Regulatory submission: BLA planned for late 2028

“LATE-2028 BLA regulatory filing” (opens the source at this quote in a new tab)
“FDA agreement to complete Cohort 4 supporting BLA submission for accelerated approval; PRV eligible” (opens the source at this quote in a new tab)
“Clear path to BLA submission to support accelerated approval” (opens the source at this quote in a new tab)

Launch: Anticipated in mid-2029

“Accelerating Danon Diagnosis: Focused Path to Launch” (opens the source at this quote in a new tab)
“At launch (Mid-2029)” (opens the source at this quote in a new tab)

Investigational status as of October 2026

“RP-A501 is investigational, and its safety and efficacy have not been established.” (opens the source at this quote in a new tab)
“Meeting the pivotal study’s efficacy success criterion would not, by itself, establish that RP-A501 will receive regulatory approval.” (opens the source at this quote in a new tab)
“Approval requires FDA review of the complete application.” (opens the source at this quote in a new tab)

“The product is not authorised anywhere in the European Community.” (opens the source at this quote in a new tab)

Current phase: pivotal phase 2 trial with cohort 4 enrolling (October 2026)

“Global, single-arm, Pivotal Phase 2 Trial of RP-A501 ongoing; 12-Patients aligned with FDA” (opens the source at this quote in a new tab)
“Enrolling and treating remaining Cohort 4 patients (n=9) in Pivotal Phase 2 Study” (opens the source at this quote in a new tab)
“Cohort 1-3 completed” (opens the source at this quote in a new tab)
“Pivotal data package: 12 patients at recalibrated dose + revised immunomodulatory regimen (Cohort 4)” (opens the source at this quote in a new tab)

“Importantly, the first three patients treated at the recalibrated dose under the modified protocol count toward the 12-patient pivotal population, leaving nine additional patients to be enrolled and treated.” (opens the source at this quote in a new tab)

“Cohort 4: Males age ≥ 8 years [N=12 subjects; enrolling]” (opens the source at this quote in a new tab)

Enrollment completion announced in September 2024 under the original design (history; the pivotal population was redefined in 2026 as the 12 participants of cohort 4)

“today announced that all patients have been enrolled in the global, pivotal Phase 2 clinical trial evaluating RP-A501 to treat male patients with Danon disease.” (opens the source at this quote in a new tab)

“In September 2024, we announced completion of enrollment of 12 patients in the Phase 2 study across sites in the U.S. and EU.” (opens the source at this quote in a new tab)

“Prior to the clinical hold, six patients with Danon disease were treated with RP-A501 in the Phase 2 study.” (opens the source at this quote in a new tab)

Phase 1 trial: seven participants treated, trial completed, long-term follow-up ongoing

“We treated seven patients in the single-arm, open-label, multi-center Phase 1 clinical trial assessing the safety and preliminary efficacy of RP-A501, which enrolled adult/older adolescent and pediatric male DD patients.” (opens the source at this quote in a new tab)
“In November 2024, we announced positive results and presented long-term safety and efficacy results of the Phase 1 study at the American Heart Association’s 2024 Late-Breaking Science sessions and simultaneously published these data in the New England Journal of Medicine.” (opens the source at this quote in a new tab)

“Phase 1: 3 years (completed)” (opens the source at this quote in a new tab)
“Phase 1 LTFU: up to 10 years (ongoing)” (opens the source at this quote in a new tab)

IND clearance in January 2019 and Fast Track designation in February 2019

“In January 2019, we announced the clearance of our IND application by the FDA for RP-A501, and in February 2019, we were notified by the FDA that we were granted Fast Track designation for RP-A501.” (opens the source at this quote in a new tab)

“Jan. 22, 2019 12:00 UTC” (opens the source at this quote in a new tab)
“today announces the clearance of the Company’s Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) for RP-A501.” (opens the source at this quote in a new tab)
“We expect to initiate a Phase 1, first-in-human study in the U.S. in the second quarter of 2019 to assess the safety, tolerability and preliminary efficacy of RP-A501.” (opens the source at this quote in a new tab)

“We have received Fast Track designation for our RP-A501, RP-L102, RP-L201, and RP-L301 product candidates.” (opens the source at this quote in a new tab)

FDA orphan drug designation: designated 1 February 2019

Field	Quoted record
Generic name	“Adeno-associated virus serotype 9 vector containing the human lysosome-associated membrane glycoprotein 2 isoform B transgene (AAV9.LAMP2B)”
Date designated	“02/01/2019”
Orphan designation	“Treatment of Danon Disease”
Orphan designation status	“Designated”
FDA orphan approval status	“Not FDA Approved for Orphan Indication”
Sponsor	“Rocket Pharmaceuticals, Inc.”

Rare pediatric disease designation: held by January 2023, grant date not stated

“Orphan Drug, Rare Pediatric and Fast Track designations in the US (eligible for PRV)” (opens the source at this quote in a new tab)

“We have received rare pediatric disease designation for RP-A501 for DD, RP-L102 for FA, and RP-L201 for LAD-I.” (opens the source at this quote in a new tab)

Regenerative medicine advanced therapy designation: announced 7 February 2023

“CRANBURY, N.J. – Feb. 7, 2023 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT)” (opens the source at this quote in a new tab)
“today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A501, the Company’s investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon Disease” (opens the source at this quote in a new tab)
“RMAT designation was granted based on positive safety and efficacy data from the Phase 1 RP-A501 clinical trial and will provide the benefits of added intensive FDA guidance and expedited review through the program’s development.” (opens the source at this quote in a new tab)
“Rocket also holds Orphan Drug (U.S.) and Rare Pediatric designations for the RP-A501 program.” (opens the source at this quote in a new tab)

Benefits of RMAT designation as the company describes them

“Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early FDA interactions to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the Biologics License Application (BLA) and other opportunities to expedite development and review.” (opens the source at this quote in a new tab)

EMA PRIME eligibility: entry in the EMA list of medicines in the PRIME scheme (date of granting 25 May 2023)

Field	Quoted record
Name	“RP-A501”
Substance type	“Advanced Therapy Medicinal Product”
Therapeutic area	“Cardiovascular Diseases”
Therapeutic indication	“Treatment of Danon disease”
Type of data supporting request	“Non-clinical + Clinical exploratory”

EMA PRIME designation: company announcement of 31 May 2023

“May 31, 2023 7:00 AM Eastern Daylight Time” (opens the source at this quote in a new tab)
“today announced that the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to RP-A501, the Company’s investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon Disease” (opens the source at this quote in a new tab)
“PRIME designation was granted based on positive safety and efficacy data from the Phase 1 clinical trial of RP-A501 in patients with Danon Disease and the potential of RP-A501 to meet the high unmet medical need in this population.” (opens the source at this quote in a new tab)
“PRIME designation offers the benefits of early and enhanced support from the EMA for the development of medicines that target unmet medical needs, as well as the opportunity for an accelerated review of the marketing application.” (opens the source at this quote in a new tab)

EU orphan designation EU/3/23/2826: designated 16 August 2023

“This medicine was designated as an orphan medicine for the treatment of Danon disease in the European Union on 16 August 2023.” (opens the source at this quote in a new tab)

Field	Quoted record
Active substance	“adeno-associated virus serotype 9 vector containing the human LAMP2 isoform B transgene”
Intended use	“Treatment of Danon disease”
Orphan designation status	“Positive”
EU designation number	“EU/3/23/2826”
Date of designation	“16/08/2023”
Sponsor	“Rocket Pharmaceuticals B.V.”

EU classification as an advanced therapy medicinal product (Committee for Advanced Therapies recommendation dated 15 June 2023)

Field	Quoted record
Public description of active substance	“Recombinant Adeno-associated virus serotype 9 vector containing the human-lysosome-associated membrane glycoprotein 2 isoform B transgene (AAV9.LAMP2B)”
Indication	“Treatment of Danon Disease”
Classification conclusion	“the EMA/CAT considers that the Product falls within the definition of a gene therapy medicinal product.”

EMA paediatric investigation plan: decision P/0004/2025 of 27 January 2025

Field	Quoted record
Decision number	“P/0004/2025”
PIP number	“EMEA-003639-PIP01-24”
Decision type	“P: decision agreeing on a investigation plan, with or without partial waiver(s) and or deferral(s)”
Decision date	“27/01/2025”
Condition or indication	“Treatment of Danon Disease”

“Having regard to the application submitted by Rocket Pharmaceuticals, B.V. on 29 April 2024 under Article 16(1) of Regulation (EC) No 1901/2006 also requesting a deferral under Article 20 of said Regulation and a waiver under Article 13 of said Regulation,” (opens the source at this quote in a new tab)

Designations held as the company lists them in 2026

“RP-A501 holds FDA RMAT, Fast Track, Rare Pediatric, and Orphan Drug designations in the U.S. along with ATMP and PRIME designations in the EU.” (opens the source at this quote in a new tab)

Clinical hold of May 2021 on the phase 1 trial (announced 10 May 2021): FDA request for protocol changes

“CRANBURY, NJ – May 10, 2021 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT)” (opens the source at this quote in a new tab)
“RP-A501 Phase 1 Trial for the Treatment of Danon Disease Placed on Clinical Hold To Address FDA Request For Additional Risk Mitigation Methods in Protocol; No New Safety Events Observed; Anticipate Delay in Enrollment by One Quarter” (opens the source at this quote in a new tab)
“The U.S. Food and Drug Administration (FDA) has requested the Company to pause patient dosing and modify the protocol and other supporting documents with revised guidelines for patient selection and management.” (opens the source at this quote in a new tab)
“No new drug-related safety events have been observed in the low- or high-dose adult cohorts of the Phase 1 trial.” (opens the source at this quote in a new tab)
“All follow-up study activities will continue and no additional data are requested.” (opens the source at this quote in a new tab)
“We have successfully treated five patients in the low- and high-dose adult cohorts.” (opens the source at this quote in a new tab)

Clinical hold of 2021: discontinuation of the higher dose by agreement with FDA (August 2021)

“No new drug-related safety events were observed in the low- or higher-dose adult cohorts; the previously disclosed SAE of thrombotic microangiopathy, which has since resolved, was reclassified as a SUSAR.” (opens the source at this quote in a new tab)
“In agreement with FDA, Rocket will no longer continue dosing patients at the higher dose (1.1e14).” (opens the source at this quote in a new tab)

Lifting of the 2021 hold: announced 16 August 2021

“Aug. 16, 2021 11:00 UTC” (opens the source at this quote in a new tab)
“today announces that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Company’s Phase 1 clinical trial of RP-A501 for the treatment of Danon Disease, allowing patient enrollment to resume.” (opens the source at this quote in a new tab)
“Rocket’s Danon Disease program was placed on clinical hold by the FDA in May of 2021 to modify the study protocol and other supporting documents with revised guidelines for patient selection and safety management.” (opens the source at this quote in a new tab)
“No new drug-related safety events were observed in the low-dose or higher-dose young adult cohorts as part of the clinical hold or during the hold.” (opens the source at this quote in a new tab)
“Additionally, given the activity observed among young adults in our low-dose cohort, in agreement with the FDA, we are now proceeding with the pediatric cohort.” (opens the source at this quote in a new tab)

Safety measures added to the protocols after the phase 1 high-dose experience

“Based on the initial efficacy observed in the low dose cohort and to mitigate complement-mediated safety concerns observed in the high dose cohort (related to thrombotic microangiopathy or TMA) and in agreement with the FDA, the Phase 2 study was initiated at the low dose (6.7e13 gc/kg).” (opens the source at this quote in a new tab)
“Additional safety measures were implemented and are reflected in the updated trial protocol for Phase 1 and the protocol for our ongoing pivotal Phase 2 study.” (opens the source at this quote in a new tab)

Clinical hold of 23 May 2025 on the phase 2 trial: announcement of 27 May 2025

“CRANBURY, NJ – May 27, 2025 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT)” (opens the source at this quote in a new tab)
“A patient participating in the Phase 2 pivotal trial of RP-A501 experienced an unexpected Serious Adverse Event (SAE).” (opens the source at this quote in a new tab)
“The SAE involved clinical complications related to a capillary leak syndrome.” (opens the source at this quote in a new tab)
“Upon learning of the initial event, Rocket voluntarily paused further dosing in the study.” (opens the source at this quote in a new tab)
“On May 23, 2025, the FDA placed a clinical hold on the trial to allow for further evaluation.” (opens the source at this quote in a new tab)
“Rocket is deeply saddened to report that this patient has since passed away after an acute systemic infection.” (opens the source at this quote in a new tab)

Clinical hold of 2025: root cause focus stated on 27 May 2025

“Rocket is conducting a comprehensive root cause analysis and remains in active dialogue with the U.S. Food and Drug Administration (FDA) and other key stakeholders, with the current focus being on the recent introduction of a novel immune suppression agent to the pre-treatment regimen that had been implemented to mitigate complement activation observed in some patients.” (opens the source at this quote in a new tab)
“This novel agent was specific to the AAV9-Danon program.” (opens the source at this quote in a new tab)
“While the clinical hold remains in place, the company is unable to provide guidance on the anticipated timing for completion of the Phase 2 trial.” (opens the source at this quote in a new tab)

Clinical hold of 2025: two participants with capillary leak syndrome and the conclusion of the investigation, as stated in 2026 filings

“In May 2025, two patients participating in the Phase 2 pivotal study of RP-A501 each experienced an unexpected SAE.” (opens the source at this quote in a new tab)
“The SAEs involved clinical complications related to a capillary leak syndrome resulting in multi-organ damage; one patient died as a result of these complications following an acute systemic infection.” (opens the source at this quote in a new tab)
“Rocket voluntarily paused further Phase 2 study dosing in the U.S. and EU, and the FDA subsequently placed the trial on clinical hold on May 23, 2025 to allow for further evaluation.” (opens the source at this quote in a new tab)

“In August 2025, the FDA lifted the clinical hold on the Phase 2 pivotal study following an investigation which concluded that the SAEs were likely the result of the combination of the C3 complement inhibitor introduced into the immunomodulation regimen and RP-A501.” (opens the source at this quote in a new tab)

Lifting of the 2025 hold: announced 20 August 2025

“CRANBURY, NJ – August 20, 2025 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT)” (opens the source at this quote in a new tab)
“today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Company’s pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease.” (opens the source at this quote in a new tab)
“In its correspondence, the FDA confirmed that Rocket satisfactorily addressed issues outlined in the clinical hold.” (opens the source at this quote in a new tab)
“To date, six patients with Danon disease have been treated in the Phase 2 study with RP-A501.” (opens the source at this quote in a new tab)

Conditions of resumption after the 2025 hold: recalibrated dose, sequential dosing, and revised immunomodulation

“The FDA authorized the pivotal study to resume first with a recalibrated dose of 3.8 x 10¹³ GC/kg of RP-A501 in three patients, treated sequentially with a minimum four-week interval between each treatment.” (opens the source at this quote in a new tab)
“The revised regimen discontinues prophylactic use of a C3 complement inhibitor, while maintaining sirolimus, rituximab, and steroids.” (opens the source at this quote in a new tab)
“Additionally, the protocol will specify a lower threshold for administering a C5 inhibitor (eculizumab) in response to impending complement activation.” (opens the source at this quote in a new tab)
“Further updates about the Phase 2 study can be expected following review of data from the next three patients.” (opens the source at this quote in a new tab)

Resumption of dosing after the 2025 hold: guidance of November 2025 and status in May and August 2026

“Dosing of additional patients for the Phase 2 study of RP-A501 for Danon disease anticipated in the first half of 2026.” (opens the source at this quote in a new tab)

“Pivotal Phase 2 trial of RP-A501 for Danon disease progressing with dosing reinitiated” (opens the source at this quote in a new tab)

“Treatment proceeded sequentially, with a minimum of four weeks between infusions.” (opens the source at this quote in a new tab)
“To date, no thrombotic microangiopathy (TMA), capillary leak syndrome, or other significant safety concerns have been observed in these patients.” (opens the source at this quote in a new tab)

FDA end-of-phase 1 meeting: registration path announced 22 December 2022

“CRANBURY, N.J. – Dec. 22, 2022 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT)” (opens the source at this quote in a new tab)
“today announces updates from the Company's end-of-Phase 1 meeting with the U.S. Food and Drug Administration (FDA) regarding RP-A501, the Company's investigational adeno-associated virus (AAV)-based gene therapy for Danon Disease.” (opens the source at this quote in a new tab)
“Based on ongoing FDA discussion, Rocket to pursue a biomarker-based composite endpoint for a single arm, open-label pivotal trial with a natural history comparator” (opens the source at this quote in a new tab)
“The FDA has also expressed an openness to considering a biomarker-based composite endpoint supported by functional and quality-of-life assessments as measures of patient benefit.” (opens the source at this quote in a new tab)

FDA alignment of September 2023 on the pivotal design (original design at 6.7 × 10¹³ GC/kg, later modified)

“CRANBURY, N.J. – September 12, 2023 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT)” (opens the source at this quote in a new tab)
“today announced that alignment has been reached with the Food and Drug Administration (FDA) on the global Phase 2 pivotal trial of RP-A501 for Danon Disease.” (opens the source at this quote in a new tab)
“12 patient study with primary endpoint assessment at 12 months to support accelerated approval” (opens the source at this quote in a new tab)
“To support accelerated approval, the study will assess the efficacy of RP-A501 as measured by the biomarker-based co-primary endpoint consisting of improvements in LAMP2 protein expression (≥ Grade 1, as measured by immunohistochemistry), and reductions in left ventricular (LV) mass.” (opens the source at this quote in a new tab)
“These endpoints could support a full approval with longer-term follow-up.” (opens the source at this quote in a new tab)

FDA alignment of September 2026 on completing the pivotal trial

“CRANBURY, N.J. – September 15, 2026 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT)” (opens the source at this quote in a new tab)
“today announced alignment with the U.S. Food and Drug Administration (FDA) on continued enrollment and dosing and key elements of the global pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease, including the recalibrated dose, 12-patient pivotal population and established 12-month co-primary endpoints.” (opens the source at this quote in a new tab)
“Following its review of the available safety data, the FDA confirmed continued enrollment and dosing under the modified protocol at the recalibrated dose.” (opens the source at this quote in a new tab)
“The FDA also confirmed that the pivotal study population will include 12 male patients treated with RP-A501 at the recalibrated dose of 3.8 × 10¹³ genome copies per kilogram (GC/kg) using commercial-grade product.” (opens the source at this quote in a new tab)

Accelerated approval path: endpoints confirmed by FDA in September 2026

“The primary assessment will occur at 12 months using the previously established co-primary endpoints of myocardial LAMP2 protein expression and a 10% reduction from baseline in left ventricular mass index.” (opens the source at this quote in a new tab)
“These endpoints are intended to support a potential accelerated approval pathway.” (opens the source at this quote in a new tab)

“To support accelerated approval, co-primary endpoint consisting of improvements in LAMP2 protein expression (≥ Grade 1 from baseline) and reductions in Left Ventricular Mass (LVMI; ≥10% ↓) at 12-month post-infusion” (opens the source at this quote in a new tab)
“FDA agreement obtained to support completion of pivotal study” (opens the source at this quote in a new tab)

Efficacy success criterion for the pivotal cohort (October 2026)

“At least 7 of 12 responders at 12 months” (opens the source at this quote in a new tab)
“Each responder must meet both criteria” (opens the source at this quote in a new tab)

“Trial success requires at least seven patients to meet both response criteria at 12 months: myocardial LAMP2 protein expression of at least Grade 1 and a reduction in left ventricular mass index of at least 10% from baseline.” (opens the source at this quote in a new tab)

Company timeline as of October 2026: dosing completion mid-2027 and topline results mid-2028

“9 additional patients in Cohort 4” (opens the source at this quote in a new tab)
“MID-2027 Dosing completion” (opens the source at this quote in a new tab)
“MID-2028 TOPLINE TARGET” (opens the source at this quote in a new tab)
“Primary efficacy readout from pivotal Cohort 4” (opens the source at this quote in a new tab)
“MID-2028 target is provisional pending alignment with dosing completion and 12-month follow-up.” (opens the source at this quote in a new tab)

“Rocket expects to complete pivotal trial dosing by mid-2027, with topline results anticipated mid-2028.” (opens the source at this quote in a new tab)

Company caveats on the approval pathway (October 2026)

“FDA’s assessment would consider the complete application, including safety, efficacy, manufacturing and product-quality information.” (opens the source at this quote in a new tab)
“Preliminary findings may change as additional data become available, and results from earlier cohorts may not predict outcomes at the recalibrated dose under the modified protocol.” (opens the source at this quote in a new tab)

“the timing and outcome of regulatory interactions, submissions and review, including whether the FDA will determine that the available evidence supports accelerated approval or approval through another pathway, and any requirements for additional studies or post-approval confirmatory trials” (opens the source at this quote in a new tab)

Priority review voucher: eligibility stated by the company

“PRV eligible; Accelerated Approval; Priority review” (opens the source at this quote in a new tab)

“A rare pediatric disease designation does not guarantee that a sponsor will receive a PRV upon approval of its BLA.” (opens the source at this quote in a new tab)
“However, a marketing application for these product candidates, if approved, may not meet the eligibility criteria for a rare pediatric disease priority review voucher.” (opens the source at this quote in a new tab)

Priority review voucher: sunset date of the rare pediatric disease voucher program

“As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029.” (opens the source at this quote in a new tab)
“FDA may not award any PRVs under this program after September 30, 2029.” (opens the source at this quote in a new tab)

Priority review voucher: sale of the voucher issued with the approval of Kresladi (company precedent)

“On April 26, 2026, the Company entered into a definitive agreement to sell its PRV that was originally issued in connection with the FDA’s approval of the BLA for KRESLADI™ for $180 million.” (opens the source at this quote in a new tab)

License from the University of California, San Diego (February 2017)

“In February 2017, we entered into a license agreement with The Regents of the University of California, represented by its San Diego campus, under which UCSD granted us an exclusive, sublicensable, worldwide license to certain intellectual property rights for the treatment of lysosomal storage diseases, including DD.” (opens the source at this quote in a new tab)
“We paid an upfront license fee of $0.05 million and are obligated to make aggregate milestone payments of up to $1.5 million to UCSD upon the achievement of specified development and regulatory milestones for the treatment of DD.” (opens the source at this quote in a new tab)
“With respect to any commercialized products covered by the agreement, we are obligated to pay a low single digit percentage royalty on net sales, subject to specified adjustments.” (opens the source at this quote in a new tab)

License from REGENXBIO for the NAV AAV9 vector (November 2018)

“On November 19, 2018, we entered into a license agreement with RGNX, pursuant to which we obtained an exclusive license for all U.S. patents and patent applications related to RGNX’s NAV AAV-9 vector for the treatment of DD in humans by in vivo gene therapy using AAV-9 to deliver any known LAMP2 transgene isoforms and all possible combinations of LAMP2 transgene isoforms, as well as an exclusive option to license all U.S. patents and patent applications for two additional NAV AAV vectors.” (opens the source at this quote in a new tab)
“The license agreement provides for royalties payable to RGNX in the high-single digits to low-teens on net sales levels of licensed products incorporating the licensed patents during the royalty term.” (opens the source at this quote in a new tab)
“If successful, we will be required to make milestone payments to RGNX of up to $13.0 million for each licensed product upon the achievement of specified clinical development and regulatory milestones in the U.S. and EU.” (opens the source at this quote in a new tab)
“In addition, we shall pay RGNX 20% of the payment fees received from a priority review voucher issued in connection with or otherwise related to a licensed product.” (opens the source at this quote in a new tab)
“We paid a $1.0 million license fee payment under the RGNX agreement upon the dosing of the first DD patient in 2019 and a $2.0 million license fee payment upon initiation of a Phase 2 pivotal trial in 2023.” (opens the source at this quote in a new tab)

“Under the terms of the agreement, REGENXBIO has granted Rocket an exclusive, worldwide license, with rights to sublicense, to REGENXBIO’s NAV AAV9 vector for the development and commercialization of gene therapy treatments for Danon disease.” (opens the source at this quote in a new tab)

July 2025 reorganization: resources concentrated on the cardiovascular AAV programs, including Danon disease

“The restructuring and reprioritization initiative focuses Rocket's resources on the adeno-associated virus (AAV) cardiovascular platform, comprised of clinical programs in Danon disease, PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM), and BAG3-associated dilated cardiomyopathy (BAG3-DCM), as well as submitting the completed responses to the CRL for KRESLADITM (formerly RP-L201) for severe leukocyte adhesion deficiency-I (LAD-I).” (opens the source at this quote in a new tab)
“The company will implement a reduction in workforce of approximately 30%, which, along with other planned cost-saving initiatives, is expected to reduce Rocket’s 12-month operating expenses by nearly 25%.” (opens the source at this quote in a new tab)

“As part of this strategic realignment, we de-prioritized further development activities related to our FA and PKD programs and implemented a workforce reduction of approximately 30%.” (opens the source at this quote in a new tab)

Funding of the program: cash runway stated in October 2026

“The facility strengthens Rocket’s financial position and provides access to additional capital to support execution of its pivotal Phase 2 study of RP-A501 for Danon disease and continued advancement of its broader cardiovascular pipeline.” (opens the source at this quote in a new tab)
“Based on its current operating plan, Rocket expects these resources, together with the initial $35 million received under the facility, to fund planned operations into the third quarter of 2028.” (opens the source at this quote in a new tab)
“An additional $30 million may become available upon achievement of a specified Danon clinical milestone, and an additional $50 million is subject to Hercules’ investment committee approval.” (opens the source at this quote in a new tab)

“Current cash runway now into Q3’28” (opens the source at this quote in a new tab)

Funding of the program: loan terms tied to FDA approval of a BLA for RP-A501

“may be further extended to October 1, 2030 if Borrower additionally achieves an approval milestone relating to FDA approval of a Biologics License Application for RP-A501 for the treatment of Danon Disease” (opens the source at this quote in a new tab)

Funding of the program: California Institute for Regenerative Medicine grant

“On August 18, 2024, CIRM awarded the Company up to $5.8 million under a CLIN2 grant award to support the clinical development of its AAV-based genetic medicines, RP-A501 for the treatment of DD.” (opens the source at this quote in a new tab)
“Through June 30, 2026, the Company has received total RP-A501 grants of $5.0 million from CIRM.” (opens the source at this quote in a new tab)