatable. Compared to AAV, which is another emerging technology, our procedure can be repeated as many times as needed. In contrast, repeatability is challenged for the “one-and-done” AAV technology, where anti-capsid antibody reactions in the patient occur from exposure to viral capsid. Our patients never see a viral capsid and will not have an anti-capsid antibody response.
• Tissue Access. Accessing certain body compartments, such as getting past the blood brain barrier (BBB), can be challenging for many therapies. In contrast, our living therapies approach uses modified white blood cells, which can reach many compartments that other technologies cannot. As a result, Our Live-cel therapy approach can achieve a stronger and more systemic relief from symptoms that any of the competing technologies.

Experienced Team

Glafabra formed as a collaboration between three founders. In the fall of 2024, discussions among the three founders, Drs. Chris Hopkins, Jeffrey Medin and Ronan Foley led them to realize that a pending 5 year followup publication of the Canadian trial data was just too compelling and a new company needed to be formed.  To aid in the formation and growth of the new company, a set of advisors were recruited: Drs. Aneal Khan, Armand Keating, Michael West, and Dan Fowler, who provide a blend of medical and drug development guidance.  On the business side, Drs Dwayne Barber and Lara Silverman, Elizabeth Wagner, Krista Casazza, Laura Viaches, and Katie Hewitt are helping coordinate our clinical trials and arrange partnering with big pharma.

The Ask

Currently asking for $2,000,000 pre-seed:

• Pre-seed - Convertible Notes in two tranches (Active).
• Early bird: $500,000 at $15 M cap, 20% discount, 6% interest.
• Standard: $1,500,000 at $20 M cap. 20% discount, 6% interest.

The pre-seed is followed by two priced rounds before Glafabra becomes well positioned for exit:

• Priced Seed - $6,000,000 at a valuation near $60,000,000 (Starts in 2026).
• Series A - $50,000,000 at a valuation near $150,000,000 (Starts in 2027).

On the use of funds, pre-seed gets us to open IND status, priced seed brings in finding on clinical efficacy for our Fabry Live-cel therapy and Series A demonstrates the broader applicability of our approach to multiple enzyme deficiency disorders, which unlocks the ability to seek a multi-billion dollar exit for the company.

Development Status

We have already made good progress on securing a clinical site. We have signed an LOI with the University of Utah to partner on two services:

1. cGMP Manufacturer.  The Cell Therapy and Regenerative Medicine (CellReGen) program at the University is planning to be our manufacturing partner in the production of modified patient cells to be used in our Live-cel therapy.
2. Clinical Site Management. The University’s comprehensive clinical Data Coordinating Center plans to help us administrate our clinical trials. They will help manage data collection for a planned trial using a Phase I/II combined, orphan-drug-indication, accelerated-approval pathway.

For intelectual property, we have moved to secure vector composition by signing an exclusive options agreement on a pending patent that would issue with claims providing protection on our transgene compositions. 

Patient Voice

Patients from the Canadian clinical trial found Live-cel therapy to be liberating. Ryan is perhaps one of our strongest advocate. He had to go back on ERT due to his therapy waning in effect at the end of year 4. However, two more of the five total patients in the trial elected to go off ERT after their Live-cel therapy transplant and have remained off ERT ever since (+5 years and counting).

Overview

To summarize,

• Market Need. We are bringing an innovative therapy approach to give long lasting relief to patients with metabolic disorders.  Current therapies are sufficient, but require frequen clinical visits for tedious infusions every-other-week to control toxic metabolite buildup. Our one time treatment approach gives patients a broad symptom relief that lasts for many years.
• Sufficient Market. We are developing an accessible segment (12%) of a $200 B addressable market.
• Funding target. We currently need $2 M to set us on the path to the clinic (obtain Orphan Drug Designation, achieve preIND meeting and file IND). Additional funding gets us into the clinical studies on the Fabry asset (priced seed) and the next funding establishes efficacy in Fabry, Pompe and Gaucher (Series A), which combine to unlock a $5 B exit potential by 2030. 
• Clinical Stage. We have completed clinic trials in Canada with our Live-cel therapy for Fabry Disease. We now need to activate similar clinical trials in the US with IND filing and clearance. We plan to bring at least two more therapies forward into the clinic (Pompe and Gaucher). Success with these assets will demonstrate the repeatable nature of our technology where any coding sequence can be swapped into the lentivector and enable treatment of a wide variety of metabolic disorders.

With adequate capitalization, Glafabra will remain on track to become an attractive acquisition to Pharma by the end of the decade.

Email Chris Hopkins at chris@glafabra.com for more information.