Rocket Pharmaceuticals retracts BLA submission for Fanskya
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Oct 03 2025
0mins
Should l Buy RCKT?
Withdrawal of Biologics License Application: Rocket Pharmaceuticals has voluntarily withdrawn its Biologics License Application for the investigational gene therapy RP-L102, also known as "Fanskya," for treating Fanconi Anemia, as part of a strategic decision to focus on programs with clearer regulatory pathways.
Future Opportunities and Safety Profile: The withdrawal is based on business considerations and does not indicate safety or efficacy concerns, as RP-L102 has shown a favorable risk-benefit profile. The company is open to exploring external partnerships to advance the therapy in the future.
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Analyst Views on RCKT
Wall Street analysts forecast RCKT stock price to rise
11 Analyst Rating
6 Buy
4 Hold
1 Sell
Moderate Buy
Current: 3.250
Low
5.00
Averages
8.63
High
16.00
Current: 3.250
Low
5.00
Averages
8.63
High
16.00
About RCKT
Rocket Pharmaceuticals, Inc. is a fully integrated, late-stage biotechnology company focused on the development of gene therapies for rare and devastating diseases. The Company has two clinical stage and one pre-clinical stage in vivo adeno-associated viral (AAV) programs in the United States, which include programs for Danon disease (DD), a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy (PKP2-ACM), an inheritable cardiac disorder that is characterized by a progressive loss of cardiac muscle mass, severe right ventricular dilation, dysplasia, fibrofatty replacement of the myocardium and a high propensity to arrhythmias and sudden death (RP-A601), and BAG3 Dilated Cardiomyopathy (BAG3-DCM), which is the most common form of cardiomyopathy and is characterized by progressive thinning of the walls of the heart resulting in enlarged heart chambers that are unable to pump blood.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
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- Loan Characteristics: The trust's loan pool comprises 5,839 fully amortizing, fixed-rate mortgages with average terms of 10 years (4.1%), 15 years (7.8%), 20 years (62.7%), and 30 years (25.5%), reflecting a diverse range of loan term options.
- Rating Methodology: KBRA employs its Residential Asset Loss Model (REALM) for loan-level analysis of the mortgage pool, supplemented by third-party due diligence results and cash flow modeling analysis, ensuring the accuracy and reliability of the ratings, thereby enhancing market confidence.
- Legal Structure Review: During the rating process, KBRA also assessed the legal structure and documentation of the transaction, ensuring compliance of all key transaction parties, which reduces investment risk and increases investor trust.
See More
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- Strengthened Clinical Validation: Avant Technologies announced that a new study from the Mayo Clinic reinforces the critical role of α-Klotho in vascular health, providing scientific backing for its Klothonova therapy developed in partnership with Singapore's Austrianova, which is expected to enhance market acceptance of the therapy.
- Significant Market Potential: The precision medicine market is projected to grow from $119 billion in 2025 to $471 billion by 2034, and Avant's technological innovations align perfectly with this trend, positioning the company to capture a significant share of the future market.
- Deepened Strategic Collaboration: The exclusive global licensing agreement with Klothea Bio grants Klothonova rights to develop and commercialize Klotho-producing cells, leveraging Austrianova's technology, which is anticipated to accelerate the product's market entry.
- Diversified Investment Strategy: Avant is also collaborating with SGAustria to provide funding over the next eight months for diabetes treatment, demonstrating its strategic intent to expand across multiple therapeutic areas.
See More
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- Strengthened Clinical Validation: Avant Technologies announced that a new study from the Mayo Clinic reinforces the critical role of the 'longevity protein' α-Klotho in vascular health, bolstering the scientific foundation for its joint venture Klothonova with Singapore's Austrianova, advancing cell therapy development.
- Significant Market Potential: As precision medicine expands, the sector is projected to grow from $119 billion in 2025 to $471 billion by 2034, positioning Avant Technologies at the forefront of this transformation, particularly in rare disease treatments.
- Innovative Technology: Klothonova's cell therapy aims to sustainably restore α-Klotho levels using genetically modified human cells, which is expected to revolutionize cardiovascular health and overall longevity, addressing the increasing market demand.
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See More
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- Loan Characteristics: The transaction consists of 5,485 newly originated closed-end second lien mortgages, with an average seasoning of less than one month, reflecting confidence and liquidity in the market for new loans.
- Interest Rate Structure: The mortgage pool features a diverse interest rate structure with fixed-rate loans of 4.0% for 10-year, 8.3% for 15-year, 65.0% for 20-year, and 22.6% for 30-year terms, catering to various borrower needs and enhancing market appeal.
- Rating Methodology: KBRA's rating approach involved loan-level analysis through its Residential Asset Loss Model (REALM), incorporating third-party due diligence results and cash flow modeling, ensuring the accuracy and reliability of the ratings, which bolsters market confidence in the transaction.
See More
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Other Stocks Declining: Several other stocks, including Alpha Technology Group Ltd and Invivyd Inc, experienced significant declines in pre-market trading, with drops of 19% and 13.4%, respectively.
Home Depot's Performance: Home Depot's shares fell 2% after reporting worse-than-expected third-quarter adjusted EPS and lowering its FY25 adjusted EPS outlook.
See More
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Quarterly Loss: Rocket Pharmaceuticals reported a third-quarter loss of $50.33 million, or $0.45 per share, an improvement from a loss of $66.72 million, or $0.71 per share, in the same quarter last year.
Analyst Expectations: The company's loss per share beat analysts' expectations, which had forecasted a loss of $0.52 per share, excluding special items.
See More
