Good morning, everyone. Welcome to CANbridge Pharmaceuticals' 2022 12-month annual result conference call. Today, it's our great honor to have Dr. James Xue, Founder, Chairman, and CEO of CANbridge Pharmaceuticals, Dr. Gerald Cox, Chief Medical Officer and Chief Development Strategist, Glenn Hassan, Chief Financial Officer, and Sophie Xie, Director of Investor Relations. Management team will give us a brief introduction of the result and company development, and then we're going to have the Q&A session. We welcome questions in both Mandarin and English. Now, I will hand over to James. Thank you.
Thank you, operator. Hello, everyone, thank you all for joining us today to share with you our 12 months annual financial results and business update. Perhaps before we get into some of the specific events that highlight our tremendous progress in this past year, I would like to provide a brief background of CANbridge. CANbridge was founded in 2012 with a vision to build a leading China-based global biopharmaceutical company, developing and commercializing drugs that treat rare and difficult to treat diseases that target large and underserved markets both in China and globally. This area represents a large global market opportunity valued in excess of $135 billion in 2020. In China, the prevalence of rare disease is nearly 4x larger than that of the United States or Europe, yet it represents less than 1% of total revenues.
We believe this presents a significant and tangible opportunity for CANbridge to pioneer the development of rare disease products in China and bring these products to global markets. To best capitalize on this enormous opportunity, we have developed a business model focused on strategically combining global collaborations and internal research capabilities to build a portfolio of products where our collaborators have established clinical proof of concept, thereby reducing the substantial development risk. To execute our business model, we continue to work to assemble a leading management team, which collectively has a track record of success developing and commercializing rare disease therapies across key markets, including China, United States, Europe, Latin America, and Southeast Asia. Over this past year, we have made significant progress building CANbridge and advancing our pipeline programs.
We have a leading pipeline that consists of 14 drug assets targeting rare disease with significant market potential, including three currently marketed products, five drug candidates in late-stage clinical trials, one at IND-enabling stage, two at preclinical stage, and another three gene therapy programs at lead identification stage. Importantly, most of our late-stage programs have now been included in China's first national list of rare diseases, which is a list identifying products that have the potential to address a large unmet need and has the potential to be used to facilitate reimbursement of these products in China. We believe that having a number of programs on this list not only validates our development strategy focused on products where there's a large need, but also supports our belief that a strong commercial opportunity will exist once these products are approved.
Based on our progress this past year, we are anticipating an exciting year ahead. We look forward to sharing a number of important program updates that we believe will drive both near-term and long-term value to our stockholders. Specifically, over the course of the year, we're anticipating important data readouts, including data from key registrational trials for CAN008 for the treatment of glioblastoma, CAN106 for the treatment of paroxysmal nocturnal hemoglobinuria, and CAN103 for the treatment of Gaucher disease. In addition to these important milestones, our CAN108 programs for the treatment of rare liver disease have been granted priority review by the NMPA. We are anticipating a potential approval in the first half of 2023.
All of these programs are being developed for China and our global markets, pending the outcome of the clinical trials could give CANbridge potentially four new commercial products that would be on the market over the next two to three years. These successes will dramatically change the profile of CANbridge and put us in position to drive significant growth and accelerate profitability. As we prepare for multiple commercial launches, we already have a full-fledged commercial team in Greater China that has generated sales of RMB 79 million in 2022, representing a growth of 153% over the prior year period. Today, our commercial team is actively marketing our first product, Hunterase, for the treatment of Hunter syndrome, MPS II. Importantly, Hunterase established a beachhead in China, for which we intend to expand our commercial efforts.
Over the course of the next five years, we plan to expand this dedicated in-house commercialization team and assemble a full-fledged commercialization team in Greater China that is capable of not just supporting countries, but commercial team that is capable of launching the next wave of products that I have just mentioned. Beyond our current clinical and commercial success, we are also progressing our next-generation technologies in gene therapies. In July 2022, we opened CANbridge Next-Generation Innovation and Process Development Facility that will house our development initiatives for potentially curative gene therapies for rare genetic diseases. In January 2023, we announced an in-license agreement from UMass Chan Medical School and signed a worldwide license agreement with LogicBio that solidified global development and commercial rights for a number of high-value gene therapies, including Pompe disease, Fabry disease, Spinal Muscular Atrophy, SMA, and other neuromuscular disorders.
These initiatives have already begun to yield success. This past year, we presented initial data from our next-generation gene therapy program for the treatment of SMA. SMA is a debilitating and deadly hereditary disease affecting the Central Nerve System of children. In 2022, we presented our preclinical data of the SMA gene therapy program at multiple medical meetings, including the American Society of Gene & Cell Therapy Annual Meeting in May, the European Society of Gene & Cell Therapy 29th Annual Congress in October, and the World Muscle Congress in October. These presentations highlight the potential of this novel second-generation self-complementary AAV9 gene therapy for treating SMA. Program like this is further validating our efforts using rational design to engineer the next generation of gene therapies that have potential to be more efficacious, have a better safety profile, and to be manufactured more efficiently.
We believe gene therapy represents the future, and this recent development make CANbridge a global leader developing these potentially one-time durable treatments for a number of rare genetic diseases. We look forward to sharing additional data and updates for many of these programs in 2023. With that, I would like to turn the call over to Dr. Gerald Cox, our Chief Medical Officer and Chief Development Strategist, to discuss in depth the progress on our pipeline.
Thank you, James. We've made significant progress with respect to our drug pipeline over the past year. Let me begin with our considerable progress advancing CAN108 or LIVMARLI, an oral minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter or IBAT in patients from Greater China. We are developing CAN108 as a potential treatment for a group of rare cholestatic liver diseases that represent a multi-billion market opportunity, including indications such as Alagille syndrome, progressive familial intrahepatic cholestasis or PFIC, and biliary atresia. maralixibat has already been extensively studied by our partner Mirum in over 1,600 patients with demonstrated safety and efficacy in multiple studies and has received FDA approval in the U.S. and Europe for the treatment of Alagille syndrome.
Last year, Mirum reported positive results from its phase III study and recently submitted a supplemental new drug application for the PFIC indication. We believe the overall data generated by Mirum in both indications significantly reduces the development risk of CAN108. Furthermore, the first patient in the phase II EMBARK study of CAN108 in biliary atresia was dosed in China at the Children's Hospital of Capital Institute of Pediatrics in Beijing. This clinical site in China is part of the global EMBARK study in biliary atresia conducted by Mirum and supported by CANbridge under the license agreement with Mirum. This multicenter randomized controlled phase II study to evaluate the efficacy and safety of CAN108 in patients with biliary atresia after Kasai surgery is expected to enroll at least 20 patients in Greater China.
From the regulatory perspective, our new drug application for CAN108 in Alagille syndrome has been accepted by China's National Medical Products Administration or NMPA and the Taiwan Food and Drug Administration. In addition, CAN108 has also been granted prior to review by NMPA, and we anticipate the potential approval of CAN108 in mainland China in Q2 of 2023. Overall, we believe CAN108 exemplifies our ability to identify de-risk products that target large potential markets and that we can efficiently progress through clinical trials and onto commercial launch. We're also very excited about CAN106, a novel long-acting monoclonal antibody for the treatment of paroxysmal nocturnal hemoglobinuria or PNH, myasthenia gravis and other complement-mediated diseases.
We obtained global rights to develop and commercialize the product from WuXi Biologics and Privus, we plan to develop CAN106 to be a best-in-class product that is competitively priced and is the preferred treatment option in China, with a view to gaining approval in Western countries later in the clinical development program. In November of 2022, the U.S. FDA granted CAN106 orphan drug designation for the treatment of myasthenia gravis, which makes it eligible for certain benefits under the Orphan Drug Act, including tax benefits and seven years of market exclusivity. Importantly, this designation is a tacit acknowledgment from FDA that CAN106 is different from the two approved anti-C5 monoclonal antibodies, which means that their orphan drug exclusivity from marketing authorization.
In 2022, we presented positive top-line results for the phase I healthy volunteer study at several major medical conferences, including the 17th National Congress on Hematology in Shanghai, the sixth annual Complement-Based Drug Development Summit in Boston, Massachusetts, USA, the European Hematology Association 2022 Congress in Vienna, Austria, and the 14th International Conference on Complement Therapeutics in June 2022 in Rhodes, Greece. The data highlighted that CAN106 was safe and well-tolerated and achieved rapid, complete, and sustained blockade of complement function. The pharmacokinetics of CAN106 was linear and dose proportional, and the half-life was approximately 31 days, which is similar to the AstraZeneca Alexion's ravulizumab and supports an extended dosing interval in patients. With this data in hand, we advanced the program and initiated a phase I-b/II study in PNH patients in China.
This multi-center, open-label phase I-b/II study will evaluate the tolerability, efficacy, safety, and PK/PD of CAN106 administered intravenously to complement inhibitor treatment-naive PNH patients. The ongoing phase I-b part of the study consists of three dose cohorts. We plan to announce top-line data from the phase I-b trial in mid-year 2023. CAN106 presents a substantial market opportunity, as evidenced by the commercial success of other anti-C5 monoclonal antibodies approved in the U.S. and other major markets. Our aim is to develop a novel product that stands out in the global market. To achieve this, we formed a scientific advisory board during the year comprised of leading experts with vast clinical experience in disease areas where C5 activation is known or believed to play a crucial role and where anti-C5 therapeutics may offer significant benefits.
With their guidance, we're continually assessing the potential for CAN106 across a wide range of indications, helping us to focus on the most promising therapeutic areas. As we move forward, we're confident that the expertise of our scientific advisory board will play a vital role in the successful development of CAN106 as a differentiated product in the global market. Moving on to CAN008, this is a glycosylated CD95-Fc fusion protein that is currently in phase II and is being developed as a potential first-line treatment for a rare form of brain cancer called glioblastoma multiforme, or GBM, in mainland China. GBM represents the deadliest form of brain cancer, accounting for 45% of all malignant brain tumors.
Our phase II clinical trial is designed to be multi-center, randomized, double-blind, and placebo-controlled to investigate the efficacy of CAN008 and to explore the correlation of different biomarkers with treatment outcome. The primary endpoint of the trial is progression-free survival, and the key secondary endpoint is overall survival. In previous clinical studies, CAN008 has demonstrated promising efficacy and a favorable safety profile in completed clinical trials, presenting a potentially new treatment option for this devastating cancer. Our collaborator recently presented long-term follow-up data from the phase I/II study of CAN008 plus temozolomide radiotherapy, or TMZ RT, in newly diagnosed GBM patients at the European Society for Medical Oncology Sarcoma and Rare Cancers Annual Congress in Lugano, Switzerland.
The study showed a remarkable median progression-free survival of 17.95 months in GBM patients who received the high dose of CAN008, which is more than double the historical median progression-free survival for standard of care GBM patients. 67% of these high-dose patients were alive after five years, compared to only 9.8 months based on historical data from the clinical site. We recently completed enrollment of the phase ii clinical trial, and we expect the planned interim analysis to read out in the middle of the year. It remains our plan to commercialize CAN008 as a first-line treatment for GBM in China as a combination therapy on top of standard of care of TMZ and radiotherapy.
We are also progressing CAN103, a recombinant human glucocerebrosidase that is the first enzyme replacement therapy for the treatment of Gaucher disease to be developed in China. CANbridge maintains proprietary rights to develop and commercialize the product in China and globally. Gaucher is one of the best-known and longest treated rare diseases, as evidenced by the large number of published research articles. Gaucher disease has more drugs approved than other most other rare diseases. In July 2022, we are pleased to dose the first patient with CAN103 in a phase I/II trial of patients with Gaucher disease Types 1 and 3 in China. This multi-center trial consists of two parts. Part A, which is phase I, is an open label study to evaluate the safety, tolerability, and pharmacokinetics of different dose levels of CAN103 in a small number of treatment-naive subjects with Gaucher disease Type 1.
Part B, which is phase II, is a study to assess the safety and efficacy of CAN103 in a larger number of subjects with Gaucher disease Type 1 or 3. Part B of the trial will serve as a potential registrational trial. The company has completed part A and initiated dosing in part B in the first quarter of 2023. We expect to be in position to file an NDA for the approval of CAN103 by mid-2024. Lastly, perhaps most exciting for the long-term growth potential of CANbridge, is the progress we've made advancing our gene therapy initiatives focused on adeno-associated virus, or AAV, as a gene delivery vehicle, with a potential as a one-time durable therapy for many different genetic diseases.
As James mentioned, we presented initial data from our gene therapy research agreement with the Horae Gene Therapy Center of the University of Massachusetts Chan Medical School at various medical meetings this past year. These data encourage us to support the continued development of the second generation vector called CAN-203 as a potential best-in-class gene therapy for spinal muscular atrophy, or SMA. This next generation gene therapy leverages the advances in gene therapy that have occurred over the past two decades. Data shared at these meetings highlight the potential of this novel second generation self-complementary AAV9 gene therapy in treating patients with SMA. The second generation vector expresses a codon-optimized human SMN1 transgene under the control of an endogenous human SMN1 promoter.
It has demonstrated superior potency, efficacy, and safety in mice with spinal muscular atrophy compared to the benchmark vector, which is a self-complementary AAV9 containing a human SMN1 native transgene under the control of a CMV promoter. This benchmark vector is similar to the vector that's used in the gene therapy approved by the U.S. FDA for the treatment of SMA. I will turn the call over to Glenn to discuss the 12-month annual financial results.
Thank you, Gerry. I will now review the financial results for the full year ended December 31st, 2022. Just to remind everyone, all of these figures are in RMB. Our cash position was approximately RMB 463.1 million as of December 31st, 2022. Our revenue increased by RMB 47.8 million or 153% from RMB 31.2 million for the year ended December 31st, 2021 to RMB 79 million for the year ended December 31st, 2022. This was mainly attributable to the increase of sales from our Hunterase and Nerlynx.
Our R&D expenses decreased by approximately RMB 116.5 million or 27% from RMB 427.7 million for the year ended December 31st, 2021 to RMB 311.2 million for the year ended December 31st, 2022. This was primarily attributable to the decrease in upfront and milestone payment made to our licensing partners and partially offset by increase in testing and clinical trial expenses. R&D expenses, excluding milestone payments, increased by RMB 37.8 million from RMB 213.9 million for the year ended December 31st, 2021 to RMB 251.7 million for the year ended December 31, 2022.
Our administrative expenses decreased by RMB 36.6 million from RMB 145 million for the year ended December 31, 2021 to RMB 108.9 million for the year ended December 31st, 2022. The decrease was primarily attributable to the decrease in listing expenses and cost control in terms of professional fees. The loss for the reporting period decreased by approximately RMB 593.5 million or 55% from RMB 1.077 billion for the year ended December 31st, 2021 to RMB 483.5 million for the year ended December 31st, 2022. This loss was primarily attributable to decrease of loss in fair value changes of convertible redeemable preferred shares and R&D costs.
The adjusted loss for the year decreased by RMB 124.6 million or 21% from RMB 581.3 million for 2021 to RMB 556.7 million for 2022. The adjusted loss for the year is arrived by excluding the effect of a one-time non-cash IFRS fair value changes of our pre-IPO convertible redeemable preferred shares and derivative financial instruments, second, the share-based payment expenses, and third, the listing expenses. Please refer to the section headed Non-IFRS measures of this announcements for details. That concludes my comments on financial results, and I will turn the call back to James for his summary comments.
Thank you, Gerry and Glenn, for the great update. To close, as you just heard, we have made significant progress and are on our way to building a leading China-based global pharmaceutical company with a focus on the large and underserved rare disease market. We're proud of our commercial and development progress over this past year, and we enter the year with a lot of momentum as we continue our work to transform CANbridge into an integrated global biopharma company. We have a special opportunity in front of us, and I am convinced that with the strength of our science, the experience of our management team, the leadership of our board, and a world-class team of employees and external partners, we are well-positioned to develop and commercialize first-in-class therapies that have the potential to dramatically improve life for those living with rare diseases.
As we look at 2023, we have a number of high-value milestones that we look forward to sharing with the investment community, including the approval by the NMPA of CAN108 for the treatment of ALGS in the second quarter of 2023. Top-line data from our phase I-b study of CAN106 for the treatment of PNH in mid-year 2023. Interim analysis data from the phase II clinical trial of CAN008 being developed as the first-line treatment of glioblastoma in mid-year 2023. Depending on the strength of the data, the results could be potentially supporting an early NDA filing. Initiation of part B of phase II registrational trial of CAN103 for the treatment of Gaucher disease with a potential NDA filing in mid-year 2024.
In gene therapy, we plan to file an IND for SMA in 2024, share initial preclinical data in DMD, and advance work in Fabry and Pompe by the end of the year. Finally, I would like to thank our employees for all your ongoing dedication and contributions to our analysts and shareholders for your trust, support in helping build CANbridge. We look forward to advancing our mission and our commitment to making a difference in the lives of millions of patients and their families. With that, let me now turn the call back to the operator and open the line for questions.
Thanks for the management's presentations. We've now come to the Q&A session. If you have any questions, please feel free to share your questions in the chat box, and we will read out the questions. The first question is, what will be the company's key growth driver in the short to midterm? What is your expectation on revenue and profit for the coming year?
I would like Glenn to answer this question.
Yeah. Thank you, James. I think, you know, in the near term, this year we don't have new launches. We're gonna continue to promote Hunterase, and that should give us measured growth. We are expecting to get LIVMARLI approved in a matter of within a month or so. That will give us another product launch sometimes at the end of the year. We hope to get into the reimbursement by next year. We're excited to have that launch, and that should give us good growth in the midterm. Obviously, over the long term, as Gerry mentioned, we have a number of clinical readouts as well as the gene therapy programs that are potentially best in class.
I think you have a balance between the short term, midterm, and the long term of the company.
I also want to add it on top of what Glenn just said. You know, it's really depending on the success or not of CAN008. You know, we definitely very much look forward as it will enter into our product pipeline, pending on the result of phase II potential registrational study for GBM.
Thank you both. Next question. It seems that the company has had a very busy year. What are the most key and important readouts and approvals you are looking forward to this year?
I will answer this question. In my closing, I already highlighted a number of high-value milestones we look forward to in 2023. You know, first, is the highly anticipated NMPA approval of LIVMARLI or CAN108 for the treatment of ALGS. You know, this is certainly one of the, you know, first, the first of the three indications that we look forward from these products. The second is the top-line readout from our CAN106 phase I-b study in PNH patients. That's we anticipated in mid-year 2023.
The third is the analysis of the interim analysis data from our phase II, potentially registrational phase study, the frontline treatment of GBM, by our CAN008 in the middle of 2023. Depending on the data, we really have the anticipation for this data could potentially support an early NDA filing. We also have one more is the initiation of our registration study for CAN103 for the treatment of Gaucher disease that we have already initiated. We certainly look forward the progress can lead to an NDA filing in mid of 2024. Thank you.
Thank you, James. Once again, if you have any questions, please feel free to share your questions in the chat box and we will read out the questions. Next question. What is the market potential for CAN008 in GBM then, and how do you plan to differentiate it from other treatments currently available?
I would like Glenn to answer this question.
Thank you, James. I think CAN008 is offering us a tremendous opportunity. There are about 50,000 incidents of GBM in China. You know, this patient, this disease hasn't really seen any new products in the past decade or so. You know, with 50,000 patients, I think that's very attractive to us, and assuming regular pricing for targeted therapies, this could be about, you know, $400 million-$500 million dollar opportunities for us. It's very exciting and we look forward to the interim readout and maybe Gerry can talk a little bit about the current competitive environment.
Sure. Thanks, Glenn. Right now, the standard of care for GBM is a chemotherapy drug called temozolomide or TMZ, along with a course of radiotherapy. The way that we designed our study was to provide CAN008 as an add-on to the current standard of care. We won't be replacing TMZ or radiotherapy, we'll be delivering it on top of that. As Glenn mentioned, you know, after TMZ, you know, which has been around for close to 20 years, there really has been no approved therapy for GBM, no drug therapy. There is a device called Tumor Treating Fields, which has some benefit, but it's a very cumbersome device that has to be worn on the skull for a good period of the day.
We really don't have competitive threats to us at this time.
Yeah. We recently announced the long-term follow-up data from our Taiwan phase I-b study that resulted by the observation of our collaborators 67% overall survival after five years as opposed to the only 9.8% based on the from the standard of care post five years based on historic data. If we can demonstrate from our ongoing phase II study with the statistics difference, I think certainly this product will reach a home run in the potential, you know, under highly underserved market of GBM.
Thank you, management. The next question is: You have an anti-C5 product called CAN106. The market already has several competitors from what I know. What's your advantage compared to them?
I'd like Gerry to answer this question.
Sure. The CAN106, the anti-C5 that we've developed, was designed and reverse engineered at a company called Privus by an MIT professor. This antibody is designed to really optimally bind to C5 at normal pH and then be released at low pH, giving it an extended half-life. The sequence that comprises the antibody is distinct from the two approved anti-C5 antibodies. Because of that, the FDA has recognized our anti-C5 as being acceptable for orphan drug designation. This means that we will not be blocked from commercializing our anti-C5 in the U.S. which is really important. We are different than the existing antibodies. It also gives us market exclusivity for any of the indications that we seek approval that are considered rare diseases.
This is important because other generics, you know, would not be able to register their products for marketing authorization, you know, for at least seven years. Compared to the future products coming along, there are several biosimilars of eculizumab that will probably hit the market in the next two years. These are essentially replicas of eculizumab, and because of that, they're dosed every two weeks. Our antibody has modifications built into it that allow for a longer dosing interval. I think we will be competitive against eculizumab and its biosimilars, and we're believing that our drug will also be competitive with ravulizumab, which is very similar to eculizumab, but dosed every eight weeks.
Thank you, Gerald. Do we have any more questions? Please feel free to share your questions in the chat box. We will read out the questions. We have a question about gene therapy. Can you please provide an update on your gene therapy program for SMA, including the license from UMass Chan Medical School and the potential market opportunity?
Sure. I will answer the business part of this question, and I will let Gerry to answer some of the data we have presented on the SMA program in the last several months. As we have already announced, throughout our partnership collaboration with UMass Chan Medical School, in particular, Dr. Guangping Gao's group, that we were able to reach to a very fruitful result. Not only we have generated a candidate that we called a second generation or next generation SMA that bear certain features of the genetic engineering that allow the product to be more efficacious, safer, and probably more efficient in the future production. Also secured CANbridge the global rights for this product's development.
We very much look forward to moving this program through the clinical to IND readiness stage and with the target to file our first IND in the gene therapy in SMA in Q4 of 2024. Gerry can further elucidate the features of this candidate.
Thanks, James. The vector that we're using is the same, the AAV9 serotype, but the contents, the genetic material within the vector, differs, and I'll explain how that's so. We have modified the genetic sequence of the SMN1 protein, where the amino acid sequence will read out to be the same as the currently approved treatment, but the nucleotide sequence has been codon-optimized, and what that means is that it gets expressed at a higher rate than the native sequence. We estimate about a threefold increase in the expression rate which means that for cells that take up the vector, we can increase the amount of expression of SMN1 in those deficient cells and potentially, have a better ability to rescue them.
Another important feature of our vector is that we use a different promoter than what's used in the currently approved product. The promoter you can think of as like an on/off switch or a dimmer switch that can provide a certain amount of expression of your transgene. The currently approved gene therapy has what we call ubiquitous or nonspecific promoter that induces a high expression level of the transgene in all cell types, so it's not organ specific. Our promoter actually is a derivative of the endogenous SMN1 promoter. What that means is that cells that need higher amounts of SMN1 will have higher expression levels off the vector, and cells that don't require very much SMN1 will have lower levels. This is important because the current gene therapy is administered intravenously, and there's very high expression levels of the vector that go to the liver.
In the currently approved gene therapy, there is a black box warning for increased risk of potential liver failure as well as serious liver injury. There have also been other serious adverse events reported, including thrombotic microangiopathy, which causes clots to develop in the body and can result in complement activation and acute renal failure. There are some potential safety issues experienced by patients who receive the current gene therapy, and we have found that with our vector, we've eliminated the liver toxicity in a mouse model. We know from our studies that that's related to lower amounts of SMN1 expressed with our vector and liver compared to the current standard of care.
Conversely, we also see better expression within the central nervous system, and this is very important because that's where most of the disease is active in SMA. From the time that children are born with the most severe forms of SMA, they're actively losing neurons. The neurons are dying out over time. So it's very important to have a therapy that can arrest the treatment very quickly and provide the much-needed SMN1 protein, and we believe that the design elements that we've made in our vector will accomplish that. We feel confident because in the animal model, the mouse model of SMA, we've actually compared the currently approved gene therapy to ours, and we see much better overall survival, as well as more rapid recovery of motor function compared to the benchmark vector.
For these reasons, we believe we have the potential to be a best in class gene therapy for SMA.
Thank you, Gerry. Next question is a follow-up on this. What about gene therapy pipeline beyond SMA? Could you give us some color on that, including any promising candidates or partnerships that you have established in this area?
Sure. We have a couple of areas that we're focused on for gene therapy. One is neuromuscular disorders, and SMA is probably the most common neuromuscular disorder. Another high visibility indication we're working on is Duchenne muscular dystrophy, which is the most common muscular dystrophy of childhood. We have collaborations with Dr. Jeffrey Chamberlain at University of Washington, who is a very high profile translational scientist who has designed many of the micro-dystrophins that are currently being investigated as gene therapies for Duchenne muscular dystrophy. I would say the current micro-dystrophins again, are based on technology that's more than 10-20 years old, and we believe that some of the new innovations that we're incorporating have the potential to produce a dystrophin that is more functionally active.
With that, we've also entered into a collaboration with a company called Scriptr that has some novel gene editing technologies that we hope to leverage for Duchenne muscular dystrophy as well that will produce a more functional dystrophin. The DMD program is coming right behind SMA. The other area that we're very interested in is lysosomal storage disorders. We know the value of treatment with enzyme replacement therapy. There are about 12 enzyme replacement therapies that have been approved that work well to very well for many of these conditions, but they require dosing every one to two weeks. Most of them are dosed intravenously.
There's one enzyme replacement therapy that's dosed into the ventricles of the brain through a shunt, this occurs, you know, every one to two weeks, or in the case of the shunt, I believe it's once a month, for the rest of the patient's life. You know, that takes up a lot of time from the patient and their family to have to get infusions. What we've thought of doing is really, trying to replace the exogenous enzyme replacement therapy with a gene therapy that would produce the same enzyme within the patient's body, and it would produce it 24/7. It'd be like a continuous enzyme replacement therapy instead of giving it every one to two weeks. That accomplishes two things.
One is that it has the potential to be a one-time treatment for patients, so they don't have to go back to the clinic every one to two weeks. We believe that the amount of enzyme that we can produce within the body will be orders of magnitude higher than what can be achieved through traditional intravenous infusions. That's important because we know that some of the lysosomal storage disorders are not completely treated with existing enzyme replacement therapies. We believe that higher doses could achieve that, but they're just not practical to give through, you know, this intermittent enzyme replacement therapy intravenously. We are designing gene therapy vectors with our collaborator, LogicBio, that has worked with us to provide a vector that has exclusive targeting to the liver.
We've designed our transgenes in a way that we can express very high amounts of enzyme from the patient's liver. Essentially, we're converting the patient's liver into an in vivo manufacturing depot of the enzyme. The two diseases that are at the forefront that we're looking at is Fabry disease and Pompe disease. With Fabry disease, we have evidence in a mouse model of Fabry that we see very nice reduction of substrate in the affected tissues that accumulate the lipid that causes Fabry disease. We're very reassured by that and optimistic that this will translate into patients as well. Those are the main programs that we have coming up after SMA.
Thank you very much. Once again, if you have any questions, please feel free to share questions in the chat box and we will read out the questions. Do we have any more questions? If not, this concludes the end of the presentation. Ladies and gentlemen, that does conclude our presentation for today. Thank you all for participating. You may disconnect now.