Global Cell Therapy Market Research Report 2021: Opportunities with the Approval of Kymriah and Yescarta Across Various Countries -…

DUBLIN--(BUSINESS WIRE)--The "Global Cell Therapy Market Size, Share & Trends Analysis Report by Use-type, by Therapy Type (Autologous, Allogenic), by Region (North America, Europe, Asia Pacific, Latin America, MEA), and Segment Forecasts, 2021-2028" report has been added to ResearchAndMarkets.com's offering.

The global cell therapy market size is expected to reach USD 23.0 billion by 2028 and is expected to expand at a CAGR of 14.5% from 2021 to 2028.

The emergence of new technologies to support the development of advanced cellular therapies has aided in market growth. Companies are leveraging new technologies not only for the expansion of their product portfolio but also for establishing out-licensing or co-development agreements with other entities to support their product development programs.

Cell-based therapies hold great potential for replacing, repairing, restoring, or regenerating damaged tissues, and organs. Researchers are making huge investments in the development of such effective and safe treatments as an alternative to conventional treatment strategies which can be further attributed to the market growth.

Out of all therapeutic areas, oncology has the highest number of ongoing clinical trials. T cells, CD34+ and/or CD133+ stem cells, mesenchymal stem/stromal cells are predominantly employed for clinical investigation.

The majority of biopharmaceutical entities have been affected by the COVID-19 pandemic, while several cellular therapy development companies have witnessed a strongly negative impact, which can be attributed to complications in logistics as well as the manufacturing models employed in this industry. In addition, substantial and stable funding is imperative to ensure successful commercial translation of cell-based therapeutics, a factor that was negatively affected in 2020, further affecting the market growth.

A survey conducted recently among executives of more than 15 European and U.S. cellular therapy companies indicated that disruption caused by the pandemic was significant, which demanded market entities to create strategies to sustain themselves and plan the next wave of innovative therapies.

Key issues faced by companies operating in the market include on-time delivery of therapies to patients at required clinical sites. In addition, the administration of these therapeutics poses several post-pandemic challenges. Hospitals are hesitant in offering services, owing to concerns over transmission of SARS-CoV-2, particularly to vulnerable individuals. Moreover, patients have not been able to visit cellular therapy centers either, owing to the lockdowns and travel bans.

Cell Therapy Market Report Highlights

Market Dynamics

Market driver analysis

Market restraint analysis

Market opportunity analysis

COVID-19 Impact Analysis

Challenges analysis

Opportunities analysis

Challenges in manufacturing cell therapies against COVID-19

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/n1u89x

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Global Cell Therapy Market Research Report 2021: Opportunities with the Approval of Kymriah and Yescarta Across Various Countries -...

Pursuing Optimal Therapy Remains a Challenge in Indolent B-Cell Lymphoma – Targeted Oncology

A significant proportion of patients worldwide live disease-free for a decade or longer following first-line therapy, with various approaches suggesting that there may be pathways to develop approaches for a cure for at least a subset of patients

Follicular lymphoma (FL) and other forms of indolent non-Hodgkin lymphoma (iNHL) are commonly considered incurable diseases with continual risk of relapse over time, shorter durations of response with each subsequent line of therapy, and a risk of transformation to a more aggressive B-cell lymphoma. Although these lymphomas are characterized as indolent, one of the most common causes of death for these patients is lymphoma.1 On the other hand, a significant proportion of patients worldwide live disease-free for a decade or longer following first-line therapy, with various approaches suggesting that there may be pathways to develop approaches for a cure for at least a subset of patients.1-5 In the Indolent B-Cell Lymphoma session during the Society of Hematologic Oncology 2021 Annual Meeting, 4 presentations were explored: Molecular Pathogenesis of Follicular Lymphoma and Its Relevance to Clinical Practice, Sequencing Therapy in Follicular Lymphoma, Novel Therapies in Indolent Lymphoma, and CAR T-Cell Therapy in Indolent Lymphoma in lectures by world-class experts Jessica Okosun, MA, MB BChir, MRCP, FRCPath, PhD, Peter Martin, MD, Loretta Nastoupil, MD, and Caron Jacobson, MD, MMSc.

Next-generation sequencing studies, led by Michael R. Green, PhD, Dr Okosun, and others, have described the panoply of genomic events involved in FL and have aided in identifying candidate genetic drivers.6-9 This work clarified the diverse genomic landscape and the temporal clonal dynamics of FL. Common genomic events that occur with t(14;18) include high frequencies of mutations affecting epigenetic regulation, disruptions in pathways such as those involved in immune recognition (eg, TNFRSF14), NF-KB (eg, CARD11), and JAK/STAT signaling (eg, STAT6).9,10 Next-generation sequencing also has been used to examine factors associated with progression of FL, transformation of iNHL to more aggressive lymphomas, and spatial heterogeneity in FL. Recent studies have explored the subclonal diversity and spatial heterogeneity observed among patients with FL that have potential clinical implications for the development of prognostic and predictive biomarkers and targeted treatment strategies. For instance, exome sequencing of FL tumors and paired germline have identified nonsynonymous somatic variants corresponding to missense (81%), indels (10%), nonsense (7%), and splice site (2%) changes.6 This work revealed spatially discordant mutations in genes such as EZH2 and EP300. One attractive treatment paradigm emerging from this work involves specifically targeting highly recurrent and truncal gene mutations that have roles in FL pathogenesis. Other studies did not identify a single compelling genetic event responsible for transformation, but instead suggest that the acquisition of certain genetic alterations may result in aggressive transformation.7,9,11 Exploration of the FL genomics in this session can clarify stratified treatment approaches targeting specific early genetic lesions identified in FL and may eventually provide strategies to eradicate these cell populations and provide pathways to cure FL.

In the past decade, many new agents have been introduced for the management of FL, and therapeutic strategies have evolved over time. Recently, my co-chair in this session, Nathan Fowler, MD, and I reviewed data from trials addressing the safety and efficacy of lenalidomide alone and in combination with rituximab as a first-line therapy and as a treatment of patients with relapsed/refractory FL.12 However, since that review other agents have received FDA approval for patients with relapsed FL. There is considerable variation in response rates for recently approved therapies ranging from objective response rates of 40% to 60% for PI3K inhibitors, 35% to 65% for EZH2 inhibitors, and greater than 70% for autologous stem cell transplantation, and CD19-directed chimeric antigen receptor (CAR) T-cell therapy.13-18 Recently approved therapies in relapsed FL have commonly been based on response rate and duration of response (DOR) demonstrated in phase 2 studies. However, despite numerous trials performed in the field, there is no single standard of care for patients with iNHL who are undergoing second-line treatment or beyond.

As a result of the patterns of relapse and transformation associated with iNHL, the clinical treatment of patients with FL and other iNHLs often requires multiple lines of therapy using various regimens with different mechanisms of action.19-22 The clinical benefits and adverse effects associated with the treatments available at relapse vary and are influenced by patient and disease characteristics at the time of progression, the duration of the interval from last treatment, and the toxicity and responses associated with the treatments previously administered. This results in a marked heterogeneity of clinical situations encountered during the treatment of these patients. Some patients with iNHL will remain well treated using available treatments, whereas others will develop disease refractory to conventional approaches and become candidates for novel treatments and clinical trials. Additional real-world data regarding patient characteristics at relapse, patterns of care, expectations of response rates and duration, and survival outcomes are lacking in the setting of relapsed and refractory iNHL. To help inform treatment decisions by health care providers treating patients with iNHL in this complex and evolving treatment landscape, Dr Martin will describe approaches for sequencing therapies. To optimally individualize treatment strategies for patients with previously untreated and relapsed iNHL, the risks and benefits of the available options should be well known. This lecture will enable providers to effectively discuss the goals of therapy with the patient at each intervention, which is also critical in providing an optimal sequence of therapy.

Although many patients with FL experience long or possibly near-normal life expectancies, there remains persistent variability in patient outcomes.19-22 Patients who relapse within 2 years of first-line chemoimmunotherapy or with histologic transformation are at risk for early mortality and are high-priority candidates for novel treatment strategies evaluated in clinical trials.23-25 Prior studies have demonstrated diminishing DOR by line of therapy.26,27

However, variability of iNHL disease biology, treatment options, and treatment patterns complicate outcome assessments based on line of therapy alone. Several novel and targeted therapies are being developed and evaluated in patients with relapsed iNHL, including cereblon inhibitors, antiCD20-CD3 bispecific antibodies, and additional anti-CD19 CAR T-cell therapies. Dr Nastoupil will provide key insights on the novel therapies available for patients in clinical trials and those that are establishing pathways toward applications in clinical settings.

CAR therapy targeting CD19 is one promising treatment for patients with relapsed or refractory FL and CD19+ iNHLs. Patients who are candidates for CAR T-cell therapy often have symptomatic disease that could be fatal if left untreated. Dr Jacobson will discuss strategies for bridging therapy, which may include chemotherapy, targeted therapy, or radiation therapy; approved and experimental CAR T-cell approaches for FL and iNHLs; and describe traditional and novel adverse events and outcomes from clinical trials involving CAR T-cell therapy.

REFERENCES:

1. Sarkozy C, Maurer MJ, Link BK, et al. Cause of death in follicular lymphoma in the first decade of the rituximab era: a pooled analysis of French and US cohorts. J Clin Oncol. 2019;37(2):144-152. doi:10.1200/JCO.18.00400

2. Bachy E, Seymour JF, Feugier P, et al. Sustained progression-free survival benefit of rituximab maintenance in patients with follicular lymphoma: long-term results of the PRIMA study. J Clin Oncol. 2019;37(31):2815-2824. doi:10.1200/JCO.19.01073.

3. Becnel MR, Nastoupil LJ, Samaniego F, et al. Lenalidomide plus rituximab (R 2 ) in previously untreated marginal zone lymphoma: subgroup analysis and long-term follow-up of an open-label phase 2 trial. Br J Haematol. 2019;185(5):874-882. doi:10.1111/bjh.15843

4. Strati P, Jain P, Johnson RJ, et al. Long-term follow-up of lenalidomide and rituximab as initial treatment of follicular lymphoma. Blood. 2021;137(8):1124-1129. doi:10.1182/blood.2020007994

5. Watanabe T, Tobinai K, Wakabayashi M, et al; JCOG0203 Collaborators. Outcomes after R-CHOP in patients with newly diagnosed advanced follicular lymphoma: a 10-year follow-up analysis of the JCOG0203 trial. Lancet Haematol. 2018;5(11):e520-e531. doi:10.1016/S2352-3026(18)30155-8

6. Araf S, Wang J, Korfi K, et al. Genomic profiling reveals spatial intra-tumor heterogeneity in follicular lymphoma [published correction appears in Leukemia. 2019;33(6):1540]. Leukemia. 2018;32(5):1261-1265. doi:10.1038/s41375-018-0043-y

7. Green MR, Gentles AJ, Nair RV, et al. Hierarchy in somatic mutations arising during genomic evolution and progression of follicular lymphoma. Blood. 2013;121(9):1604-1611. doi:10.1182/blood-2012-09-457283

8. Green MR, Kihira S, Liu CL, et al. Mutations in early follicular lymphoma progenitors are associated with suppressed antigen presentation. Proc Natl Acad Sci U S A. 2015;112(10):E1116-E1125. doi:10.1073/pnas.1501199112

9. Okosun J, Bdr C, Wang J, et al. Integrated genomic analysis identifies recurrent mutations and evolution patterns driving the initiation and progression of follicular lymphoma. Nat Genet. 2014;46(2):176-181. doi:10.1038/ng.2856

10. Kumar E, Pickard L, Okosun J. Pathogenesis of follicular lymphoma: genetics to the microenvironment to clinical translation. Br J Haematol. Published online March 10, 2021. doi:10.1111/bjh.17383

11. Okosun J, Montoto S, Fitzgibbon J. The routes for transformation of follicular lymphoma. Curr Opin Hematol. 2016;23(4):385-391. doi:10.1097/MOH.0000000000000255

12. Flowers CR, Leonard JP, Fowler NH. Lenalidomide in follicular lymphoma. Blood. 2020;135(24):2133-2136. doi:10.1182/blood.2019001751

13. Dreyling M, Santoro A, Mollica L, et al. Phosphatidylinositol 3-kinase inhibition by copanlisib in relapsed or refractory indolent lymphoma. J Clin Oncol. 2017;35(35):3898-3905. doi:10.1200/JCO.2017.75.4648

14. Flinn IW, Miller CB, Ardeshna KM, et al. DYNAMO: a phase II study of duvelisib (IPI-145) in patients with refractory indolent non-Hodgkin lymphoma. J Clin Oncol. 2019;37(11):912-922. doi:10.1200/JCO.18.00915

15. Gopal AK, Kahl BS, de Vos S, et al. PI3K inhibition by idelalisib in patients with relapsed indolent lymphoma. N Engl J Med. 2014;370(11):1008-1018. doi:10.1056/NEJMoa1314583

16. Jacobson C, Chavez JC, Sehgal AR, et al. Primary analysis of zuma-5: a phase 2 study of axicabtagene ciloleucel (axi-cel) in patients with relapsed/refractory (r/r) indolent non-Hodgkin lymphoma (iNHL). Blood. 2020;136(suppl 1):40-41. doi:10.1182/blood-2020-136834

17. Metzner B, Pott C, Mller TH, et al. Long-term clinical and molecular remissions in patients with follicular lymphoma following high-dose therapy and autologous stem cell transplantation. Ann Oncol. 2013;24(6):1609-1615. doi:10.1093/annonc/mds657

18. Morschhauser F, Tilly H, Chaidos A, et al. Tazemetostat for patients with relapsed or refractory follicular lymphoma: an open-label, single-arm, multicentre, phase 2 trial. Lancet Oncol. 2020;21(11):1433-1442. doi:10.1016/S1470-2045(20)30441-1

19. Flowers CR, Leonard JP, Nastoupil LJ. Novel immunotherapy approaches to follicular lymphoma. Hematology Am Soc Hematol Educ Program. 2018;2018(1):194-199. doi:10.1182/asheducation-2018.1.194

20. Leonard JP, Nastoupil LJ, Flowers CR. Where to start? Upfront therapy for follicular lymphoma in 2018. Hematology Am Soc Hematol Educ Program. 2018;2018(1):185-188. doi:10.1182/asheducation-2018.1.185

21. Nastoupil LJ, Flowers CR, Leonard JP. Sequencing of therapies in relapsed follicular lymphoma. Hematology Am Soc Hematol Educ Program. 2018;2018(1):189-193. doi:10.1182/asheducation-2018.1.189

22. Salles G. How do I sequence therapy for follicular lymphoma? Hematology Am Soc Hematol Educ Program. 2020;2020(1):287-294. doi:10.1182/hematology.2020000156

23. Casulo C, Byrtek M, Dawson KL, et al. Early relapse of follicular lymphoma after rituximab plus cyclophosphamide, doxorubicin, vincristine, and prednisone defines patients at high risk for death: an analysis from the National LymphoCare Study. J Clin Oncol. 2015;33(23):2516-2522. doi:10.1200/JCO.2014.59.7534

24. Casulo C, Friedberg JW, Ahn KW, et al. Autologous transplantation in follicular lymphoma with early therapy failure: a National LymphoCare Study and Center for International Blood and Marrow Transplant Research analysis. Biol Blood Marrow Transplant. 2018;24(6):1163-1171. doi:10.1016/j.bbmt.2017.12.771

25. Casulo C, Nastoupil L, Fowler NH, Friedberg JW, Flowers CR. Unmet needs in the first-line treatment of follicular lymphoma. Ann Oncol. 2017;28(9):2094-2106. doi:10.1093/annonc/mdx189

26. Batlevi CL, Sha F, Alperovich A, et al. Follicular lymphoma in the modern era: survival, treatment outcomes, and identification of high-risk subgroups. Blood Cancer J. 2020;10(7):74. doi:10.1038/s41408-020-00340-z

27. Link BK, Day BM, Zhou X, et al. Second-line and subsequent therapy and outcomes for follicular lymphoma in the United States: data from the observational National LymphoCare Study. Br J Haematol. 2019;184(4):660-663. doi:10.1111/bjh.15149

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Pursuing Optimal Therapy Remains a Challenge in Indolent B-Cell Lymphoma - Targeted Oncology

Stem Cell Therapy Market Research Report by Cell Source, by Type, by Therapeutic Application, by End-User, by Region – Global Forecast to 2026 -…

Stem Cell Therapy Market Research Report by Cell Source (Adipose tissue-derived MSCs (mesenchymal stem cells),, Bone marrow-derived MSCs,, and Placental/umbilical cord-derived MSCs), by Type (Allogeneic Stem Cell Therapy and Autologous Stem Cell Therapy), by Therapeutic Application, by End-User, by Region (Americas, Asia-Pacific, and Europe, Middle East & Africa) - Global Forecast to 2026 - Cumulative Impact of COVID-19

New York, Oct. 13, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Stem Cell Therapy Market Research Report by Cell Source, by Type, by Therapeutic Application, by End-User, by Region - Global Forecast to 2026 - Cumulative Impact of COVID-19" - https://www.reportlinker.com/p06175517/?utm_source=GNW

The Global Stem Cell Therapy Market size was estimated at USD 202.87 million in 2020 and expected to reach USD 240.88 million in 2021, at a CAGR 19.07% to reach USD 578.27 million by 2026.

Market Statistics: The report provides market sizing and forecast across five major currencies - USD, EUR GBP, JPY, and AUD. It helps organization leaders make better decisions when currency exchange data is readily available. In this report, the years 2018 and 2019 are considered historical years, 2020 as the base year, 2021 as the estimated year, and years from 2022 to 2026 are considered the forecast period.

Market Segmentation & Coverage: This research report categorizes the Stem Cell Therapy to forecast the revenues and analyze the trends in each of the following sub-markets:

Based on Cell Source, the market was studied across Adipose tissue-derived MSCs (mesenchymal stem cells),, Bone marrow-derived MSCs,, and Placental/umbilical cord-derived MSCs.

Based on Type, the market was studied across Allogeneic Stem Cell Therapy and Autologous Stem Cell Therapy.

Based on Therapeutic Application, the market was studied across Cardiovascular Diseases Surgeries, Inflammatory & Autoimmune Diseases, Musculoskeletal Disorders, Neurological Disorders, Other Therapeutic Applications, and Wounds & Injuries.

Based on End-User, the market was studied across Academic and Research Centers, Ambulatory Surgical Centers (ASCs), and Hospitals & Clinics.

Based on Region, the market was studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas is further studied across Argentina, Brazil, Canada, Mexico, and United States. The United States is further studied across California, Florida, Illinois, New York, Ohio, Pennsylvania, and Texas. The Asia-Pacific is further studied across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, Singapore, South Korea, Taiwan, and Thailand. The Europe, Middle East & Africa is further studied across France, Germany, Italy, Netherlands, Qatar, Russia, Saudi Arabia, South Africa, Spain, United Arab Emirates, and United Kingdom.

Cumulative Impact of COVID-19: COVID-19 is an incomparable global public health emergency that has affected almost every industry, and the long-term effects are projected to impact the industry growth during the forecast period. Our ongoing research amplifies our research framework to ensure the inclusion of underlying COVID-19 issues and potential paths forward. The report delivers insights on COVID-19 considering the changes in consumer behavior and demand, purchasing patterns, re-routing of the supply chain, dynamics of current market forces, and the significant interventions of governments. The updated study provides insights, analysis, estimations, and forecasts, considering the COVID-19 impact on the market.

Competitive Strategic Window: The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies to help the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. It describes the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth during a forecast period.

FPNV Positioning Matrix: The FPNV Positioning Matrix evaluates and categorizes the vendors in the Stem Cell Therapy Market based on Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Market Share Analysis: The Market Share Analysis offers the analysis of vendors considering their contribution to the overall market. It provides the idea of its revenue generation into the overall market compared to other vendors in the space. It provides insights into how vendors are performing in terms of revenue generation and customer base compared to others. Knowing market share offers an idea of the size and competitiveness of the vendors for the base year. It reveals the market characteristics in terms of accumulation, fragmentation, dominance, and amalgamation traits.

Competitive Scenario: The Competitive Scenario provides an outlook analysis of the various business growth strategies adopted by the vendors. The news covered in this section deliver valuable thoughts at the different stage while keeping up-to-date with the business and engage stakeholders in the economic debate. The competitive scenario represents press releases or news of the companies categorized into Merger & Acquisition, Agreement, Collaboration, & Partnership, New Product Launch & Enhancement, Investment & Funding, and Award, Recognition, & Expansion. All the news collected help vendor to understand the gaps in the marketplace and competitors strength and weakness thereby, providing insights to enhance product and service.

Company Usability Profiles: The report profoundly explores the recent significant developments by the leading vendors and innovation profiles in the Global Stem Cell Therapy Market, including Advanced Cell Technology, Inc., AlloSource, Inc., Anterogen Co., Ltd., Bioheart Inc., BioTime, Inc., BrainStorm Cell Therapeutics Inc., Celgene Corporation, Cellartis AB, CellGenix GmbH, Cellular Engineering Technologies Inc., Gamida Cell Ltd, Gilead Sciences, Inc., Holostem Terapie Avanzate Srl, JCR Pharmaceuticals Co., Ltd., Lonza Group AG, Medipost Co., Ltd., Nuvasive, Inc., Osiris Therapeutics, Inc., Pharmicell Co., Ltd., Pluristem Therapeutics Inc., PromoCell GmbH, RTI Surgical, Inc., STEMCELL Technologies, Inc., Takeda Pharmaceutical Company Limited, Vericel Corporation, and VistaGen Therapeutics, Inc..

The report provides insights on the following pointers: 1. Market Penetration: Provides comprehensive information on the market offered by the key players 2. Market Development: Provides in-depth information about lucrative emerging markets and analyze penetration across mature segments of the markets 3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments 4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, certification, regulatory approvals, patent landscape, and manufacturing capabilities of the leading players 5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and breakthrough product developments

The report answers questions such as: 1. What is the market size and forecast of the Global Stem Cell Therapy Market? 2. What are the inhibiting factors and impact of COVID-19 shaping the Global Stem Cell Therapy Market during the forecast period? 3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Stem Cell Therapy Market? 4. What is the competitive strategic window for opportunities in the Global Stem Cell Therapy Market? 5. What are the technology trends and regulatory frameworks in the Global Stem Cell Therapy Market? 6. What is the market share of the leading vendors in the Global Stem Cell Therapy Market? 7. What modes and strategic moves are considered suitable for entering the Global Stem Cell Therapy Market? Read the full report: https://www.reportlinker.com/p06175517/?utm_source=GNW

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Harvard’s R&D alliance with Resilience to advance manufacture of complex medicines – Harvard Gazette

Harvard University and National Resilience, Inc. (Resilience), a manufacturing and technology company, have established a five-year R&D alliance with a $30 million commitment from Resilience directed toward the development of complex medicines, including biologics, vaccines, nucleic acids, and cell and gene therapies.

Under the alliance agreement coordinated by Harvards Office of Technology Development (OTD), Resilience will fund faculty-initiated research focused on certain novel therapeutic and biomanufacturing technologies pioneered in University labs. The alliance also anticipates that these Harvard innovations may be commercially advanced by new companies formed by Resilience expressly to drive these technologies into clinical development and commercialization.

An initial technology platform has already been identified for incubation under the alliance, with promising applications in skeletal muscle disorders. In the Harvard lab of Lee Rubin, professor of Stem Cell and Regenerative Biology, researchers have developed a means to culture millions of cells in vitro that behave like skeletal muscle stem cells (satellite cells), retaining their regenerative potential, for use in possible cell therapies. Resilience is now funding the labs continuing work on the platform, aiming to further validate it, in a project led by staff scientist Feodor Price.

Meanwhile, Resilience has formed an entity called Circle Therapeutics, anticipating that Circle may carry the technology forward under license.

For six decades since the discovery of the satellite cell, it has not been possible to expand therapeutic numbers of satellite cells in vitro, until we made real headway on it at Harvard, said Rubin. Were truly excited for the possible therapeutic impact of our innovations.

Our mission at Resilience is to make a new generation of complex medicines, such as curative gene therapies, life-saving vaccines and immune-system-boosting cell therapies, more accessible to people in need, said Rahul Singhvi, chief executive officer of Resilience. Current biomanufacturing processes pose significant hurdles to making these medicines quickly, and at scale, which is why we are excited to work with researchers at Harvard to identify and develop the technologies needed to make this future a reality.

The Rubin Labs platform to expand and maintain in vitro-derived satellite cells could lead to transformative cell therapies, said Vivian Berlin, executive director, HMS, at Harvard OTD, who leads OTDs Corporate Alliances team. With prior support from the Blavatnik Biomedical Accelerator, the team has compellingly demonstrated the clinical relevance of this work. Now with Resiliences focused funding and experience in the development of complex medicines, we hope to set it on a clear path toward benefiting patients.

Going forward, Resilience and Harvard will jointly issue a call for proposals to identify additional research projects to be funded at Harvard. Under the terms of the alliance, Resilience will receive an option to license technologies arising from funded projects.

This research alliance with Resilience will help support biomedical innovation at Harvard, said Isaac Kohlberg, Harvards chief technology development officer and senior associate provost. Collaborating to both advance Harvard science and place arising technologies with dedicated new ventures, we can provide yet another valuable source of support and industry expertise to translational biomedical researchers across Harvards Schools as they seek to impact human health for the better.

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Harvard's R&D alliance with Resilience to advance manufacture of complex medicines - Harvard Gazette

A New Line of Defense in Blood Cancer: Natural Killer Cell Therapy – Curetoday.com

Bob Schulz knew something was wrong when he had a hard time walking up the hill while golfing in December 2018. At 73, he still walked the 18 holes at the Albuquerque, New Mexico, golf course every week. After a chest X-ray, his doctor sent him to the hospital immediately. Two liters of fluid were removed from Scholzs lungs several times during his four-day hospital stay. Extensive testing revealed malignant pleural effusion, or excess fluid and cancer cells between the tissues separating the lungs from the chest cavity.

Scholz sought a second opinion at The University of Texas MD Anderson Cancer Center, in Houston, a 13-hour drive away. There, he received a diagnosis of diffuse large B-cell lymphoma. He and his wife, Cindy, quickly packed up and moved to Houston for six months of R-CHOP chemotherapy, a combination of five drugs infused to kill cancer cells.

After chemotherapy, Scholz thought he was cancer-free, but in late 2020 he lost his voice completely, which sent him back to his oncologist at MD Anderson. A positron emission tomography scan revealed a recurrence of lymphoma in his throat, lung and liver. This time his doctor offered him treatment through a clinical trial for natural killer (NK) cell therapy, a type of infusion therapy that uses the bodys natural killer immune cells or donor NK cells, which are grown into larger quantities and sometimes genetically engineered with additional targeting abilities.

NK cells are a type of white blood cell in the immune system that can kill cancer and virally infected cells. They have the innate ability to recognize and attack cells infected with viruses or cancer cells, says Dr. Sarah Holstein, a multiple myeloma researcher and an associate professor of internal medicine at the University of Nebraska Medical Center in Omaha. However, cancer cells can sometimes evade NK cells ability to interact with and kill cancer cells. The idea behind NK cell therapy is to augment the bodys natural NK cell response and increase it and, hopefully, lead to a more direct cell-killing effect against the cancer cell, she explains.

Over the past two decades, researchers have studied various ways to do this; for example, by collecting the patients NK cells, growing them and then reinfusing them. When using the patients cells, its called an autologous adoptive transfer. Doctors also are growing cells from donors, called allogeneic adoptive transfer. These cells come from sources such as cell lines, peripheral blood or pluripotent stem cells, which can be found in neonatal foreskin or the umbilical cord, for example. Pluripotent stem cells have the ability to differentiate into many types of mature cells and can develop into NK cells or other needed cell types. One cell in the lab can produce millions of NK cells, says Dr. Paolo Strati, an assistant professor in the department of lymphoma and myeloma and the department of translational molecular pathology at MD Anderson Cancer Center. More recently the field has evolved to study genetically engineered NK cells, such as chimeric antigen receptor (CAR)-NK cells, that have the ability to recognize a specific target on the cancer cell.

Following three days of chemotherapy to prepare his immune system, the doctors gave Scholz three infusions of modified NK cells. He finished his treatment in early 2021 and is in remission. Im thankful every day about how fortunate I was to go there. Im thankful to have that kind of a place with treatments with that chance of success, he says.

A Growing Research Field

Dr. Jeffrey Miller, a professor of medicine in the division of hematology, oncology and transplantation at the University of Minnesota in Minneapolis, has been researching NK cell treatments for more than 25 years. He published a paper in 2005 about administering haploidentical allogeneic NK cells, which were taken from a related donor, to patients. The research showed that the cells can persist and expand in the body and may have a treatment role. His 2014 update, which was published in Blood, included 57 patients with relapsed/refractory acute myeloid leukemia (AML). Researchers used the immunotoxin interleukin (IL)-2 diphtheria toxin fusion to deplete T regulatory cells and thereby help improve NK cell growth rates. In the study, successful NK cell expansion correlated with remission. Patients were given NK cells, cytokines and lymphodepleting therapy.

There was excitement in the field when we started to see (complete) response rates between 25% and 40% with those updates, Miller says. These were patients who progressed after standard therapy and had no other options. The response allowed some patients to become eligible for allogeneic bone marrow transplants, even when they were not previously eligible.

Today, researchers are trying different trial designs, including an NK multidose strategy from allogeneic cells. We couldnt do it when we had to collect cells from individual donors. That only gave us one cell dose, Miller explains. Allogeneic cells can be expanded much faster, allowing for multiple doses and freezer storage until needed. Some trials are now giving up to six weekly doses of these off-the- shelf cell products, and doctors can infuse the cells in an cell expansion correlated with remission. Patients were given NK cells, cytokines and lymphodepleting therapy.

There was excitement in the field when we started to see (complete) response rates between 25% and 40% with those updates, Miller says. These were patients who progressed after standard therapy and had no other options. The response allowed some patients to become eligible for allogeneic bone marrow transplants, even when they were not previously eligible.

Today, researchers are trying different trial designs, including an NK multidose strategy from allogeneic cells. We couldnt do it when we had to collect cells from individual donors. That only gave us one cell dose, Miller explains. Allogeneic cells can be expanded much faster, allowing for multiple doses and freezer storage until needed. Some trials are now giving up to six weekly doses of these off-the- shelf cell products, and doctors can infuse the cells in an outpatient clinic instead of during a hospital stay. The cells are thawed at the bedside and given, and the patients are watched for a few hours for allergic reactions, Miller says.

The idea behind multidosing is that NK cells dont persist in the body for as long as T cells, which are used in CAR-T cell therapy. Think of it as a living drug, Holstein says. Once you put them in, those engineered cells persist and continue to fight against the tumor, should there be any remaining tumor cells that flare up again. Researchers dont think the NK cells can live as long as T cells, but we dont know if they need to live that long. Perhaps theyre super effective early on and we dont need them to persist, Holstein says.

In her multiple myeloma research, Holstein led a study that explored the use of off-the-shelf NK cell therapy given shortly after the time of a stem cell transplant. There are data showing that early recovery of the patients own NK cells after a stem cell transplant is associated with improved outcomes. It is hypothesized that this early recovery of NK cells is contributing to the killing off of residual myeloma cells, she says. By giving multiple doses of off-the-shelf NK cells or allogeneic cells researchers are hoping to boost the effect, ensuring that theres enough time for NK cells to attack any errant myeloma cells during the critical bone marrow recovery time. At this time, were not sure yet if this approach is effective, Holstein explains.

Although more recent trials are studying multiple dosing, earlier trials such as Holsteins used one dose. Thats partly because it was difficult to grow enough cells for multiple doses per patient, even using donor cells. Nancy Gessmann was 59 years old when she enrolled in Holsteins earlier trial in 2017.

She hadnt heard of multiple myeloma before back problems and a fever sent her to her primary care doctor in Harlan, Iowa, in 2016. After receiving her diagnosis, Gessmann sought treatment an hour away at the University of Nebraska Medical Center, where she received chemotherapy followed by a stem cell transplant in May 2017.

During her 18 days in the hospital for the transplant, she received a single dose of allogeneic NK cells as part of Holsteins phase 1 study, along with a series of seven cytokine shots (they help stimulate the NK cells) to help the cells expand. It gave me hope that if there was anything out there that could help me, it was worth trying, she explains. Aside from feeling tired after the transplant and growth factor shots which are given to aid the therapy Gessmann does not think she experienced any side effects from the NK cell infusion.

With the clinical trial, I had the opportunity to possibly help myself, my family and others. I benefited from clinical research done by others before me with stem cell transplants and chemotherapies. Others helped my treatment plan and made it easier for me. Im paying it forward, Gessmann says.

CAR-T Versus NK Cell Therapy

NK cell therapy may have advantages over T cells. Infused CAR-T cells will recognize a cancer cell and attack it. One attack method involves releasing toxins called cytokines, which can lead to a hyperinflammatory state known as cytokine release syndrome (CRS). CRS is caused when a large number of cytokines, proteins made by some immune cells, are quickly released into the blood from immune cells. They can lead to CRS symptoms such as fever, but patients can also experience low blood pressure, low blood oxygen and neuro- toxicities such as difficulty finding words, and severe issues such as a seizure or coma. About 10% of patients receiving CAR-T cell therapy for lymphoma experience severe CRS, and 40% experience severe neurotoxity. Its a real problem; hence, we need to look into different treatments, Strati says.

NK cells potentially can be less toxic than, and as effective as, T-cell therapy. Treatment for me was extremely easy, and the results were great, Scholz says. It wasnt like serious chemotherapies. I didnt feel real good for a couple of days, but it was minor. There were no repercussions from treatment.

The good thing about NK cells compared with T cells, Miller says, is that NK cells dont induce graft-versus-host disease, which is when infused allogeneic T cells attack the patients healthy cells. NK cells are missing the mechanism in T cells that cause it. For NK therapy, as far as we know, no known neurotoxicity or CRS has been reported in any consistent way today, Miller says.

The CAR technology also is being used for some NK cell treatments. With CAR, we engineer NK cells in the lab, Strati says. We make them able to recognize specific proteins on top of lymphoma. Using donor cells, both CAR-T and CAR NK cells can be available to patients more quickly than the patients cells.

The first in-human trial in the United States with CAR NK cells was for relapsed/refractory CD19-positive B lymphoid malignancies. The trial encoded NK cells to recognize CD19 and express cytokine IL-15 to improve persistence. Results were published in a 2020 New England Journal of Medicine study, and it continues to receive a lot of attention, Holstein says. The phase 1 and 2 study showed proof of concept that CAR-NK therapy is possible and effective. Of the 11 patients, 8 had a response and 7 had a complete remission.

The Future of NK Cell Therapy

Researchers developed data for NK cells having a similar cancer-killing strategy but different recognition pattern as T cells, leading to a crazy interest in NK cells, Miller says. Until the past decade, people mostly ignored NK cells.

Its not just academic labs pursuing them but also cell companies with their own constructs and expansion strategies. The field opened up considerably with the ability to grow billions of cells for off-the-shelf usage in the past 10 years.

Given the multibillion dollar market for anticancer anti- body therapy and the ability of cell therapy companies to genetically manipulate cells with CARs, I would expect were going to see somebody close to clinical approval in the next three to five years, Miller says.

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Dr. Kahl on the Potential Utility of Frontline CAR T-Cell Therapy in MCL – OncLive

Brad S. Kahl, MD, discusses the potential utility of frontline CAR T-cell therapy in mantle cell lymphoma.

Brad S. Kahl, MD, professor of medicine, Department of Medicine, Oncology Division, Medical Oncology, Washington University School of Medicine in St. Louis, discusses the potential utility of frontline CAR T-cell therapy in mantle cell lymphoma (MCL).

CAR T-cell therapy has the potential to move into earlier lines of treatment, including the frontline setting, in MCL; however, longer follow-up is needed with CAR T-cell therapy in the relapsed/refractory setting before frontline clinical trials can be explored, Kahl says. Currently, CAR T-cell therapy is demonstrating high response rates at 12 and 18 months of follow-up in the relapsed/refractory setting, but it is unknown whether these responses will remain durable at 3 or 5 years.

Positive 3-year data in the relapsed/refractory setting could provide the clinical rationale to evaluate CAR T-cell therapy in the frontline setting, Kahl says. Pending these results, CAR T-cell therapy could replace autologous stem cell transplant as consolidative therapy or offer a standard option for patients with high-risk biologic features, such as TP53 mutations, Kahl concludes.

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Dr. Kahl on the Potential Utility of Frontline CAR T-Cell Therapy in MCL - OncLive

Rheumatoid Arthritis Stem Cell Therapy Market By Type (Allogeneic Mesenchymal Stem Cells, Bone Marrow Transplant, Adipose Tissue Stem Cells) and By…

250 Pages Rheumatoid Arthritis Stem Cell Therapy Market Survey by Fact MR, A Leading Business and Competitive Intelligence Provider

Rheumatoid arthritis stem cell therapy has been demonstrated to induce profound healing activity, halt arthritic conditions, and in many cases, reverse and regenerate joint tissue. Today, bone marrow transplant, adipose or fat-derived stem cells, and allogeneic mesenchymal stem cells (human umbilical cord tissue) are used for rheumatoid arthritis stem cell therapy.

The Market Research Survey by Fact.MR, highlights the key reasons behind increasing demand and sales of Rheumatoid Arthritis Stem Cell Therapy.Rheumatoid Arthritis Stem Cell Therapy market driversand constraints, threats and opportunities, regional segmentation and opportunity assessment, end-use/application prospects review are addressed in the Rheumatoid Arthritis Stem Cell Therapy market survey report. The survey report provides a comprehensive analysis of Rheumatoid Arthritis Stem Cell Therapy market key trends and insights on Rheumatoid Arthritis Stem Cell Therapy market size and share.

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Rheumatoid Arthritis Stem Cell Therapy Market By Type (Allogeneic Mesenchymal Stem Cells, Bone Marrow Transplant, Adipose Tissue Stem Cells) and By...

Orchard Therapeutics Outlines Comprehensive Presence at the European Society of Gene & Cell Therapy Congress – Yahoo Finance

Nine accepted abstracts demonstrate broad potential of the companys HSC gene therapy approach to treat severe neurodegenerative diseases and immunological disorders

BOSTON and LONDON, Oct. 13, 2021 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the acceptance of nine abstracts at the upcoming European Society of Gene & Cell Therapy Congress (ESGCT) taking place virtually from October 19-22.

Clinical and pre-clinical data from across the companys hematopoietic stem cell (HSC) gene therapy portfolio will be featured in two oral and seven poster presentations, including an update on the ongoing proof-of-concept study of OTL-201 for the treatment of Mucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A), pre-clinical data from OTL-204 in frontotemporal dementia (FTD), as well as proof-of-principle for longitudinal monitoring of vector integration sites using Liquid Biopsy Integration Site sequencing (LiBIS-seq).

Additionally, Orchards scientific advisory board member and clinical collaborator Alessandra Biffi, M.D., professor of pediatrics, University of Padua and chief of the Pediatric Onco-hematology Unit of Padua Hospital, will be giving an invited presentation on the HSC gene therapy landscape for the treatment of neurodegenerative disorders, which will include an overview of several of the companys investigational programs.

The presentations are listed below, and the full program is available online on the ESGCT website. All times are Central European Summer Time (CEST).

Oral Presentation Details:

Haematopoietic reconstitution dynamics of mobilized peripheral blood- and bone marrow-derived haematopoietic stem/progenitor cells after gene therapy Presenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene Therapy Abstract Number: OR049 Date/Time: Friday, October 22, 2021 at 10:01 CEST

Longitudinal monitoring of vector integration sites in in vivo GT approaches by Liquid-Biopsy-Integration-Site-Sequencing Presenting Author: Daniela Cesana, Ph.D., San Raffaele Telethon Institute for Gene Therapy Abstract Number: OR058 Date/Time: Friday, October 22, 2021 at 12:46 CEST

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Poster Presentation Details:

All posters will be available on demand starting October 19, 2021 on the ESGCT website.

Development of an ex vivo Gene Therapy for Frontotemporal Dementia (FTD) Presenting Author: Yuri Ciervo, Ph.D., division of pediatric Hematology,Oncology and Stem Cell Transplantation, Womans and Child Health Department, University of Padova, Padova, Italy Abstract Number: P077

Optimized Lentiviral Transduction Process for ex vivo CD34+ Hematopoietic Stem Cell Gene Therapy Drug Product Manufacture Presenting Author: Saranya Elavazhagan, Orchard Therapeutics Abstract Number: P271

Clinical Trial Update: Ex-vivo autologous stem cell gene therapy in MPSIIIA Presenting Author: Brian Bigger, Ph.D., University of Manchester Abstract Number: P361

Dissecting bone remodelling mechanisms and hematopoietic stem cell gene therapy impact in Mucopolysaccharidosis type I Hurler bone defects Presenting Author: Ludovica Santi, Ph.D., San Raffaele Telethon Institute for Gene Therapy Abstract Number: P157

Hematopoietic reconstitution and lineage commitment in HSC GT patients are influenced by the disease background Presenting Author: Andrea Calabria, Ph.D., San Raffaele Telethon Institute for Gene Therapy Abstract Number: P181

Kinetics and composition of haematopoietic stem/progenitors mobilized cells upon G-CSF and Plerixafor administration in transplant donor or patients undergoing autologous gene therapy Presenting Author: Luca Basso-Ricci, San Raffaele Telethon Institute for Gene Therapy Abstract Number: P174

Role of peripheral blood circulating haematopoietic stem/progenitor cells during physiological haematopoietic maturation and after gene therapy Presenting Author: Pamela Quaranta, San Raffaele Telethon Institute for Gene Therapy Abstract Number: P186

About Orchard Therapeutics At Orchard Therapeutics, our vision is to end the devastation caused by genetic and other severe diseases. We aim to do this by discovering, developing and commercializing new treatments that tap into the curative potential of hematopoietic stem cell (HSC) gene therapy. In this approach, a patients own blood stem cells are genetically modified outside of the body and then reinserted, with the goal of correcting the underlying cause of disease in a single treatment.

In 2018, the company acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Today, Orchard has a deep pipeline spanning pre-clinical, clinical and commercial stage HSC gene therapies designed to address serious diseases where the burden is immense for patients, families and society and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Contacts

Investors Renee Leck Director, Investor Relations +1 862-242-0764 Renee.Leck@orchard-tx.com

Media Benjamin Navon Director, Corporate Communications +1 857-248-9454 Benjamin.Navon@orchard-tx.com

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Orchard Therapeutics Outlines Comprehensive Presence at the European Society of Gene & Cell Therapy Congress - Yahoo Finance

Dr. Erba on the Evolution of Treatment in MCL – OncLive

Harry Paul Erba, MD, PhD, discusses the evolution of treatment in mantle cell lymphoma.

Harry Paul Erba, MD, PhD, instructor, clinical investigator, Division of Hematologic Malignancies and Cellular Therapy, Department of Medicine, Duke University School of Medicine, member, Duke Cancer Institute, director, Leukemia Program and Phase I Development in Hematologic Malignancies, Duke Health, discusses the evolution of treatment in mantle cell lymphoma (MCL).

Similar to acute myeloid leukemia, the goal of therapy in MCL should be the key focus from treatment initiation. For example, a younger patient with limited comorbidities should be considered for curative-intent therapy or treatment with a time-limited regimen to elicit deep responses and prolonged progression-free survival, Erba says.

High-dose cytarabine-based therapies, such as the Nordic regimen known as maxi-CHOP, and autologous stem cell transplant could be considered for patients with MCL, Erba explains. Oral therapies, including BTK inhibitors, are also available options for patients with relapsed/refractory MCL or older patients who cannot tolerate intensive chemotherapy, Erba concludes.

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Dr. Erba on the Evolution of Treatment in MCL - OncLive

Taking aim at the brain, Takeda strikes up cell therapy R&D alliance with Immusoft – MedCity News

The first cell therapies made from a patients own cells were cancer treatments T cells engineered to hit tumors. Immusoft is taking a similar approach with a different type of immune cell, aiming to deliver these cell therapies into the brain. The biotech startup is still preclinical, but Takeda Pharmaceutical sees enough promise to begin a research and development alliance that could yield new treatments for rare neurometabolic diseases.

Seattle-based Immusoft develops its treatments by reprogramming B cells, a type of white blood cell. The process for making this treatment is nearly identical to that of CAR T, the first autologous cancer cell therapies. Blood is collected from the patient and the desired immune cells are selected. Those cells are engineered, then multiplied in a lab. After the process produces enough cells, that treatment is infused into the patient.

Besides the type of cell that is used, the key difference between CAR T and Immusofts approach is the engineering step. In CAR T, this stage involves engineering T cells to recognize a particular antigen on tumors. For Immusoft, this step means programing B cells with DNA that enables them to produce large amounts of therapeutic protein. Immusoft calls its technology Immune System Programing, or ISP.

Takeda and Immusoft did not specify the diseases they aim to address under their new alliance, though the companies said the collaboration will focus on delivering therapies across the blood-brain barrier, the protective layer that keeps certain substances, including some drugs, from reaching the organ. By reaching the brain, these B cell therapies have the potential to address a range of neurological disorders.

We continue to build our internal capabilities as well as partner with innovative companies early on in the discovery process to advance our next-generation gene and cell therapy ambitions for rare genetic and hematologic diseases, Takeda Rare Diseases Drug Discovery Unit Head Madhu Natarajan said in a prepared statement. Working together with Immusoft, we hope to validate their ISP technology for [central nervous system] delivery of innovative therapeutics for rare neurometabolic diseases.

Rare neurometabolic disease is already one of the areas of focus for Immusoft. The rare enzyme deficiency Hurler syndrome is the target for its most advanced internal program, ISP-001. That cell therapy candidate is on track for an investigational new drug application filing by the end of this year, according to the companys website.

According to deal terms announced Wednesday, Takeda will pay Seattle-based Immusoft an upfront payment as well as research funding. The specific amounts were not disclosed. When the drug candidates covered under the partnership reach preclinical development, Takeda may elect to choose an unspecified number of them to continue their development. The Japanese pharma giant would owe option fees plus milestone payments tied to the progress of those programs. The companies gave no specific breakdown of those payments, other than to say the total value could top $900 million if all options are exercised and all milestones are achieved.

Working with B cells offer several advantages over other approaches to treating disease. Gene therapies delivered via engineered viruses cant be re-dosed because the antibodies that patients develop to the virus will render subsequent doses ineffective. By using a patients own cells, Immusoft aims to produce therapies that can be dosed multiple times. Also, gene therapies made by collecting a patients stem cells require a preconditioning step that knocks out the immune system to make room for the transplanted stem cells to grow. This step opens the door to a range of potential complications. Takedas recent dealmaking shows the pharma giants interest in avoiding therapies that employ viral delivery. In unveiling a multi-program alliance with Poseida Therapeutics on Tuesday, the pharma giant cited the biotechs non-viral technologies.

There are other companies developing ways to engineer B cells into new therapies. South San Francisco-based Walking Fish Therapeutics unveiled a $50 million Series A round of funding last month to support its research, still preclinical. Be Biopharma of Boston emerged nearly a year ago with a $52 million Series A financing.

Immusofts research so far has produced a preclinical drug pipeline spanning both rare and common diseases. Besides the Hurler syndrome candidate, the other rare disease programs include potential treatments for muscle-wasting disorders amyotrophic lateral sclerosis and Duchenne muscular dystrophy; the metabolic disease Hunter syndrome; and Pompe and Gaucher diseases, both enzyme deficiency disorders. Immusofts common disease research is still in the discovery stage. For common diseases, Immusoft is developing treatments for cardiovascular disorders, rheumatoid arthritis, and Parkinsons disease.

Image by Jolygon via Getty Images

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Taking aim at the brain, Takeda strikes up cell therapy R&D alliance with Immusoft - MedCity News