▸ Federal legislative and regulatory efforts to implement reference pricing or most-favored-nation pricing models could impact our future product revenues and materially harm our business.· · · · ● 1 ▸ If our business plans are unsuccessful, our stockholders could lose all or a substantial portion of their investment in us.· · · · ● 1 ▸ MFN pricing models in the U.S. could also affect our international commercial strategy, directly and/or through our commercial partners, and future decisions on reimbursement and commercialization in certain jurisdictions.· · · · ● 1 ▸ Short sellers may engage in market manipulating activities and seek to drive down the market price of our common shares.· · · · ● 1 ▸ Tariffs imposed by foreign governments in retaliation for U.S. trade measures could adversely affect our international sales, results of operations, and financial condition.· · · · ● 1 ▸ The manufacture of cell-based therapies such as Deramiocel is complex, highly regulated and subject to multiple operational and technical risks.· · · · ● 1 ▸ We have a history of net losses, expect to continue to incur substantial net losses for the foreseeable future, and may never achieve or maintain profitability. Our operations to date have been primarily limited to organizing and staffing· · · · ● 1 ▸ We have limited commercial manufacturing experience and our manufacturing facilities may not receive or maintain the regulatory approvals required to support commercialization.· · · · ● 1 ▸ We have received orphan drug status for Deramiocel for the treatment of DMD from the U.S. and EMA. Even though we have received orphan drug designation (“ODD”) as described above, we may not be the first to obtain marketing· · · · ● 1 ▸ Disruptions in our supply chain or failure to adequately forecast product demand could result in significant delays or lost sales.· · · ● ● 2 ▸ From inception, we have financed our operations through private and public sales of our equity securities, government grants and payments from distribution agreements and collaboration partners.· · · ● ● 2 ▸ Our results of operations could be materially harmed if we or our distributor are unable to accurately forecast customer demand for our products and manage our inventory.· · · ● ● 2 ▸ The Company’s cell therapy technology (Deramiocel) is in late-stage development but not yet an approved product, and its exosome technology is still in preclinical development.· · · ● ● 2 ▸ We will need to increase our manufacturing capacity in the future and we may encounter problems at our current manufacturing facilities.· · · ● ● 2 ▸ Even if our product candidates receive regulatory approval, they may still face future development and regulatory difficulties.· · ● ● ● 3 ▸ Our certificate of incorporation and by-laws contain provisions that may discourage, delay or prevent a change in our management team that stockholders may consider favorable.· ● ● ● ● 4 ▸ The FDA has granted orphan drug status and an RMAT designation to Deramiocel for the treatment of DMD, but we may be unable to maintain or receive the benefits associated with orphan drug status, including market exclusivity, or an RMAT designation.· ● ● ● ● 4 rw ▸ We may need to rely exclusively on third parties to formulate and manufacture our product candidates and provide us with the devices and other products necessary to administer such a product.· ● ● ● ● 4 ▸ As a public company, we are subject to the reporting requirements of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), and other applicable securities rules and regulations, and are subject to the listing· · · ● · 1 ▸ Biologic products for commercial purposes must also be produced under cGMP. Our failure or any failure by these third parties to comply with these regulations or to recruit a sufficient number of patients may require us to repeat· · · ● · 1 ▸ Exosome-based therapeutics and vaccines are novel and unproven therapies which may not gain the acceptance of the public, patients or the medical community. To date, efforts by others to leverage natural exosomes have generally· · · ● · 1 ▸ Modifications to informed consent or other clinical trial requirements may affect enrollment or retention of patients, require modifications to trial documents and may cause delays to the trial.· · · ● · 1 ▸ Our future success will depend in part on our ability to maintain a competitive position with respect to evolving therapies as well as other novel technologies. Existing or future therapies developed by others may render our potential· · · ● · 1 ▸ Our research and development activities, preclinical studies, clinical trials, and manufacturing and marketing of our potential products are subject to extensive regulation by the FDA and other regulatory authorities in the United States,· · · ● · 1 ▸ If our business plans are not successful, we may not be able to continue operations as a going concern and our stockholders may lose their entire investment in us.· · ● · · 1 ▸ An epidemic or pandemic disease outbreak, including COVID-19, could severely disrupt our operations or the operations of third parties that we depend on, including any third-party contract manufacturer, our CROs, clinical data· ● · · · 1 ▸ Our ability to commercialize any products successfully also will depend in part on the extent to which coverage and reimbursement for these products and related treatments will be available from government health administration· ● · · · 1 ▸ Our resources and expertise to formulate or manufacture our product candidates on a large or commercial scale basis are still very limited. If we need to secure an additional manufacturer of our product candidates, demand for third-· ● · · · 1 ▸ A breakdown or breach of our information technology systems could subject us to liability or interrupt the operation of our business.● ● · · · 2 ▸ Delays in the commencement, enrollment, and completion of clinical testing could result in increased costs to us and delay or limit our ability to obtain regulatory approval for our product candidates.● ● ● · · 3 ▸ Each of these risks could delay our clinical trials, the approval, if any, of our product candidates by the FDA, or the commercialization of our product candidates, or result in higher costs or deprive us of potential product revenues.● ● ● ● · 4 ▸ If our business plans are not successful, our stockholders may lose their entire investment in us.● ● ● ● · 4 ▸ If we fail to comply with California laws governing the diversity of corporate boards of directors, we could be fined by the California Secretary of State.● · · · · 1 ▸ In the event we fail to satisfy any of the listing requirements of The Nasdaq Capital Market, our common stock may be delisted, which could affect our market price and liquidity.● ● ● · · 3 ▸ Nippon Shinyaku, any revenues we receive will depend upon the efforts of such third parties, and there can be no assurance that such efforts will be successful.● · · · · 1 ▸ Our internal computer systems, or those used by our CROs or other contractors or consultants, may fail or suffer security breaches.● ● · · · 2 ▸ The Company’s technology is not yet proven and each of our product candidates is still in clinical or preclinical development.● ● ● · · 3 ▸ The coronavirus outbreak could adversely impact our business.● ● · · · 2 ▸ The process of manufacturing our products is complex and we may encounter difficulties in production, particularly with respect to process development or scaling-up of our manufacturing capabilities.● ● ● ● · 4 ▸ We are subject to a number of manufacturing risks, any of which could substantially increase our costs and limit supply of our product candidates.● ● ● ● · 4 ▸ We have limited manufacturing capability and may not be able to maintain our manufacturing licenses.● ● ● ● · 4 ▸ We have no prior experience in manufacturing products for large, late-stage clinical trials or commercial use.● ● · · · 2 ▸ We are actively looking into potential additional strategic partnerships for our product candidates, particularly for Deramiocel in additional territories outside the United States and Japan, and for our exosomes product candidates. For● · · · ● 2 rw ▸ Any drugs we develop may become subject to unfavorable pricing regulations, third-party coverage, and reimbursement practices or healthcare reform initiatives, thereby harming our future business prospects.● ● ● ● ● 5 rw ▸ Any of these events could have a material and adverse effect on our operations and business and could adversely impact our stock price.● ● ● ● ● 5 ▸ Business disruptions such as natural disasters, widespread infectious diseases, or pandemics or geopolitical conflicts could seriously harm our future revenues and financial condition and increase our costs and expenses.● ● ● ● ● 5 rw ▸ Even if any of our product candidates receive marketing approval, they may fail to achieve the degree of market acceptance by physicians, patients, healthcare payors and others in the medical community necessary for commercial success.● ● ● ● ● 5 ▸ Even if our product candidates are approved, our ability to generate product revenues will be diminished if our products sell for inadequate prices or patients are unable to obtain adequate levels of reimbursement.● ● ● ● ● 5 rw ▸ Even if our product candidates receive regulatory approval, we may still face future development and FDA regulatory difficulties.● ● ● ● ● 5 ▸ Even if we were to obtain approval for Deramiocel for the treatment of DMD with the rare pediatric disease designation, the Rare Pediatric Disease Priority Review Voucher Program may no longer be in effect at the time of such approval.● ● ● ● ● 5 rw ▸ Failure to achieve and maintain effective internal controls in accordance with Section 404 of the Sarbanes-Oxley Act of 2002 could have a material adverse effect on our business and stock price.● ● ● ● ● 5 ▸ If any of our product candidates for which we receive regulatory approval do not achieve broad market acceptance, the revenues that we generate from their sales, if any, will be limited.● ● ● ● ● 5 ▸ If securities analysts do not publish research or reports about our business or if they publish negative evaluations of our stock, the price of our stock could decline.● ● ● ● ● 5 ▸ If we achieve our near-term product development milestones, we may not be able to manage any subsequent growth.● ● ● ● ● 5 ▸ If we do not establish strategic partnerships, we will have to undertake development and commercialization efforts on our own, which would be costly and delay our ability to commercialize any future products or product candidates.● ● ● ● ● 5 ▸ If we enter into strategic partnerships, we may be required to relinquish important rights to and control over the development of our product candidates or otherwise be subject to terms unfavorable to us.● ● ● ● ● 5 ▸ Market and economic conditions may adversely affect our industry, business and ability to obtain financing.● ● ● ● ● 5 ▸ Negative developments in the field of exosomes could damage public perception of any product candidates that we develop, which could adversely affect our ability to conduct our business or obtain regulatory approvals for such product candidates.● ● ● ● ● 5 ▸ Our ability to obtain reimbursement or funding for our programs from the federal government may be impacted by possible reductions in federal spending.● ● ● ● ● 5 ▸ Our business depends on compliance with ever-changing environmental and human health and safety laws.● ● ● ● ● 5 ▸ Our business faces significant government regulation, and there is no guarantee that our product candidates will receive regulatory approval.● ● ● ● ● 5 ▸ Our business involves risk associated with handling hazardous and other dangerous materials.● ● ● ● ● 5 ▸ Our development of a potential vaccine for COVID-19 or other indications is at an early stage and is subject to significant risks.● ● ● ● ● 5 ▸ Our employees and consultants may engage in misconduct or other improper activities, including noncompliance with regulatory standards and requirements.● ● ● ● ● 5 ▸ Our exosome technologies are based on a novel therapeutic approach which makes it difficult to predict the time and cost of development and the probability of subsequently obtaining regulatory approval, if at all.● ● ● ● ● 5 rw ▸ Our products face a risk of failure due to adverse immunological reactions.● ● ● ● ● 5 ▸ Our products may expose us to potential product liability, and there is no guarantee that we will be able to obtain and maintain adequate insurance to cover these liabilities.● ● ● ● ● 5 ▸ Our products will likely face intense competition.● ● ● ● ● 5 ▸ Our success depends upon the viability of our product candidates and we cannot be certain any of them will receive regulatory approval to be commercialized.● ● ● ● ● 5 ▸ Providing product for use in third-party trials or for compassionate use poses risks to our product candidates.● ● ● ● ● 5 rw ▸ group Risks Related to Clinical and Commercialization Activities● ● ● ● ● 5 ▸ group Risks Related to Competitive Factors● ● ● ● ● 5 ▸ group Risks Related to Our Business● ● ● ● ● 5 ▸ group Risks Related to Our Common Stock● ● ● ● ● 5 ▸ group Risks Related to Our Intellectual Property● ● ● ● ● 5 ▸ group Risks Related to Our Relationships with Third Parties● ● ● ● ● 5 ▸ group Risks Related to Product and Environmental Liability● ● ● ● ● 5 ▸ group Risks Related to the Manufacturing of our Product Candidates● ● ● ● ● 5 ▸ The Company’s ability to utilize Nile’s net operating loss and tax credit carryforwards in the future is subject to substantial limitations and may further be limited as a result of the merger with Capricor.● ● ● ● ● 5 ▸ The operational and other projections and forecasts that we may make from time to time are subject to inherent risks, many of which are beyond our control.● ● ● ● ● 5 ▸ The regulatory pathway for COVID-19 or other infectious disease vaccines is continually evolving and may result in unexpected or unforeseen challenges.● ● ● ● ● 5 rw ▸ The requirements of being a public company may strain our resources and divert management’s attention from operating our business.● ● ● ● ● 5 rw ▸ The third parties we use in the manufacturing process for our product candidates may fail to comply with cGMP regulations.● ● ● ● ● 5 ▸ There are additional risks involved in conducting clinical trials internationally.● ● ● ● ● 5 ▸ These provisions could make it more difficult for our stockholders to affect our corporate policies or make changes in our Board of Directors and for a third-party to acquire us, even if doing so would benefit our stockholders.● ● ● ● ● 5 rw ▸ Vaccines carry unique risks and uncertainties, which could have a negative impact on future results of operations.● ● ● ● ● 5 ▸ We are dependent on our relationships with our licensors and collaborators and there is no guarantee that such relationships will be maintained or continued.● ● ● ● ● 5 rw ▸ We depend on intellectual property licensed from third parties and termination of any of these licenses could result in the loss of significant rights, which would harm our business.● ● ● ● ● 5 ▸ We expect that our stock price will continue to fluctuate significantly.● ● ● ● ● 5 ▸ We face substantial competition, which may result in others discovering, developing or commercializing products before or more successfully than we do.● ● ● ● ● 5 ▸ We have limited experience in conducting late-stage clinical trials, which are complex and subject to strict regulatory oversight.● ● ● ● ● 5 rw ▸ We have never paid dividends and we do not anticipate paying dividends in the future.● ● ● ● ● 5 ▸ We have no experience selling, marketing, or distributing products and no current internal capability to do so.● ● ● ● ● 5 ▸ We have received government grants and a loan award which impose certain conditions on our operations.● ● ● ● ● 5 ▸ We may be at risk of securities class action litigation or litigation initiated by individual stockholders.● ● ● ● ● 5 ▸ We may be subject to claims that we or our employees, consultants or independent contractors have wrongfully used or disclosed confidential information of third parties.● ● ● ● ● 5 ▸ We may face uncertainty and difficulty in obtaining and enforcing our patents and other proprietary rights.● ● ● ● ● 5 ▸ We may incur substantial costs as a result of litigation or other adversarial proceedings relating to patent and other intellectual property rights and we may be unable to protect our rights to, or use of, our technology.● ● ● ● ● 5 ▸ We may issue shares of blank check preferred stock without stockholder approval in the future.● ● ● ● ● 5 ▸ We may not be able to file INDs to commence additional clinical trials on the timelines we expect, and even if we are ableto do so, the FDA may not permit us to proceed.● ● ● ● ● 5 ▸ We may not be successful in our efforts to identify or discover additional potential product candidates or additional indications for our existing product candidates.● ● ● ● ● 5 rw ▸ We will depend on our exclusive distributor, Nippon Shinyaku, for the commercial sale of Deramiocel in Duchenne muscular dystrophy (“DMD”) in the United States and Japan, if regulatory approval is obtained in those territories.● ● ● ● ● 5 rw ▸ You may experience future dilution as a result of future equity offerings.● ● ● ● ● 5 
