Rocket Pharmaceuticals Future Growth
Future criteria checks 2/6
Rocket Pharmaceuticals is forecast to grow earnings and revenue by 31.2% and 58.7% per annum respectively. EPS is expected to grow by 39.2% per annum. Return on equity is forecast to be -54.5% in 3 years.
Key information
31.2%
Earnings growth rate
39.19%
EPS growth rate
| Biotechs earnings growth | 25.2% |
| Revenue growth rate | 58.7% |
| Future return on equity | -54.47% |
| Analyst coverage | Good |
| Last updated | 12 May 2026 |
Recent future growth updates
Recent updates
Seeking Alpha • Dec 26
Rocket Pharmaceuticals: Deep Dislocation Offers Asymmetric Upside On LAD-I Approval And Amended Danon Program
Summary Rocket Pharmaceuticals is rated Buy with a $17 price target, reflecting a deep disconnect between intrinsic value and current valuation. RCKT's bullish thesis hinges on a high-probability LAD-1 approval in March 2026 and the pivotal Danon's disease trial resuming with an amended, safer protocol. Risk-adjusted DCF values Danon's and LAD-1 programs at ~$700M, far exceeding the current $151M EV, even under conservative or bear-case assumptions. Near-term catalysts include LAD-1's PDUFA date and potential $150M PRV, providing non-dilutive capital and extending cash runway into 2027. Read the full article on Seeking AlphaAnalysis Article • Dec 09
We're Keeping An Eye On Rocket Pharmaceuticals' (NASDAQ:RCKT) Cash Burn Rate
There's no doubt that money can be made by owning shares of unprofitable businesses. For example, although...Analysis Article • Aug 21
Will Rocket Pharmaceuticals (NASDAQ:RCKT) Spend Its Cash Wisely?
NasdaqGM:RCKT 1 Year Share Price vs Fair Value Explore Rocket Pharmaceuticals's Fair Values from the Community and...Analysis Article • Apr 02
We're Keeping An Eye On Rocket Pharmaceuticals' (NASDAQ:RCKT) Cash Burn Rate
There's no doubt that money can be made by owning shares of unprofitable businesses. For example, although...Seeking Alpha • Feb 12
Rocket Pharma Is A Buy At These Prices With Promising Data
Summary Rocket Pharmaceuticals, Inc.'s RP-L201 for LAD-I faces FDA rejection but shows promising data; potential approval could unlock a $350M market. RP-L102 for Fanconi Anemia and RP-A501 for Danon Disease show strong clinical data, indicating significant market potential. Financially, Rocket Pharma has a limited cash runway of 2–4 quarters, dependent on FDA approvals and successful public offerings. Despite risks, RCKT's low current price and promising pipeline make it an attractive buy, with potential markets exceeding $1B. Read the full article on Seeking AlphaSeeking Alpha • Dec 02
Rocket Pharmaceuticals: Not Bad Valuation But Reflexivity Risks Remain
Summary Rocket Pharmaceuticals' gene therapy treatments for rare diseases show promise, but high cash burn and potential dilution pose significant risks for investors. The company's Danon disease treatment, RP-A501, is a key focus, with promising Phase I results and progression to Phase II. Valuation is complicated by high costs of capital and reflexivity risk, with the company needing more funding in just over a year. Despite attractive valuation relative to peers, uncertainties in market penetration and reimbursement remain significant concerns in addition to reflexivity. We don't think the environment lends itself yet to plays like these. Read the full article on Seeking AlphaAnalysis Article • Sep 23
Will Rocket Pharmaceuticals (NASDAQ:RCKT) Spend Its Cash Wisely?
There's no doubt that money can be made by owning shares of unprofitable businesses. For example, biotech and mining...Seeking Alpha • Sep 15
Rocket Pharmaceuticals: 2 Gene Therapy Candidates Anticipating Near-Term Review
Summary Rocket's ~$1.7 billion market cap is not supported by its financial metrics. KRESLADI has endured several FDA delays but will likely see a new BLA filing in the near future. RP-L102, Rocket's gene therapy in treatment of Fanconi Anemia, will likely have a BLA filing in Q4, 2024. Read the full article on Seeking AlphaSeeking Alpha • Jun 28
Rocket Pharmaceuticals: CRL For Kresladi Unwelcome, But Likely A Quick Fix
Summary Rocket Pharmaceuticals stock dropped pre-market after management reported receiving a Complete Response Letter from the FDA in relation to Kresladi, indicated for severe leukocyte adhesion deficiency-I. The rejection was based on Chemistry Manufacturing and Controls issues, which can typically be addressed quickly, suggesting a good chance of approval upon resubmission. Rocket's delay in approval for Kresladi should not impact its overall investment opportunity, with a diverse pipeline and potentially valuable assets in its portfolio. The key to the company's fortunes is its in vivo therapy targeting Danon Disease. Unless the CMC issues identified by the FDA seriously affect Rocket's manufacturing plant, causing delays, today's setback should not be viewed as too problematic. Read the full article on Seeking AlphaAnalysis Article • Jun 09
We're Hopeful That Rocket Pharmaceuticals (NASDAQ:RCKT) Will Use Its Cash Wisely
Just because a business does not make any money, does not mean that the stock will go down. For example, although...Seeking Alpha • Apr 05
Rocket Pharmaceuticals: Cautiously Optimistic (Rating Upgrade)
Summary Rocket Pharmaceuticals, Inc. has potential FDA approvals for its gene therapies on the horizon. The company focuses on developing gene therapies for rare diseases, targeting a market that is expected to triple in value by the end of the decade. Rocket's pipeline includes promising therapies for severe leukocyte adhesion deficiency-I, Danon Disease, and Fanconi Anemia, among others. An updated analysis around Rocket Pharmaceuticals follows in the paragraphs below. Read the full article on Seeking AlphaSeeking Alpha • Mar 01
Rocket Pharmaceuticals' Promising Path In Gene Therapy
Summary Rocket Pharmaceuticals, Inc. stock surged 76% post a "Buy" recommendation, fueled by positive Phase 2 trial news for RP-A501 in Danon disease. RP-A501, targeting a rare disease without current treatments, promises significant market potential, pending trial outcomes and approval. Financial analysis shows Rocket with a strong liquidity position and a 25-month cash runway, despite a notable monthly cash burn. I recommend a "Buy" on Rocket due to its unique RP-A501 market position, financial health, and robust market sentiment, with caution advised. Read the full article on Seeking AlphaAnalysis Article • Nov 07
Companies Like Rocket Pharmaceuticals (NASDAQ:RCKT) Are In A Position To Invest In Growth
Even when a business is losing money, it's possible for shareholders to make money if they buy a good business at the...Seeking Alpha • Oct 03
Rocket Pharmaceuticals slides over 6% after hours on $100M stock offering
Rocket Pharmaceuticals (NASDAQ:RCKT) stock slid 6.5% postmarket on Monday after the firm announced a $100M underwritten public stock offering. RCKT intends to grant underwriters a 30-day option to buy up to an additional $15M of shares. Shares of RCKT have fallen 34.5% YTD.Seeking Alpha • Aug 25
Rocket Pharmaceuticals: Updates To Thesis, Clinical Momentum Continues To Accelerate
Summary Shares have lost 15% year to date. Company wisely adopted a platform-agnostic approach to address first-in-class opportunities across the gene therapy pipeline. Danon disease presents their most lucrative opportunity, with conservative peak sales potential exceeding $2 billion. Promising data sets across all four programs with clear paths to market reflects leadership's execution and experience in this space. RCKT is a Buy and only appropriate for long-term investors. Key risks include regulatory setbacks, delays in the clinic, adverse safety events and more dilution within a year. Platform-agnostic gene therapy pioneer Rocket Pharmaceuticals (RCKT) gave us a 200% to 316% gain in ROTY's model portfolio the first time we owned this name, cutting it loose when the share price spiked after initial data for the Danon disease program in late 2020 to early 2021. From the peak above $60, shares have lost over two-thirds of their value as the company certainly took a hit along with the rest of the biotech sector during the recent downturn. Pessimism took a hold here in part due to safety concerns, both for the overall gene therapy space (patient deaths including for Novartis' Zolgensma and Astellas' AT132 for XLMTM (liver failure, sepsis & GI bleeding) as well as for the Danon program at the higher (discontinued) dose. As data across all four clinical programs continues to read positive and pipeline momentum accelerates, I wish to revisit this one for readers to determine upside prospects for the years ahead. Chart FinViz Figure 1: RCKT weekly chart (Source: Finviz) When looking at charts, clarity often comes from taking a look at distinct time frames in order to determine important technical levels and get a feel for what's going on. In the weekly chart above, we can see shares trend upward in 2020 as Rocket reported positive data sets across multiple gene therapy programs (albeit with smaller market potential). From there, in December there was a parabolic spike in share price after promising initial data was presented for the first-in-class Danon disease program (blockbuster potential indication). As the euphoria dissipated, share price steadily declined due to wider concerns surrounding viability for gene therapy companies as well as specific toxicity issues for high dose of Danon program. They bottomed at the $10 level and since have rebounded to the high teens. My initial take is that long term investors who are willing to hold patiently will do well to accumulate dips to the $15 level (20-day moving average). Overview In my prior writeup, I touched on the following keys to a bullish thesis: Management appeared quite optimistic on the firm's prospects, talking up their first-mover advantage and pursuit of a variety of best-in-class indications. Corporate Slides Figure 2: Pipeline (Source: corporate presentation) The first opportunity started with Fanconi Anemia. RP-L102 is a lentiviral vector-based gene therapy candidate and I pointed out that there's no cure for this disease, which is caused by FANC gene mutations impairing DNA repair function. This in turn leads to bone marrow failure and later on down the road cancer. Presently patients are treated with HSCT (hematopoietic stem cell transplantation), which unfortunately comes with the risk of graft-versus-host disease. Prevalence was noted to be around 2,000 patients in the United States and Europe (currently 30% to 40% of these patients receive a transplant), of which 250 per year could be treated by RP-L102. Lastly, I noted that via pivotal studies the company would shoot for accelerated approval on the basis of prevention of bone marrow failure. So far, safety looked highly favorable positioning RP-L102 as a first-line treatment option (no signs of dysplasia). Additionally, efficacy was quite good including 6 of 9 patients showing preliminary signs of engraftment and 5 of 9 showed increasing evidence of engraftment with BM MMC resistance ranging from 51%-94% and ≥20% at two consecutive timepoints. Corporate Slides Figure 3: Increasing phenotypic correction over 1 to 2 years after treatment with RP-L102 (Source: corporate presentation) As for other assets of interest, I touched on their LAD-I (Leukocyte Adhesion Deficiency-I) and PKD (Pyruvate Kinase Deficiency) programs. The asset for IMO (Infantile Malignant Osteopetrosis) drug candidate was discontinued, unfortunately. LAD-I market opportunity was quite small (perhaps $25M to $50M), but it's hard to understate patient impact for this monogenic immunodeficiency as over 50% of patient shave the severe phenotype (60% to 75% mortality by age 2). As with Fanconi, the currently available treatment is allogeneic hematopoietic stem cell transplant which is associated with significant graft failure and acute GvHD. Estimated market is just 25 to 50 patients with potential for up to 100 in expansion to the moderate LAD-I population. Data was a homerun, with 100% engraftment and CD18 constitution across all patients (CD18+ PMN expression ranging from ~19.6% to ~87.4%, keeping in mind that levels over 10% are associated with survival for decades). There was evidence of spontaneous resolution of disease-related rash, restoration of wound repair capabilities, 100% overall survival one year post-treatment and significant reduction in disease-related hospitalizations and severe infections in all 9 of 9 patients. Corporate Slides Figure 4: LAD-I data was a homerun as reflected in improvement in clinical outcomes (Source: corporate presentation) As for RP-L301 in Pyruvate Kinase Deficiency ((PKD)), I noted that this was the largest of the lentiviral indications with ~250-500 patients/year for transfusion-dependent post-splenectomy patients and 3,000 to 8,000 total patients in the US, Europe and rest of world combined. Data here was only in a couple patients, but they were able to show marked hemoglobin improvement from low single digit levels to 13.0-14.8 g/dL and no transfusions required following engraftment. Importantly, there were no IP-related serious adverse events and they have commercial grade product along with centralized testing available for all patients. As for my favorite opportunity in Danon disease (the crown jewel of the pipeline), this is a monogenic heart failure syndrome that affects multiple systems of the body and is caused by X-linked dominant LAMP2 mutations. Progressive cardiomyopathy is the main cause of early death in these young male patients where median overall survival is just 19 years. For females, prognosis is better as presentation is generally delayed due to possession of an additional X chromosome. One would simply think heart transplant is the logical solution, but unfortunately these are associated with substantial morbidity and mortality (10 year overall survival of just 50%). Also, only 20% of these patients are able to get them. Market opportunity is outsized here at estimated US + EU prevalence of 15,000 to 30,000 patients. If I use very conservative numbers (5,000 patients and $500,000 price tag), that still equates to a $2.5 billion market opportunity. I noted that initial data was a landmark moment as they were able to achieve greater than 50% of normal LAMP2B in two patients and also showed reduction of myocardial cell disarray and accumulation of autophagic vacuoles, a hallmark of Danon Disease. This in turn translated into early stabilization and suggests a trend toward improvement in functional measures. Importantly, safety profile looked manageable with elevation in transaminases observed in all three low-dose patients and returned to baseline within the first one to two months post-treatment. These elevations were largely responsive to corticosteroids and other immunosuppressive therapies. Unfortunately, one of the two high dose patients experienced reversible thrombocytopenia and acute kidney injury (returned to baseline in three weeks and regained normal kidney function). I can understand why management decided to try a higher dose (see how high they could push efficacy including extending benefit to all organs), but I am glad they discontinued it. CEO Shah stated that the slide on vacuoles stopped him in his tracks¨, showing the change from extensive vacuoles and no muscle architecture to fewer vacuoles and relevation of intact, restored muscle architecture (¨the vacuum cleaner is working¨). Significant improvements in cardiac output were observed consistent with BNP decreases and high levels of protein expression. Again, I liked seeing improvement across multiple endpoints (more convincing). Corporate Slides Figure 5: Changes in LAMP2 cardiac protein expression is quite striking to my eyes (Source: corporate presentation) Corporate Slides Figure 6: Improvement in functional clinical status of adult cohorts (Source: corporate presentation) Lastly, I cited a key green flag with noteworthy institutional investors (RTW Investments had almost 15 million shares) and stacked management team (hailing from the likes of Novartis, Bristol-Myers Squibb, Biogen, Eli Lilly, AstraZeneca and more). Select Recent Developments In January, management started the year in their typical manner by transparently sharing projected clinical milestones. Of note, pediatric data for Danon was expected in Q3 and initiation of pivotal phase 2 study activities was guided for Q4. For Q2, they set out the goal of achieving in-house AAV GMP (Good Manufacturing Practice) manufacturing, which I consider vital for value creation in cell therapy and gene therapy companies. For the PKD program, I was surprised that with so few patients' worth of data, they plan to initiate a pivotal phase 2 study in Q4. On May 16th, the company reported updated data across three programs (Danon, Fanconi, PKD). Starting with Danon as it is the focus of my investment thesis for this company, pediatric data with April 30th cutoff showed the low dose of RP-A501 was well tolerated in both patients (normal-range platelets, diminished complement activation and no complement-related adverse events). Importantly, all adverse events in pediatric and adult cohorts were reversible with no lasting renal, hepatic, or other sequelae. As for signs of activity, patients demonstrated evidence of cardiac LAMP2B expression versus normal control, echocardiograms showed decreased cardiac wall thickness with improved or stabilized ejection fraction and perhaps most impressively patients demonstrated sustained improvement or stabilization in BNP and NHYA (New York Heart Association) class, 6-minute walk and most importantly increases in physical activity. As for the Fanconi Anemia study, 5 of 9 evaluable patients had increased resistance to MMC in bone marrow-derived colony forming cells (21% to 42% at 18 months, compared to 51%-94% at 18 to 21 months). This is important, keeping in mind that the primary endpoint of the study has thus been achieved as it required minimum five patients to attain increased MMC resistance at least 10% above baseline at two or more timepoints. As for the PKD program, as noted prior both patients at 18 months post-infusion experienced sustained transgene expression, normalized hemoglobin, improved hemolysis, no red blood cell transfusion requirements post-engraftment and improved quality of life both reported anecdotally and as documented via formal quality of life assessments. Pediatric cohort is enrolling, and importantly safety profile appeared favorable with only transient transaminase elevation observed in both patients post-therapy/conditioning (no clinical stigmata of liver injury). A few days later, astoundingly positive LAD-I data update was shared and it surprised me the stock responded so well considering this program presents a tiny market opportunity. At 3 to 24 months post RP-L201 infusion, all nine patients sustained stable CD18 expression (median: 56%) with no therapy-related serious adverse events. The plan here is to initiate discussions with regulatory authorities and complete filings in the first half of 2023. Finally, on July 27th the company disclosed an important new hire in the form of Mayo Pujols as Chief Technical Officer (served prior as Head of Global Cell and Gene Technical Development and Manufacturing for Novartis Pharmaceuticals). This experience is highly relevant for the person who will lead the technical operations function and chemistry, manufacturing and controls ((CMC)) for all lentiviral programs as well as AAV manufacturing facility for a planned phase 2 pivotal study in Danon Disease. Other Information For the second quarter of 2022, the company reported cash and equivalents of $321M as compared to elevated net loss of $54M. Research and development expenses rose to $41M, while G&A costs came in at nearly $13M. It would seem management is aware of the impending cash crunch, as in June the company sold 1.3M shares via at the market facility and received $17.3M in proceeds. They are guiding for current resources to provide them operational runway to 1H 2024 (includes buildout and initiation of AAV cGMP manufacturing capabilities and facility as well as "future pipeline programs"). Gene therapy is certainly an expensive area of biotech to operate, and so far the company has an accumulated deficit of $491M as per the 10-K filing. Moving on to management's presentation at UBS Global Healthcare conference, here were a few nuggets that stood out to me: Shah describes Rocket as multi-platform and platform agnostic. They have two platforms, one being an in-vivo AAV platform applied to cardiology disease and ex-vivo lenti programs applied to three bone marrow-derived diseases. Fanconi anemia and LAD-I programs are nearing the end of their phase 2 pivotal studies. Danon disease and PKD will enter phase 2 by the end of the year or into 2023. Both platforms had further validation with promising data sets presented in May. "When gene therapy works, it really works" and LAD-I data is used as an example. These children otherwise pass away in early single digit years and restored CD18 function allows them to live normal, happy lives (normal lifespan). Fanconi Anemia hit the primary endpoint ahead of planned schedule, regulatory filings expected for FA and LAD-I in 2023. For Danon disease, it was a big deal that they presented favorable safety data in pediatric patients. They were able to translate safety in the mid e13 range, both patients had minimal complement activation and no clinical AEs. The total Danon natural history study will be up to 200 patients, not all will be followed for every single time point they are interested in for phase 2. They are finding a good number of them are followed for lab markers, for echo (wall thickness, EF, etc) and some are followed for NYHA class & 6-minute walk test. So far, they have been able to identify a couple markers that trend very differently from patients in their study (BNP and posterior wall thickness). Based on the delta they see (differences), they think they can design a well-powered, modest study potentially as single arm design. Danon patients' ejection fraction is usually preserved until late in the disease, the last one or two years. Most of Rockets' patients have 50% or 60% EF and there is not much to measure as an outcome (did improve overall). Other parameters such as wall thickness are grossly deranged and much easier to follow (mechanistic rationale for posterior wall thinning). Greater magnitude of benefit is observed here letting them design a smaller pivotal study. As for protein expression as a possible endpoint, Shah states a little protein in each cell goes a long way. "You need to turn on the vacuum cleaner just a little bit and give it enough time to clean up the cell". If they turn it on too much, they could theoretically damage the cell. The right goldilocks zone has a little protein and a lot of cells, so IHC is the better measure (want broad expression and takes 9 to 12 months to see true clinical effect in these patients). Shah states that there is a lot of interest from female Danon patients and they do get sick. They tend to live to a longer age but still have cardiomyopathy and pass away in their 40s. This study could start sometime in 2023, but the first goal is to get pivotal male study up and running. As for overall opportunity in Danon, Shah states it will be all comers males and females (at least 8 years old, potentially earlier). Males should be everybody except males who have pre-existing AAV immunity or patients too far gone in fibrosis. With females, Danon disease manifests differently so they might wait until there are more symptoms before treating. As for Fanconi Anemia market opportunity, Shah states that prevalence is 4,000 EU+ US. As for cash burn and runway, they are guiding for runway to 1H 24. As they start thinking about launches for LAD and Fanconi, at some point those could result in Priority Review Vouchers to generate internal revenue. Moving onto IP position, most of the IP around lenti programs is expired. For AAV in Danon, they have a license from REGENXBIO for the AAV capsid and beyond that they own everything. Shah confirms that big pharma is more interested in derisked companies with programs in late stage or ready for launch. As for prior financing, aside from the recent use of ATM facility, upsize secondary offering in December took place at $56/share (~3x upside from current levels). As for institutional investors of note, Perceptive Advisors sold nearly half of its take and owns 3% of the company. RTW Investments continues to incrementally add to its position and owns a whopping 25% stake. As for insiders, Chief Medical Officer Jonathan Schwartz owns over 80,000 shares and has been continually selling down his stake from over 200,000 originally. CEO Guarav Shah owns over 500,000 shares. Moving on to the management team, CEO Guarav Shah served prior as Global Program Head in the Cell & Gene Therapies Unit at Novartis, where he had strategic oversight of 12 functions and helped spearhead pivotal trials with CART-19 for patients with leukemia and lymphoma. I touched on two new key hires above and their relevant technical expertise as well. Chief Commercial Officer Carlos Martin served prior as U.S. Commercial Head of Advanced Accelerator Applications ((AAA)), a Novartis Oncology Company. Uniquely of interest, Chief Development Officer Gayatri Rao served prior as Director of the Office of Orphan Products Development (OOPD) within the FDA.Analysis Article • Jul 22
We're Not Very Worried About Rocket Pharmaceuticals' (NASDAQ:RCKT) Cash Burn Rate
Even when a business is losing money, it's possible for shareholders to make money if they buy a good business at the...Seeking Alpha • Jul 08
Rocket Pharmaceuticals an outperform at Raymond James on gene therapy programs
Raymond James has initiated Rocket Pharmaceuticals (NASDAQ:RCKT) with an outperform rating seeing opportunity with four of its gene therapy programs. The firm has a $22 price target (~36% upside based on Thursday's close). Analyst Timur Ivannikov highlighted the company's four rare disease candidates: RP-L102 (Fanconi anemia), RP-L201 (leukocyte adhesion deficiency-I), RP-A501 (Danon Disease), and RP-301 (pyruvate kinase deficiency). For the first two programs, Ivannikov said that Rocket could file applications with the U.S. FDA for both in 2023. He added that RP-A501 presents potentially the biggest opportunity for the company with peak revenue of ~$1.1B. He gives it a 40% chance of success. Pediatric cohort data is expected this quarter. RP-L301 is the company's earliest stage candidate and Ivannikov has assigned it a 50% probability of success. Additional data is expected in Q4. Check out Seeking Alpha contributor Avisol Capital Partners' bullish take on Rocket (RCKT).Seeking Alpha • Jun 30
Rocket Pharma: Poised For Growth In The Rare Disease Space
Rocket is running a number of successful rare disease programs. Their data so far is excellent. They have a decent cash runway. Rocket Pharmaceuticals, Inc. (RCKT) aims to cure rare diseases using gene therapy. Its pipeline looks like this: PIPELINE (Co website) About the company, they claim the following in their Corporate Presentation: they have 4 programs with compelling clinical proof of concept. Each molecule is first, best, and only-in-class, with clear mechanisms of action and clinical endpoints. Their approach is directed towards less-expensive treatment options for diseases currently needing expensive transplants and lifelong care. They have a 100,00 sqft in-house AAV (adeno-associated vector) manufacturing capability in New Jersey. Management has experience developing over 20 commercial drugs. The company has three clinical-stage ex vivo lentiviral vector (“LVV”) programs. These target Fanconi Anemia (‘FA), Leukocyte Adhesion Deficiency-I (“LAD-I”), and Pyruvate Kinase Deficiency (“PKD”). FA is a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. LAD-I is a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells. PKD is a genetic disorder that causes the immune system to malfunction. [These descriptions are taken from the 10-K]. The company says that the phase 2 FA program and the phase 1/2 LAD-I program are in registration-enabling studies. This is possible in such earlier stage studies because these are extremely rare diseases. The company also has a program in Danon disease, which is a multi-organ lysosomal-associated disorder leading to early death due to heart failure. This program uses the AAV vector. Data is available from a number of programs. In 2018, positive data was announced from a phase 1/2 trial in FA. Data in five patients showed a treatment effect, with all patients showing continuing improvement in restoration of functionality of hematopoietic stem cells in bone marrow. One patient had a 44% improvement in peripheral blood vector copy number ((VCN)), a measure of the transduction (transfer of genetic material) of hematopoietic progenitor cells. 12-month follow-up data on four patients showed durable engraftment, ranging from 18 - 30 months since gene therapy administration as well as improvements in two key measures of functional and phenotypic correction in FA. In 2020, the company presented early phase 1 data from the Danon disease program. A low dose cohort saw a positive increase in cardiac protein expression. There was also a decrease in BNP, a cardiac biomarker, of more than 50%, and stabilization of clinical biomarkers CK-MB and Transaminases in two patients. Researchers also observed visible reduction of autophagic vacuoles (a primary hallmark of Danon disease) in heart muscle. In May 2021 the FDA placed a clinical hold on this program to have the company add additional risk mitigation measures in the study protocol. By August, the hold was lifted. In November, Rocket stock crashed after the company decided to exclude the high dose group from the study after one of the two subjects who received the higher dose developed dose-dependent toxicity that led to thrombotic microangiopathy ((TMA)). Although the issue was resolved, the company decided not to take any risks. Last year, in the phase 1 LAD-I trial, RP-L301 showed durable normalization of hemoglobin levels up to 6 months following therapy and a similar 3-month trend in the second patient treated. In interim data from a phase 1/2 trial, the following data were observed: The data are from the initial seven patients with severe LAD-I, as defined by CD18 expression of less than 2%, who were treated with RP-L201, Rocket’s ex-vivo lentiviral gene therapy candidate. All seven patients demonstrated durable neutrophil CD18 expression that exceeded the 4-10% threshold associated with survival into adulthood and consistent with reversal of the severe LAD-I phenotype, the company said. Peripheral blood vector copy number ((VCN)) levels have been stable and in the 0.5 – 2.5 copy per genome range. The company has decided to pursue full approval instead of accelerated approval by collecting survival data. This came after the company unveiled 100% survival data in 7 patients from the phase 2 pivotal trial at the 25th Annual Meeting of the American Society of Gene and Cell Therapy in May. An NDA is being planned in 2023. Financials RCKT has a market cap of $918mn after steadily going down over the last several quarters and losing nearly $2bn in market cap. It has a cash balance of $346mn. Research and development expenses were $30.8 million for the three months ended March 31, 2022, while general and administrative expenses were $11.7 million. At that rate, and given the small size of their trials given the rare nature of the target indications, they have a cash runway of nearly 2 years.Seeking Alpha • Jan 30
Rocket Pharmaceuticals: A Struggling Company With Long-Term Upside
The biotech market has seen a massive influx of revenue due to the surging Covid pandemic. While Rocket does not benefit from the Covid related market, it does have five new gene therapy programs in clinical trials. The company has delivered favorable updates on its new program’s progress towards becoming a marketable product in Q4 2021. Despite the downfall in share price, Rocket’s financials show it is still well capitalized.Analysis Article • Nov 09
We're Hopeful That Rocket Pharmaceuticals (NASDAQ:RCKT) Will Use Its Cash Wisely
Even when a business is losing money, it's possible for shareholders to make money if they buy a good business at the...Seeking Alpha • Oct 01
Rocket Pharmaceuticals Stock Forecast: Can It Rebound To $50 Levels? A Resounding Yes
Rocket Pharmaceuticals, Inc. is a clinical-stage, multi-platform biotechnology company developing gene therapies with a focus on direct on-target mechanisms of action for rare and devastating orphan diseases. Rocket Pharmaceuticals currently has five ongoing gene-therapy clinical programs covering both AAV and LVV-mediated gene delivery. The Company is well-capitalized and in a strong financial position with ~4 years of cash runway and average analyst revenue expectations of $581M by FY 2024. RCKT has a sizeable catalyst upcoming in 4Q 21 where the company expects to present very critical updates across their five ongoing programs highlighting much anticipated therapeutic risk data. In summary, the author projects Rocket Pharmaceuticals, Inc. as a strong "buy" at a 2-3 year Wall Street price target of $69.70/share (+152% upside).Earnings and Revenue Growth Forecasts
| Date | Revenue | Earnings | Free Cash Flow | Cash from Op | Avg. No. Analysts |
|---|---|---|---|---|---|
| 12/31/2028 | 65 | -153 | -91 | -159 | 8 |
| 12/31/2027 | 20 | -183 | -168 | -162 | 12 |
| 12/31/2026 | 17 | -133 | -124 | -89 | 12 |
| 3/31/2026 | N/A | -209 | -180 | -180 | N/A |
| 12/31/2025 | N/A | -223 | -190 | -190 | N/A |
| 9/30/2025 | N/A | -241 | -203 | -202 | N/A |
| 6/30/2025 | N/A | -257 | -206 | -204 | N/A |
| 3/31/2025 | N/A | -258 | -213 | -209 | N/A |
| 12/31/2024 | N/A | -259 | -216 | -210 | N/A |
| 9/30/2024 | N/A | -258 | -202 | -192 | N/A |
| 6/30/2024 | N/A | -253 | -210 | -197 | N/A |
| 3/31/2024 | N/A | -249 | -210 | -194 | N/A |
| 12/31/2023 | N/A | -246 | -211 | -195 | N/A |
| 9/30/2023 | N/A | -253 | -236 | -222 | N/A |
| 6/30/2023 | N/A | -249 | -219 | -208 | N/A |
| 3/31/2023 | N/A | -237 | -206 | -196 | N/A |
| 12/31/2022 | N/A | -222 | -186 | -178 | N/A |
| 9/30/2022 | N/A | -199 | -160 | -153 | N/A |
| 6/30/2022 | N/A | -192 | -147 | -137 | N/A |
| 3/31/2022 | N/A | -172 | -145 | -136 | N/A |
| 12/31/2021 | N/A | -169 | -129 | -121 | N/A |
| 9/30/2021 | N/A | -186 | -122 | -103 | N/A |
| 6/30/2021 | N/A | -165 | -108 | -93 | N/A |
| 3/31/2021 | N/A | -155 | -92 | -77 | N/A |
| 12/31/2020 | N/A | -140 | -95 | -75 | N/A |
| 9/30/2020 | N/A | -99 | -98 | -81 | N/A |
| 6/30/2020 | N/A | -89 | -98 | -75 | N/A |
| 3/31/2020 | N/A | -82 | -99 | -71 | N/A |
| 12/31/2019 | N/A | -77 | N/A | -65 | N/A |
| 9/30/2019 | N/A | -85 | N/A | -66 | N/A |
| 6/30/2019 | N/A | -82 | N/A | -67 | N/A |
| 3/31/2019 | N/A | -79 | N/A | -58 | N/A |
| 12/31/2018 | N/A | -75 | N/A | -54 | N/A |
| 9/30/2018 | N/A | -54 | N/A | -39 | N/A |
| 6/30/2018 | N/A | -44 | N/A | -31 | N/A |
| 3/31/2018 | N/A | -32 | N/A | -24 | N/A |
| 12/31/2017 | N/A | -20 | N/A | -16 | N/A |
| 9/30/2017 | N/A | -16 | N/A | -12 | N/A |
| 6/30/2017 | N/A | -11 | N/A | -9 | N/A |
| 3/31/2017 | N/A | -9 | N/A | -8 | N/A |
| 12/31/2016 | N/A | -8 | N/A | -6 | N/A |
Analyst Future Growth Forecasts
Earnings vs Savings Rate: RCKT is forecast to remain unprofitable over the next 3 years.
Earnings vs Market: RCKT is forecast to remain unprofitable over the next 3 years.
High Growth Earnings: RCKT is forecast to remain unprofitable over the next 3 years.
Revenue vs Market: RCKT's revenue (58.7% per year) is forecast to grow faster than the US market (11.7% per year).
High Growth Revenue: RCKT's revenue (58.7% per year) is forecast to grow faster than 20% per year.
Earnings per Share Growth Forecasts
Future Return on Equity
Future ROE: RCKT is forecast to be unprofitable in 3 years.
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Company Analysis and Financial Data Status
| Data | Last Updated (UTC time) |
|---|---|
| Company Analysis | 2026/05/15 06:42 |
| End of Day Share Price | 2026/05/15 00:00 |
| Earnings | 2026/03/31 |
| Annual Earnings | 2025/12/31 |
Data Sources
The data used in our company analysis is from S&P Global Market Intelligence LLC. The following data is used in our analysis model to generate this report. Data is normalised which can introduce a delay from the source being available.
| Package | Data | Timeframe | Example US Source * |
|---|---|---|---|
| Company Financials | 10 years |
| |
| Analyst Consensus Estimates | +3 years |
|
|
| Market Prices | 30 years |
| |
| Ownership | 10 years |
| |
| Management | 10 years |
| |
| Key Developments | 10 years |
|
* Example for US securities, for non-US equivalent regulatory forms and sources are used.
Unless specified all financial data is based on a yearly period but updated quarterly. This is known as Trailing Twelve Month (TTM) or Last Twelve Month (LTM) Data. Learn more.
Analysis Model and Snowflake
Details of the analysis model used to generate this report is available on our Github page, we also have guides on how to use our reports and tutorials on Youtube.
Learn about the world class team who designed and built the Simply Wall St analysis model.
Industry and Sector Metrics
Our industry and section metrics are calculated every 6 hours by Simply Wall St, details of our process are available on Github.
Analyst Sources
Rocket Pharmaceuticals, Inc. is covered by 23 analysts. 12 of those analysts submitted the estimates of revenue or earnings used as inputs to our report. Analysts submissions are updated throughout the day.
| Analyst | Institution |
|---|---|
| Jason Zemansky | BofA Global Research |
| Whitney Ijem | Canaccord Genuity |
| Joshua Schimmer | Cantor Fitzgerald & Co. |