Invest in Glafabra

Value Proposition

Glafabra is using cell therapy to provide freedom to patients living with metabolic genetic disorders. From one procedure, the patient gets relief lasting years, not weeks.

Market Problem

Standard of care is tedious. It requires a clinical visit every other week. Patients need to build their lives around these visits. The procedure involves taking an enzyme replacement therapy by infusion through a PIC line inserted into their arm upon every visit.

The beneficial activity of the therapy is short lived. It requires twice-a-month infusions to maintain enough activity to reduce the toxic metabolites that occur, due to the patients missing a crucial metabolic enzyme.

Market Solution

In a Canadian clinical trial, our cell therapy approach was found to be safe, effective and highly durable in reducing toxic metabolites (lyso-Gb3). In published results, one procedure gives the patient many years of relief - the majority of patients are still free of lyso-Gb3 at more 5 years after cell therapy procedure (PMID 39794302)

The procedure occurs in three steps:

1. Harvest cells from the patient,
2. Modify cells to express the missing protein,
3. Return 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 cell therapy.

Product Portfolio

Glafabra’s clinical plan is to create cell therapies for three type of metabolic disorders:

• Fabry: 1/10,000 (PMID 28613767) - deficiency in alpha-galactosidase A (GLA) - asset in clinical stage
• Pompe:  1/20,000 (PMID 36299500) - deficiency in acid alpha-glucosidase (GAA)
• Gaucher:  1/50,000 (PMID 36614898) - deficiency in glucocerebrosidase (GBA1)

Combined, the prevalence numbers indicate the worldwide population approaches 1.3 M for Fabry, Pompe and Gaucher combined. We plan to demonstrate the platform nature of our therapeutic approach, by showing that the same cell therapy method for Fabry can be easily reconfigured to make a cell therapy for Pompe and Gaucher. Additionally, the approach can easily be applied to any one of the other 50 LSDs, and it may even be impactful for +900 genes known to cause metabolic disorders.

Market Opportunity

There is sufficient market potential in metabolic disorders.  

Market segment calculations can be made from price of therapy at $0.5 M:

• TAM (Total Addressable Market) - Nearly 1000 metabolic disorders affect 0.1% of population - $200 B (5% addressed)
• SAM (Service Addressable Market) - 1.3 million patients for Fabry, Pompe and Gaucher - $25 B (5% addressed)
• SOM (Service Obtainable Market)  - 2000 patients (displaces nearly 25% of existing standard of care) - $1 B

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 cell therapy appears to be nearly 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 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. This is due to anti-capsid antibody reactions in the patient 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. Because our lentivector approach uses modified white blood cells, we can reach many compartments that other technologies cannot. As a result we can expect better biodistribution profiles from our cell therapy approach.

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: Dr. Dwayne Barber joined to lead the organization and to oversee US clinical trials. Drs. Armand Keating, Michael West, and Dan Fowler were recruited to provide a blend of medical and drug development guidance.  On the business side Dr. Lara Silverman joined Glafabra to coordinate the submission of IND with the FDA and Laura Viaches to help in market strategy planning..

The Ask

We currently are raising $2 M in SAFEs:

• SAFE terms - 20% discount and $17 M cap.
• Bonus deal - first $500,000 investors get $15 M cap.

The funds will help us get into US clinical trials. For use of SAFE proceeds, we need to submit an IND, identify our clinical sites, secure manufacturing of the cells, and recruit patients. Clinical progress will position us for a priced Series A one year from now, and will require $50 M to set us on course for a $5 B exit with large pharma..

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 cell therapy.
2. Clinical Site Management. The University’s Comprehensive Clinical Data Coordinating Center plans to help us pursue clinical trials. They will help manage data collection for a planned trial using a Phase I/II combined, orphan-drug-indication, accelerated-approval pathway.

In additional developments, we have moved to secure vector composition IP by signing an exclusive options agreement on a pending patent that would provide protection on our transgene compositions. 

Patient Voice

Patients from the Canadian clinical trial found cell therapy to be liberating. Ryan is perhaps one of our strongest advocates as he had to go back on ERT due to his therapy waning in effect. However, two more of the five total patients in the trial elected to go off ERT after their cell 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 longer lasting relief to patients with metabolic disorders.  Current therapies are sufficient, but tedious. Our approach offers a long lasting relief, which is demonstrated to be at least 5 years durability.
• 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. Once IND is filed, we will seek a priced round near $50 M in 2026, which we expect will unlock a $5 B exit potential by 2030. 
• Clinical Stage. We are in the clinic with our lead asset for Fabry Disease. We plan to bring two more therapies forward in Pompe and Gaucher. Success with these assets will demonstrate the platform 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.