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Business Overview We are a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases.
We have initiated clinical development of PBFT02 in FTD- C9orf72 patients in the upliFT-D trial for this population.
We have an active preclinical research program to develop a genetic medicine to treat Huntington s disease through our research, collaboration and license agreement, or the Gemma Collaboration Agreement, with Gemma Biotherapeutics, Inc., or Gemma.
Huntington s disease, or HD, is an adult-onset, progressive neurodegenerative disease characterized by motor, cognitive, and behavioral deterioration, ultimately leading to death within approximately 15 to 20 years after symptom onset.
There are currently no disease-modifying therapies approved for the treatment of HD, and we estimate the prevalence of HD in the United States and Europe is approximately 70,000, based on available literature.
We are also party to a series of sublicense agreements, as amended, with Gemma in connection with the outlicensing of three pediatric programs we had previously advanced to clinical stage development, collectively the Outlicensed Programs, and such agreements, the Amended Gemma Sublicenses.
In addition, we entered into a Transition Services Agreement, as amended, with Gemma.
Prior to the execution of the Outlicense Transaction Agreements, we advanced our preclinical programs through our research collaboration with the Trustees of the University of Pennsylvania s, or Penn s, Gene Therapy Program, or GTP.
Our development programs consist of: US/EU prevalence per third-party sources In addition to the indications above, we believe amyotrophic lateral sclerosis, or ALS, and Alzheimer s disease, or AD, represent future potential pipeline expansion opportunities for PBFT02.
PBFT02 for the Treatment of FTD-GRN We are currently developing PBFT02, a gene replacement therapy which utilizes an AAV1 capsid to deliver a functional copy of GRN encoding for PGRN, for the treatment of FTD- GRN .
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Business Overview We are a clinical stage genetic medicines company on a mission to improve the lives of patients with neurodegenerative diseases.
We are proceeding with clinical development of PBFT02 in FTD- C9orf72 patients and plan to initiate dosing in the first half of 2025.
On July 31, 2024, we entered into a series of sublicense agreements with Gemma Biotherapeutics, Inc., or Gemma, a newly formed genetic medicines company co-founded by Dr.
James Wilson in connection with the outlicensing of PBGM01 for the treatment of GM1 gangliosidosis, or GM1, PBKR03 for the treatment of Krabbe disease, and PBML04 for the treatment of metachromatic leukodystrophy, or MLD, collectively the Outlicensed Programs, and such agreements, the Gemma Sublicenses.
Pursuant to the Gemma Sublicenses, Gemma will also be responsible for all payments due to the Trustees of the University of Pennsylvania, or Penn, under the Penn License Agreement, as further described below, related to the Outlicensed Programs.
We also entered into a transition services agreement with Gemma, or the Transition Services Agreement, as amended by the First Amendment to the Transition Services Agreement, dated January 31, 2025, pursuant to which, we will provide transitional services at cost to Gemma through May 31, 2025, unless terminated earlier, and be entitled to reimbursement for transitional services performed retroactively from March 1, 2024, related to the transfer of the Outlicensed Programs.
As of December 31, 2024, we have collected $5.0 million in initial payments and $3.2 million in transition services payments under these agreements.
Subsequent to December 31, 2024, we have received an additional $0.5 million in transition services payments.
As a result of the Outlicense Transaction Agreements, we also entered into an Amended and Restated Research, Collaboration and License Arrangement with Penn as of July 31, 2024, or the Penn License Agreement, to (i) terminate our funding of discovery research; (ii) terminate the research and exploratory research programs being conducted by Penn; (iii) terminate the remaining options we had to select new research programs in the CNS field; and (iv) terminate the transaction fee due to Penn as a result of certain corporate transactions.
Prior to the execution of the Outlicense Transaction Agreements, we progressed four product candidates from preclinical to clinical stage development and had one active preclinical program in Huntington s disease through our research collaboration with Penn s Gene Therapy Program, or GTP.