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Rocket Pharmaceuticals develops gene therapies for severe and rare inherited diseases. It uses an AAV (Adeno-Associated Virus) platform to deliver corrected genes directly into patients, with the goal of producing a therapeutic protein in targeted cells of the heart, liver, eye, or central nervous system to modify the disease. The therapy work involves engineering AAV constructs so the corrected gene is expressed in diseased cells. The company aims to create first-in-class or best-in-class treatments and generates revenue through partnerships with larger pharma companies, grants, and potential future sales of approved therapies. Core values include Trust, Generosity, Curiosity, and Elevation, guiding a patient-centric approach that puts families first and supports ongoing scientific advancement. Overall goal: provide life-changing gene therapies for patients with few options and expand treatment options for rare genetic disorders.
Industries
Biotechnology
Healthcare
Company Size
201-500
Company Stage
IPO
Headquarters
Cranbury Township, New Jersey
Founded
2015
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Total Funding
$726.5M
Above
Industry Average
Funded Over
11 Rounds
Rocket Pharmaceuticals receives FDA approval for KRESLADI gene therapy. Alexandra Moore Published Mar 30, 2026 - 04:18 Smallest Font Largest Font Rocket Pharmaceuticals recently announced a significant regulatory achievement, as the U.S. Food and Drug Administration (FDA) granted approval for KRESLADI. This gene therapy targets a rare pediatric disorder, marking the company's inaugural marketed product and a pivotal regulatory success after an extended review process, as reported by Detik Finance. This approval is particularly notable as KRESLADI becomes the first FDA-sanctioned gene therapy for this specific condition. The development positions Rocket Pharmaceuticals to establish a commercial presence within the ultra-rare disease market. Beyond the product approval itself, Rocket Pharmaceuticals also secured a Rare Pediatric Disease Priority Review Voucher. This asset offers biotech companies a valuable monetization opportunity. Management is actively exploring various strategic options for the voucher to bolster the company's financial flexibility. The company further stated its intentions to make KRESLADI accessible to eligible patients across the United States. Rocket Pharmaceuticals has indicated a controlled, rather than widespread, commercial rollout for KRESLADI. Recent company disclosures describe a tailored commercial strategy designed to address the very limited patient population. Reuters previously reported that the launch is anticipated to commence through a select number of specialized treatment centers. As of December 31, 2025, Rocket Pharmaceuticals held $188.9 million in cash, cash equivalents, and investments. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) operates as a biotechnology firm dedicated to developing genetic therapies for various rare disorders. Its research pipeline encompasses both lentiviral and AAV-based programs, which are aimed at addressing immunological and cardiovascular diseases.
Rocket Pharmaceuticals has received FDA accelerated approval for KRESLADI, a gene therapy for pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) without an available matched sibling donor for transplant. The approval is based on increased neutrophil CD18 and CD11a surface expression, with continued approval contingent on verification of clinical benefit. LAD-I is an ultra-rare genetic disease affecting approximately one in 100,000 to 200,000 live births in the US, with two-thirds classified as severe. Patients suffer from recurrent, life-threatening bacterial and fungal infections requiring frequent hospitalisation. The FDA granted Rocket a Rare Pediatric Disease Priority Review Voucher, which the company intends to monetise. KRESLADI is now available to eligible patients in the United States through www.KRESLADI.com.
Rocket Pharmaceuticals has signed an equity sales agreement with Cantor Fitzgerald & Co for an At-the-Market offering programme worth up to $100 million, according to a filing with the US Securities and Exchange Commission. The agreement allows the clinical-stage biotechnology company to issue and sell common stock to Cantor Fitzgerald based on market conditions. The ATM mechanism enables listed companies to raise capital in batches according to funding needs, helping optimise financing timing whilst reducing stock price impact. The financing will support Rocket Pharmaceuticals' development of gene therapies for rare diseases. Details of the agreement were filed via Form 8-K with the SEC.
Rocket Pharmaceuticals (RCKT) launches $100M at-the-market stock offering with Cantor. Filing Impact (Moderate) Filing Sentiment Rhea-AI Filing summary. Rocket Pharmaceuticals entered into a new sales agreement with Cantor Fitzgerald & Co. to establish an at-the-market stock offering program. Under this arrangement, the company may, at its discretion, sell shares of its common stock with an aggregate offering price of up to $100,000,000 through Cantor as sales agent. The shares will be issued under an existing shelf registration statement on Form S-3, supported by a prospectus supplement filed the same day. Cantor will sell the shares in transactions deemed at-the-market on Nasdaq, earning a cash commission of up to 3.0% of gross proceeds. Rocket can suspend or terminate sales at any time, and the program ends once all shares are sold or the agreement is otherwise terminated. In connection with this new arrangement, Rocket terminated its prior at-the-market sales agreement with Cowen and Company, LLC. Legal opinions and the full sales agreement are filed as exhibits, providing the formal framework for potential future equity issuance. 03/10/2026 - 05:24 PM Faq. What did Rocket Pharmaceuticals (RCKT) announce in this 8-K filing? Rocket Pharmaceuticals established a new at-the-market stock offering program with Cantor Fitzgerald, allowing it to sell up to $100,000,000 of common stock over time, using its existing Form S-3 shelf registration and a new prospectus supplement. How much stock can Rocket Pharmaceuticals (RCKT) sell under the new at-the-market program? Rocket Pharmaceuticals may sell common stock with an aggregate offering price of up to $100,000,000. Sales will occur from time to time at the company's discretion through Cantor Fitzgerald & Co. as sales agent under the sales agreement. Who is the sales agent for Rocket Pharmaceuticals' new at-the-market offering? Cantor Fitzgerald & Co. will act as sales agent for Rocket Pharmaceuticals. It will use commercially reasonable efforts to sell shares in at-the-market transactions on Nasdaq or other markets, consistent with its normal trading and sales practices and applicable regulations. What commission will Cantor Fitzgerald receive in Rocket Pharmaceuticals' ATM program? Cantor Fitzgerald is entitled to a cash commission of up to 3.0% of gross proceeds from any Rocket Pharmaceuticals common stock sold under the at-the-market program, plus reimbursement of certain expenses and customary indemnification and contribution protections. What happened to Rocket Pharmaceuticals' prior at-the-market agreement? Upon entering the new sales agreement with Cantor Fitzgerald, Rocket Pharmaceuticals terminated its prior at-the-market sales agreement with Cowen and Company, LLC, which had allowed Cowen to act as agent for periodic common stock sales. Under which registration statement will Rocket Pharmaceuticals' ATM shares be issued? The shares sold through the at-the-market program will be issued under Rocket Pharmaceuticals' shelf registration statement on Form S-3, File No. 333-293925, with a related prospectus supplement filed to cover sales under the Cantor sales agreement. Filing exhibits & attachments. 5 documents Agreements & contracts.
Rocket Pharmaceuticals reported a fourth-quarter loss of 38 cents per share, narrower than the Zacks Consensus Estimate of a loss of 42 cents and improved from a loss of 62 cents in the year-ago quarter. The company recorded no revenues as it has no marketed products. Research and development expenses fell 21.6% year over year to $29.3 million, reflecting disciplined spending following recent restructuring. The company held $188.9 million in cash and investments as of 31 December 2025, expected to fund operations into the second quarter of 2027. The FDA is reviewing Rocket's biologics licence application for gene therapy Kresladi to treat severe leukocyte adhesion deficiency-I, with a decision expected on 28 March 2026.
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Industries
Biotechnology
Healthcare
Company Size
201-500
Company Stage
IPO
Headquarters
Cranbury Township, New Jersey
Founded
2015
Find jobs on Simplify and start your career today