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

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

The beneficially 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 the a crucial metabolic enzyme..  

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.

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 be reconfigured 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.

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 Accessible Market) - 1.3 million for Fabry, Pompe and Gaucher - $25 B (5% addressed)

  • SOM (Service Obtainable Market)  - 2000 patients (displace 25% of existing standard of care) - $1 B

Glafabra's approach is superior over our competitors. 

  • Long Duration. Compared to existing ERT therapy, we can achieve an effect that last for years, not weeks. The clinical team’s latest published study is a 5 year follow up (PMID 39794302) and it show 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 at least 5 years.  However, it remains to be seen how long the therapy effect will occur, and many of the patients in the trial are on track for 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 profile from our cell therapy approach.

Glafabra has a seasoned team. Chris Hopkins as CEO is a serial entrepreneur. He is classically trained in human genetics and biochemistry, and he obtained an MBA to help foster his past entrepreneurial success. He is backed by two cofounders. Dr. Jeffrey Medin, PhD and CSO, and Dr. Ronan Foley, MD and CMO, who are pioneers in development of lentivector cell therapies. We also have onboard a high quality advisory team. Dr. Dwayne Barber joined to lead the the organization and oversight of US clinical trials. Drs Armand Keating, Michael West, and Dan Fowler were recruited to provide a mixture of medical and drug development guidance. On the business side Dr. Lara Silverman joined to aid in submission of IND with FDA and Laura Viaches was recruited to help us in market strategy planning.

We currently are raising $2 M in SAFEs at a 30% discount and $17 M cap (bonus deal available for the first $500,000 invested). The funds will be used to submit an IND, identify our clinical sites, secure CMC, and recruit patients. Clinical progress will position us for a priced Series A one year from now that ask for $45 M, which sets us on course for a $2 B exit with large pharma.

We have already made good progress on securing a clinical site. We have signed an LOI with the University of Utah to be our cGMP manufacturer under its Cell Therapy and Regenerative Medicine (CellReGen) program, and clinical partner pursuing clinical trials for Phase I/II orphan-indication, accelerated-approval path with the assistance of the University’s Comprehensive Clinical Data Coordinating Center. Furthermore, we have moved on securing IP by signed an exclusive options agreement on a pending patent that would provide protection on our transgene compositions. 

Patients from the Canadian trial found the cell therapy to be liberating.  Ryan is perhaps one of our strongest advocates as he had to go back on ERT due to the his therapy waning in effect. However, two more of the five total patients in the trail elected to go off ERT after their cell therapy transplant and have remained off ERT ever since (+5 years and counting).

In Summary

  • Market Need. We are bringing an innovative therapy approach to give long lasting relief to patients with metabolic disorders.  Current therapies are sufficient, but tedious. Our approach offers long lasting relief, which is demonstrated to be at least 5 year durability.

  • Sufficient Market. Our therapies are accessing a $25 B market. We have line-of-site to $ 1 B revenue after year 5 on the market.

  • $2 Million Ask. We currently need $2 M to set us on the path to the clinic. Once IND is filed we will start to seek a priced round near $45 M in 2026. This expect to unlock a potential in funding to unlock a $2B exit potential to occur near 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. This will demonstrate the platform nature of our therapy 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.