PASG — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-05-12

• We have a preclinical research program to develop a genetic medicine to treat Huntington’s disease through the Gemma Collaboration Agreement. Huntington’s disease, or HD, is an adult-onset, progressive neurodegenerative disease characterized by motor, cognitive, and behavioral deterioration, ultimat…

• 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 addit…

• 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. This collaboration provided access to differentiated scientific exp…

• In addition to the indications above, we believe ALS and AD represent future potential pipeline expansion opportunities for PBFT02. However, as described above, we are currently evaluating potential next steps in the clinical development of PBFT02.

• PBFT02 is 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. FTD-GRN is an inheritable form of FTD caused by reductions in PGRN production due to mutations in the GRN gene. PGRN is a complex and highly conserv…

Text removed vs the prior filing · source: 10-Q · 2025-11-10

• We are party to a series of sublicense agreements, as amended, 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 o…

• 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 the Trustees of the University of Pennsylvania’…

• 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. FTD-GRN is an inheritable form of FTD caused by reductions in PGRN production due to mutations in the GRN gene. PGRN is a …

• Currently, there are no disease-modifying therapies approved for the treatment of FTD-GRN, and we estimate the prevalence of FTD-GRN in the United States and Europe is approximately 18,000, based on available literature. Supported by findings in preclinical studies, we believe that PBFT02 may provid…

• In June 2025, we reported biomarker data from patients in our upliFT-D trial. Dose 1 of PBFT02 (3.3e10 genome copies/g estimated brain weight, or 4.5e13 total genome copies) resulted in robust and durable increases in CSF PGRN levels, with concentrations increasing from below 3.0 ng/mL at baseline t…

Text added vs the prior filing · source: 10-Q · 2026-05-12

• In addition to the other information set forth in this report, you should carefully consider the factors discussed in Part I, “Item 1A. Risk Factors” in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025, as filed with the SEC on March 3, 2026, which could materially affect o…

• Except as set forth below, there were no material changes to the risk factors previously disclosed in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025, as filed with the SEC on March 3, 2026.

• If our continued exploration of strategic alternatives is unsuccessful, our financial condition and results of operations may be materially adversely affected.

• On April 20, 2026, we announced that we have initiated a review of strategic alternatives to maximize shareholder value. These strategic alternatives may include, but are not limited to, merger or acquisition transactions, a reverse merger, a sale of assets of the Company, strategic partnerships, li…

• specific action or otherwise determines that disclosure is appropriate or required. There can be no assurance that the process will result in any such transaction. Any potential strategic alternative would be dependent on a number of factors that may be beyond our control, including, among other thi…

Text removed vs the prior filing · source: 10-Q · 2025-11-10

• Investing in our common stock involves a high degree of risk. Before making your decision to invest in shares of our common stock, you should carefully consider the risks and uncertainties described below, together with the other information contained in this quarterly report, including our financia…

• Our business is subject to a number of risks and uncertainties, including those immediately following this summary. Some of these risks are:

• ●We are a clinical stage genetic medicines company with a history of operating losses, and we may not achieve or sustain profitability. We anticipate that we will continue to incur losses for the foreseeable future. Our limited operating history may make it difficult for you to evaluate our success …

• ●We will need to raise additional funding before we can expect to become profitable from any potential future sales of our products;

• ●PBFT02 is currently our sole clinical stage product candidate and we may not be able to successfully develop and commercialize PBFT02;