Category Archives: Cell Therapy

AUSTIN, Texas & HAMILTON, Ontario--(BUSINESS WIRE)--

Designation Confirms Significant Unmet Need Remains in DLBCL

Triumvira Immunologics, Inc. (Triumvira), a private, clinical-stage biopharmaceutical company developing a novel platform for engineering T-cells to attack cancers, today announced that the U.S. Food & Drug Administration (FDA) has granted Fast Track designation for its novel T-cell therapy product TAC01-CD19 in patients with relapsed or refractory Diffuse Large B-Cell Lymphoma (DLBCL) after at least 2 prior systemic therapies. A Phase 1/2 study (TACTIC-19) conducted in patients with CD19-positive B-cell malignancies, including DLBCL, is expected to be initiated by the end of 2019.

FDAs decision to grant Fast Track designation to TAC01-CD19 is an important recognition of both Triumviras differentiated cell therapy technology and the critical need to develop new therapies to address the unmet medical need in the treatment of B-cell lymphomas, commented Paul Lammers, MD, MSc., President and CEO of Triumvira. With our innovative TAC technology, we hope to significantly improve upon the limitations of existing cell therapies, including the risk of cytokine release syndrome (CRS) and neurotoxicity, which would allow us to expand the number of patients eligible to receive this type of treatment.

TAC01-CD19 will be tested at four leading lymphoma clinical study centers in the U.S. and Canada. Based on its preclinical profile, TAC01-CD19 has the potential to represent a significant advancement in T-cell therapy.

About TAC01-CD19

Despite transformational efficacy with existing approved Chimeric Antigen Receptor T-Cells (CAR-T), a significant unmet need remains due to substantial CAR-T toxicities and limited tumor types where CAR-T is effective. Triumvira is developing a proprietary T-Cell Antigen Coupler (TAC) receptor which is structurally and biologically distinct from CAR-T. The first of our pipeline product candidates, TAC01-CD19 is a novel T-cell therapy product targeting CD19, a validated target in lymphomas and leukemias. The product comprises patient-derived T-cells that have been genetically engineered to express the CD19 T-cell Antigen Coupler (TAC). Preclinical data suggest that TAC01-CD19 has the potential for being highly efficacious with minimal side effects in hematological malignancies.

About CD19 and Diffuse Large B-Cell Lymphoma (DLBCL)

CD19 is a B-cell marker and is expressed on the surface of B-cell malignancies such as Diffuse Large B-Cell Lymphoma (DLBCL). DLBCL is a subtype of Non-Hodgkin Lymphomas (NHLs). DLBCL impacted approximately 26,000 patients in the U.S. in 2018. While the objective response rates of 50 70% observed for treatment of large B-cell lymphomas with currently marketed patient-derived T-cell products are good, there still exists a significant percentage of patients who either do not benefit from treatment or who are not able to tolerate the serious toxicities associated with these products.

About U.S. FDAs Fast Track Designation Program

The FDAs Fast Track program was established to facilitate the development and expedite the review of drugs with the potential to treat serious conditions and address an unmet medical need. Companies that receive Fast Track designation are provided the opportunity for more frequent interactions with FDA during clinical development and are eligible for accelerated approval and/or priority review, if relevant criteria are met. Additionally, companies that receive Fast Track designation are allowed to submit completed sections of their New Drug Application (NDA) or Biologics License Application (BLA) for the drug on a rolling basis, resulting in the potential for an expedited FDA review process.

About Triumvira Immunologics

Triumvira Immunologics, Inc. (Triumvira) is a clinical stage immunotherapy company with the vision of developing novel T-cell therapies that are safer and more efficacious than current cell therapy cancer treatments, including chimeric antigen receptor (CAR) and engineered T-cell receptor (TCR) therapies. Our proprietary T-cell Antigen Coupler (TAC) technology recruits the entire natural T-cell receptor and functions independent of the Major Histocompatibility Complex (MHC), allowing for the development of better therapies for a broader range of patients with solid or liquid malignancies and with diseases other than cancer. With operations spanning North America, our corporate offices are in Austin, Texas, with our research facilities in Hamilton, Ontario. For more information, visit http://www.triumvira.com or send email inquiries to partners@triumvira.com.

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Triumvira Immunologics Announces Fast Track Designation for Its First TAC T-Cell Therapeutic Product Candidate: TAC01-CD19 - Yahoo Finance

LONDON, Nov. 07, 2019 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc(Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, today announced its operational and financial results for the third quarter ended September 30, 2019.

We are excited about the opportunity to share data updates at ASH on AUTO1 in ALL in three oral presentations, as well as an oral presentation on AUTO3 in DLBCL. We are also looking forward to presenting data on our other hematological clinical programs at ASH, and non-clinical data on our lead solid tumor program AUTO6NG at SITC, stated Dr. Christian Itin, chairman and chief executive officer of Autolus. This quarter we have made significant operational progress, delivering cell products from our new manufacturing operations at the Cell and Gene Therapy Catapult and further strengthening our management team. Supported by a strong balance sheet, our key focus is on moving AUTO1 into our first pivotal clinical program in adult patients with ALL.

Pipeline Updates:

Solid tumors (AUTO6NG) - AUTO6NG: Next generation GD2-targeting CAR T-cell therapy with improved persistence and insensitivity to TGFb and checkpoint inhibition for relapsed/refractory neuroblastoma (Saturday November 9, poster presentation)

Adult ALL (AUTO1) - AUTO1, a novel fast off CD19 CAR delivers durable remissions and prolonged CAR T cell persistence with low CRS or neurotoxicity in adult ALL (Saturday December 7, oral presentation)

Pediatric ALL (AUTO1) - Therapy of pediatric B-ALL with a lower affinity CD19 CAR leads to enhanced expansion and prolonged CAR T cell persistence in patients with low bone marrow tumor burden, and is associated with a favorable toxicity profile (Saturday December 7, oral presentation)

Integration Site Analysis (AUTO1) - Clonal dynamics of early responder and long-term persisting CAR-T cells in humans (Saturday December 7, oral presentation)

DLBCL (AUTO3) - Phase 1/2 study of AUTO3 the first bicistronic chimeric antigen receptor (CAR) targeting CD19 and CD22 followed by an anti-PD1 in patients with relapsed/refractory (r/r) Diffuse Large B Cell Lymphoma (DLBCL): Results of Cohort 1 and 2 of the ALEXANDER study (Saturday December 7, oral presentation)

Multiple Myeloma (AUTO2) - Phase 1 First-in-Human study of AUTO2, the first chimeric antigen receptor (CAR) T cell targeting APRIL for patients with relapsed/refractory Multiple Myeloma (RRMM) (Sunday December 8, poster presentation)

Pediatric ALL (AUTO3) - Phase 1 Study of AUTO3, a Bicistronic Chimeric Antigen Receptor (CAR) T-cell Therapy Targeting of CD19 and CD22, in Pediatric Patients with Relapsed/Refractory B-cell Acute Lymphoblastic Leukemia (r/r B-ALL): AMELIA Study (Sunday December 8, poster presentation)

Operational and Corporate Highlights:

The Cell and Gene Therapy Catapult site is fully operational and is delivering clinical products for patients in both Europe and the US

In September, PPF Group announced that they had acquired, mainly from Woodford Investment Management, an approximate 19% holding of Autolus. Control of all the remaining shares of Autolus held by Woodford Investment Management are in the process of being transferred to Schroder UK Public Private Trust plc.

In September, Autolus announced that the journal Nature Medicine has published both pre-clinical results and clinical data from the ongoing Phase 1 CARPALL trial of AUTO1, demonstrating the potential of the companys novel CAR T therapy targeting CD19 in development for the treatment of pediatric acute lymphoblastic leukemia (ALL).

David Brochu has been named Senior Vice President, Head of Product Delivery to lead the transition of the companys manufacturing organization to deliver products for late-stage clinical studies and commercial sale. In addition, Vishal Mehta has been named Vice President, Head of Clinical Operations.

Key Upcoming Clinical Milestones:

Financial results for third quarter 2019

Cash and equivalents at September 30, 2019 totaled $229.4 million, compared with $247.1 million at September 30, 2018.

Net total operating expenses for the three months ended September 30, 2019 were $35.6 million, net of grant income of $0.3 million, as compared to net operating expenses of $17.1 million, net of grant income of $0.3 million, for the same period in 2018. The increase was due, in general, to the increase in development activity, increased headcount primarily in our development and manufacturing functions, and the cost of being a public company.

Research and development expenses increased to $27.3 million for the three months ended September 30, 2019 from $10.1 million for the three months ended September 30, 2018. Cash costs, which exclude depreciation as well as share-based compensation, increased to $21.6 million from $9.0 million. The increase in research and development cash costs of $12.6million consisted primarily of an increase of compensation-related costs of $5.2million primarily due to an increase in employee headcount to support the advancement of our product candidates in clinical development, an increase of $3.6 million in research and development program expenses related to the activities necessary to prepare, activate, and monitor clinical trial programs, including the manufacturing technical transfer activities required for AUTO1 to enable the commencement at the end of 2019 of a registration study in Adult Acute Lymphoblastic Leukemia, an increase of $2.6 million in facilities costs supporting the expansion of our research and translational science capability and investment in manufacturing facilities and equipment, an increase of $0.7 million in telecom and software costs, and an increase of $0.5 million in other costs.

General and administrative expenses increased to $8.6 million for the three months ended September30, 2019 from $7.3 million for the three months ended September30, 2018. Cash costs, which exclude depreciation expense as well as share-based expense compensation decreased to $5.6 million from $5.7 million. Compensation related expenses decreased by $0.6 million and IT, telecommunication, and general office expense costs decreased by $0.7 million which were offset by an increase in legal and professional fees of $0.9 million and an increase of $0.3 million in commercial costs.

Net loss attributable to ordinary shareholders was $27.2 million for the three months ended September 30, 2019, compared to $12.9 million for the same period in 2018.

The basic and diluted net loss per ordinary share for the three months ended September 30, 2019 totaled $(0.61) compared to a basic and diluted net loss per ordinary share of $(0.33) for the three months ended September 30, 2018.

Autolus anticipates that cash on hand provides a runway into the second half of 2021.

Conference Call and Presentation Information

Autolus management will host a conference call today, November 7, at 8:30 a.m. EST/ 1:30pm GMT, to discuss the companys financial results and operational update.

To listen to the webcast and view the accompanying slide presentation, please go to: https://www.autolus.com/investor-relations/news-events/events.

The call may also be accessed by dialing (866) 679-5407 for U.S. and Canada callers or (409) 217-8320 for international callers. Please reference conference ID 5075598. After the conference call, a replay will be available for one week. To access the replay, please dial (855) 859-2056 for U.S. and Canada callers or (404) 537-3406 for international callers. Please reference conference ID 5075598.

About Autolus Therapeutics plc

Autolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer. Using a broad suite of proprietary and modular T cell programming technologies, the company is engineering precisely targeted, controlled and highly active T cell therapies that are designed to better recognize cancer cells, break down their defense mechanisms and eliminate these cells. Autolus has a pipeline of product candidates in development for the treatment of hematological malignancies and solid tumors. For more information please visit http://www.autolus.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding Autolus financial condition and results of operations, as well as statements regarding the anticipated development of Autolus product candidates, including its intentions regarding the timing for providing further updates on the development of its product candidates, and the sufficiency of its cash resources. Any forward-looking statements are based on management's current views and assumptions and involve risks and uncertainties that could cause actual results, performance or events to differ materially from those expressed or implied in such statements. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section titled "Risk Factors" in Autolus' Annual Report on Form 20-F filed on November 23, 2018 as well as discussions of potential risks, uncertainties, and other important factors in Autolus' future filings with the Securities and Exchange Commission from time to time. All information in this press release is as of the date of the release, and the company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required by law.

Investor and media contact: Silvia TaylorVice President, Corporate Affairs and Communications Autolus+1-240-801-3850s.taylor@autolus.com

UK:Julia Wilson+44 (0) 7818 430877j.wilson@autolus.com

Autolus Therapeutics PLCCondensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited)(In thousands, except share and per share amounts)

Autolus Therapeutics PLCCondensed Consolidated Balance Sheets(In thousands, except share and per share amounts)

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Autolus Therapeutics Reports Third Quarter 2019 Financial Results and Operational Progress - GlobeNewswire

WATERTOWN, Mass. and RESEARCH TRIANGLE PARK, N.C., Nov. 07, 2019 (GLOBE NEWSWIRE) -- SQZ Biotechnologies (SQZ), and Asklepios BioPharmaceutical, Inc. (AskBio), announced a research collaboration to create tolerizing antigen carriers (TACs) containing AAV (adeno-associated virus) components to solve one of gene therapys biggest challenges the barrier to treatment posed by patients immune systems generating neutralizing antibodies toward therapeutic AAVs. SQZ and AskBio will combine their proprietary cell and gene therapy platform technologies to open the door to new treatment paradigms with potential impact across many genetic diseases.

Gene therapies utilizing AAV vectors can be transformative for patients with genetic diseases, but neutralizing antibodies can prevent large populations of patients from benefitting from AAV gene therapies. Patients immune systems develop neutralizing antibodies after receiving their first dose of AAV, or they can be pre-existing. This collaboration will strive to give these patients access to novel therapeutics and enable them to take multiple or repetitive doses to gain the full, durable benefit these treatments can provide. Expanding patient eligibility and allowing repeat treatment could change the future of how products are developed and significantly impact the long-term health of millions in need.

This is a tremendous opportunity to bring together the power of both cell and gene therapy for patients. AskBio has been an innovative leader in gene therapy and shares our patient-centric philosophy. By working together and leveraging the potential of both our platforms, we hope to bring more effective, more durable treatments to patients suffering from devastating rare genetic disorders, said Armon Sharei, PhD, founder and chief executive officer of SQZ Biotech.

The collaboration between SQZ and AskBio will evaluate the administration of SQZ TACs and AskBios gene therapies to potentially address AAV immunogenicity. SQZ is a pioneer in cell therapy, and the companys knowledge and expertise, as well as their advance capabilities in manufacturing, are critical to this collaborations approach to synergizing cell and gene therapies. Preclinical data from SQZ has demonstrated that SQZ TACs specifically inhibit undesired immune responses in multiple contexts, including AAV models. As a leader in the AAV field, AskBio brings expertise in AAV technology, capsid design, clinical processes and manufacturing that would allow for application of these novel methods to overcome immunogenicity. The two companies have a shared goal to increase world-wide access of transformative therapeutics.

R. Jude Samulski, PhD, chief scientific officer and co-founder of AskBio, noted, AskBio is firmly committed to improving the lives of underserved patients, such as those suffering from Pompe, Huntingtons and various neuromuscular and central nervous system diseases. Addressing AAV immunogenicity is essential to the future of gene therapy as it is one of the most significant limiting factors plaguing the gene therapy space today. SQZs pioneering approach to tolerance could offer a solution to this problem. Our collaboration with SQZ is exemplary of our goal to broadly explore potential redosing of AAV gene therapies, added Sheila Mikhail, chief executive officer and co-founder of AskBio. We are thrilled to be working with SQZ and are hopeful that this initial research collaboration utilizing two of the most promising therapeutic modalities currently available, cell and gene therapy, will ultimately provide options to improve patients immune response to gene therapy.

About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy platform company dedicated to improving the lives of children and adults with genetic disorders. AskBios gene therapy platform includes an industry-leading proprietary cell line manufacturing process known as Pro10 and an extensive AAV capsid library. Based in Research Triangle Park, N.C., the company has generated hundreds of proprietary third-generation gene vectors, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production, chimeric vectors, and self-complementary DNA. AskBio maintains a portfolio of clinical programs across a range neurodegenerative and neuromuscular indications with a current pipeline that includes therapeutics for Pompe disease, Limb Girdle Muscular Dystrophy and congestive heart failure as well as out-license clinical indications for Hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne Muscular Dystrophy (Bamboo Therapeutics acquired by Pfizer). For more information, visit http://www.askbio.com.

About SQZ BiotechSQZ Biotech is a privately held, clinical-stage company creating innovative treatments by transforming cells into sophisticated therapeutics. Using its proprietary platform, SQZ has the unique ability to precision engineer virtually any cell type and deliver multiple materials, potentially resulting in powerful, multifunctional cell therapies for a range of diseases with an initial focus on cancer and autoimmune disease. The companys initial applications leverage SQZs ability to generate target-specific immune responses, both in activation for the treatment of solid tumors, and immune suppression for the treatment of immune reactions and diseases. For more information please visit http://www.sqzbiotech.com.

About SQZ TACsSQZ tolerizing antigen carriers (TACs) are being developed to induce tolerance to aberrant or unwanted immune activity. TACs are developed from red blood cells (RBCs) SQZd with target-specific antigens and piggyback on the natural process of RBC destruction in the body, also known as eryptosis. A process moderated by our liver and spleen, eryptosis causes macrophages to take up aged or senescent RBCs. When our bodies process RBCs for destruction, their components are presented in a tolerogenic manner, reminding our immune systems not to attack our own red blood cells. SQZ TACs drive targeted antigensthrough this powerful natural mechanism, specifically tolerizing the immune system, potentially stopping undesired immune responses.

AskBio Contacts: Mark Rosenbergmark@trueparallel.com919-412-7378

Roger Friedensen, APRroger@trueparallel.com919-349-3206

SQZ Contacts:Rebecca CohenSenior Manager, Corporate Relationsrebecca.cohen@sqzbiotech.com617-758-8672 ext. 728

Cait Williamson, PhDLifeSci Public Relations cait@lifescipublicrelations.com646-751-4366

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SQZ Biotech and AskBio Announce Research Collaboration to Create Immune Tolerization Products for AAV Gene Therapies - GlobeNewswire

Gosselies, Belgium, 6November 2019, 7am CET BONE THERAPEUTICS(Euronext Brussels and Paris: BOTHE), the leading biotech company focused on the development of innovative cell and biological therapies to address high unmet medical needs in orthopaedics and bone diseases, today reports its financial results for the first nine months of 2019 and provides a business update for the third quarter ended 30 September 2019.

Thomas Lienard, Chief Executive Officer of Bone Therapeutics commented: We are making strong progress across the entire business and we are now approaching key value inflection points. As we near the completion of the necessary regulatory and operational measures, including the successful production of clinical batches, we are fully prepared to start late-stage clinical development with our allogenic cell therapy product, ALLOB, in patients with difficult-to-heal fractures, and our enriched protein solution, JTA-004, in patients with knee osteoarthritis. With most preparations in place, we look forward to further executing on our strategy and to communicating the next milestones in the following months.

Operational highlights Q3 2019 to date

Financial highlights first nine months 2019(1)

Outlook for the remainder of 2019

(1)Unaudited numbers

About Bone Therapeutics

Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopaedics and bone diseases. Based in Gosselies, Belgium, the Company has a broad, diversified portfolio of bone cell therapy and an innovative biological product in later-stage clinical development across a number of disease areas, which target markets with large unmet medical needs and limited innovation.

Bone Therapeutics core technology is based on its allogeneic cell therapy platform (ALLOB) which uses a unique, proprietary approach to bone regeneration, which turns undifferentiated stem cells from healthy donors into bone-forming cells. These cells can be administered via a minimally invasive procedure, avoiding the need for invasive surgery, and are produced via a proprietary, cutting-edge manufacturing process.

The Companys ALLOB product pipeline includes a cell therapy product candidate that is expected to enter Phase IIb clinical development for the treatment of difficult-to-heal fractures and a Phase II asset in patients undergoing a spinal fusion procedure. In addition, the Company is also developing an off-the-shelf protein solution, JTA-004, which is expected to enter Phase III development for the treatment of pain in knee osteoarthritis.

Bone Therapeutics cell therapy products are manufactured to the highest GMP (Good Manufacturing Practices) standards and are protected by a broad IP (Intellectual Property) portfolio covering ten patent families as well as knowhow. Further information is available atwww.bonetherapeutics.com.

Contacts

Bone Therapeutics SAThomas Lienard, Chief Executive OfficerJean-Luc Vandebroek, Chief Financial OfficerTel: +32 (0) 71 12 10 00investorrelations@bonetherapeutics.com

International Media Enquiries:Consilium Strategic CommunicationsMarieke VermeerschTel: +44 (0) 20 3709 5701bonetherapeutics@consilium-comms.com

For French Media and Investor Enquiries:NewCap Investor Relations & Financial CommunicationsPierre Laurent, Louis-Victor Delouvrier and Arthur RouillTel: + 33 (0)1 44 71 94 94bone@newcap.eu

Certain statements, beliefs and opinions in this press release are forward-looking, which reflect the Company or, as appropriate, the Company directors` current expectations and projections about future events. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. These risks, uncertainties and assumptions could adversely affect the outcome and financial effects of the plans and events described herein. A multitude of factors including, but not limited to, changes in demand, competition and technology, can cause actual events, performance or results to differ significantly from any anticipated development. Forward looking statements contained in this press release regarding past trends or activities should not be taken as a representation that such trends or activities will continue in the future. As a result, the Company expressly disclaims any obligation or undertaking to release any update or revisions to any forward-looking statements in this press release as a result of any change in expectations or any change in events, conditions, assumptions or circumstances on which these forward-looking statements are based. Neither the Company nor its advisers or representatives nor any of its subsidiary undertakings or any such person`s officers or employees guarantees that the assumptions underlying such forward-looking statements are free from errors nor does either accept any responsibility for the future accuracy of the forward-looking statements contained in this press release or the actual occurrence of the forecasted developments. You should not place undue reliance on forward-looking statements, which speak only as of the date of this press release.

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Bone Therapeutics Reports Financial Results for the First Nine Months of 2019 and Provides Third Quarter 2019 Business Update - OrthoSpineNews

Company to Present Four Oral Presentations and Two Posters

LONDON, Nov. 06, 2019 (GLOBE NEWSWIRE) -- Autolus Therapeutics plc(AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer, announced four oral and two poster presentations related to its AUTO1, AUTO2 and AUTO3 programs at the 61st American Society of Hematology (ASH) Annual Meeting and Exposition, to be held December 7-10, 2019 in Orlando, FL.

We are pleased that AUTO1 data will be presented in three oral presentations at ASH. The data form the basis for our decision to move AUTO1 into a pivotal clinical trial in adult ALL, our highest priority program, said Dr. Christian Itin, chairman and chief executive officer of Autolus. We are also looking forward to presenting data on our other hematological clinical programs at ASH. These presentations will further illustrate the significant progress we have made across our clinical portfolio this year.

The abstracts have been published today and are available on the ASH website at https://www.hematology.org/Annual-Meeting/Abstracts/.

The oral presentation details are as follows:

Title: AUTO1 A novel fast off CD19CAR delivers durable remissions and prolonged CAR T cell persistence with low CRS or neurotoxicity in adult ALLPresenter: Dr Claire RoddieSession Date and Time: Saturday, December 7, 2:45 PM Eastern Time

Title: AUTO1 Therapy of pediatric B-ALL with a lower affinity CD19 CAR leads to enhanced expansion and prolonged CAR T cell persistence in patients with low bone marrow tumor burden, and is associated with a favorable toxicity profilePresenter: Dr Sara GhorashianSession Date and Time: Saturday, December 7, 2:30 PM Eastern Time

Title: AUTO1 Clonal dynamics of early responder and long-term persisting CAR-T cells in humansPresenter: Dr Luca BiascoSession Date and Time: Saturday, December 7, 8:15 AM Eastern Time

Title: AUTO3 Ongoing Phase 1/2 ALEXANDER clinical trial in patients with relapsed/refractory diffuse large B cell lymphoma (DLBCL)Presenter: Dr Kirit ArdeshnaSession Date and Time: Saturday, December 7, 3:15 PM Eastern Time

The poster presentation details are as follows:

Title: AUTO2 Phase 1 First-in-Human study of AUTO2, the first chimeric antigen receptor (CAR) T cell targeting APRIL for patients with relapsed/refractory Multiple Myeloma (RRMM)Presenter: Dr Rakesh PopatSession Date and Time: Sunday, December 8, 6:00 PM 8:00 PM Eastern Time

Title: AUTO3 Phase 1/2 AMELIA clinical trial of AUTO3 in patients with relapsed/refractory pediatric acute lymphoblastic leukemia (pALL)Presenter: Professor Persis AmroliaSession Date and Time: Sunday, December 8, 6:00 PM 8:00 PM Eastern Time

About AUTO1

AUTO1 is a CD19 CAR T cell investigational therapy designed to overcome the limitations in safety - while maintaining similar levels of efficacy - compared to current CD19 CAR T cell therapies.Designed to have a fast target binding off-rate to minimize excessive activation of the programmed T cells, AUTO1 may reduce toxicity and be less prone to T cell exhaustion, which could enhance persistence and improve the T cells' abilities to engage in serial killing of target cancer cells. In 2018, Autolus signed a license agreement under which Autolus acquired global rights fromUCL Business plc(UCLB), the technology-transfer company of UCL, to develop and commercialize AUTO1 for the treatment of B cell malignancies. AUTO1 is currently being evaluated in two Phase 1 studies, one in pediatric ALL and one in adult ALL.

About AUTO3

AUTO3 is a programmed T cell therapy containing two independent chimeric antigen receptors targeting CD19 and CD22 that have each been independently optimized for single target activity. By simultaneously targeting two B cell antigens, AUTO3 is designed to minimize relapse due to single antigen loss in patients with B cell malignancies. AUTO3 is currently being tested in pediatric ALL in the AMELIA clinical trial and in diffuse large B cell lymphoma in the ALEXANDER clinical trial.

About AUTO2

AUTO2 is the first dual-targeting programmed T cell product candidate binding to two targets on multiple myeloma cells. AUTO2 uses a human ligand, known as APRIL, which binds to two antigens, B cell Maturation Antigen, or BCMA, and the transmembrane activator and CAML interactor, or TACI, both of which are expressed on the surface of multiple myeloma cancer cells. AUTO2 is designed to address a key escape route used by hematological cancers in response to T cell therapies.

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AboutAutolus Therapeutics plc

Autolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer. Using a broad suite of proprietary and modular T cell programming technologies, the company is engineering precisely targeted, controlled and highly active T cell therapies that are designed to better recognize cancer cells, break down their defense mechanisms and eliminate these cells. Autolus has a pipeline of product candidates in development for the treatment of hematological malignancies and solid tumors. For more information please visit http://www.autolus.com.

Forward-Looking Statement

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding Autolus financial condition and results of operations, as well as statements regarding the anticipated development of Autolus product candidates, including its intentions regarding the timing for providing further updates on the development of its product candidates, and the sufficiency of its cash resources. Any forward-looking statements are based on management's current views and assumptions and involve risks and uncertainties that could cause actual results, performance or events to differ materially from those expressed or implied in such statements. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section titled "Risk Factors" in Autolus' Annual Report on Form 20-F filed on November 23, 2018 as well as discussions of potential risks, uncertainties, and other important factors in Autolus' future filings with the Securities and Exchange Commission from time to time. All information in this press release is as of the date of the release, and the company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required by law.

Investor and media contact: Silvia TaylorVice President, Corporate Affairs and Communications Autolus+1-240-801-3850s.taylor@autolus.com

UK:Julia Wilson+44 (0) 7818 430877j.wilson@autolus.com

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Autolus Therapeutics to Present New Data on Its Advanced Programmed T Cell Therapies at the 61st ASH Annual Meeting - Yahoo Finance

ArsenalBio has exited stealth with $85 million to discover and develop cell therapies. The biotech aims to differentiate itself from the ever-growing pack of cell therapy startups with technology that enables the insertion of large DNA sequences without the use of viral vectors.

The design and production of first-generation T-cell therapies entails using a viral vector to insert one cell-targeting transgene. ArsenalBio wants to use CRISPR-based genome engineering to rewrite far larger sections of DNA, potentially leading to better treatments that are faster and simpler to design and manufacture.

Some big names have bought into ArsenalBios idea. Beth Seidenbergs Westlake Village BioPartners, the Parker Institute for Cancer Immunotherapy (PICI) and Kleiner Perkins participated in the round.

Industry Insight Survey: Direct-to-Patient Distribution of Clinical Supplies

This industry survey seeks to gain insight on trial sponsors' perspective on offering a DTP option and their current level of awareness and understanding of any factors that may influence their ability to do so. The first 50 qualified respondents will receive a $5 Amazon gift card.

Responsibility for overseeing the use of the $85 million will fall on Ken Drazan, the former president of Grail. Drazen, CEO of ArsenalBio, is joined on the management team by Jane Grogan, Michael Kalos and Tarjei Mikkelsen. Grogan, Kalos and Mikkelsen used to work at Genentech, Johnson & Johnson and 10x Genomics, respectively.

The management team will build on the work of ArsenalBios scientific founders, who were brought into each others orbits through Sean Parkers PICI. Broad Institutes Bradley Bernstein, Kole Roybal from the University of California, San Fransisco (UCSF), the University of Pennsylvania's John Wherry and Nicholas Haining, formerly of Dana-Farber Cancer Institute, are among the scientific founders.

Alexander Marson and Theodore Roth, both of UCSF, are the other two scientific founders. Marson and Roth were part of a large group that authored a Nature paper last year on the reprogramming of human T-cell function and specificity with nonviral genome targeting.

The paper describes the use of an approach in line with that sketched out by ArsenalBio. Marson, Roth and their collaborators used a CRISPR-Cas9 targeting system to insert a 1.5-kb DNA cassette encoding for a TCR beta chain into a specific part of the T-cell genome. The researchers, who also used the approach to correct a pathogenic autoimmune mutation, see multiple benefits.

In approximately one week, novel gRNAs and DNA repair templates can be designed, synthesized, and the DNA integrated into primary human T cells that remain viable, expandable, and functional. The whole process and all required materials can be easily adapted to good manufacturing practices for clinical use. Avoiding the use of viral vectors will accelerate research and clinical applications, reduce the cost of genome targeting, and potentially improve safety, the researchers wrote.

ArsenalBio will initially focus on applying its CRISPR-based genome engineering technology to cancer indications, but its longer-term vision is to develop immune cell therapies more broadly.

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ArsenalBio joins next-gen cell therapy field with $85M A round - FierceBiotech

ROCKVILLE, Md., Oct. 17, 2019 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, today announced the presentation of two posters at the European Society of Gene & Cell Therapy (ESGCT) 27th Annual Congress in Barcelona, Spain, taking place from October 22 to 25, 2019.

The data will be presented as follows:

Abstract Title: Characterization of a Novel AAV Capsid with Enhanced Brain Transduction Following Systemic Delivery (poster #P011)Presenter: Subha Karumuthil-Melethil, Ph.D., Senior Scientist, Target Discovery, REGENXBIOSession Title: Poster Session IDate/Time: Wednesday, October 23, 2019, 1:00 p.m. to 3:00 p.m. CESTLocation:Multipurpose Hall P011

Abstract Title: AAV9.hCLN2 (RGX-181) Improves Survival and Neuropathology in TPP1m1J Mice, a Model for CLN2 Batten Disease (poster #P018)Presenter: Nicholas Buss, Ph.D., Director, Preclinical Development, REGENXBIOSession Title: Poster Session II Date/Time: Thursday, October 24, 2019, 1:15 p.m. to 2:45 p.m. CESTLocation:Multipurpose Hall P018

About REGENXBIO Inc.

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas. For more information, visit http://www.regenxbio.com.

Contacts:Tricia TruehartInvestor Relations and Corporate Communications347-926-7709ttruehart@regenxbio.com

Investors:Heather Savelle, 212-600-1902heather@argotpartners.com

Media:David Rosen, 212-600-1902david.rosen@argotpartners.com

SOURCE REGENXBIO Inc.

http://www.regenxbio.com

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REGENXBIO Announces Presentations at the European Society of Gene & Cell Therapy 27th Annual Congress - PRNewswire

You can rack up one more high-profile debut effort in the wave of activity forming around cell therapy 2.0. Its another appealing Bay Area group thats attracted some of the top hands in the business to a multi-year effort to create a breakthrough. And they have $85 million in hand to make that first big step to the clinic.

Today its Ken Drazan and the team at South San Francisco-based ArsenalBio that are coming from behind the curtain for a public bow, backed by billionaire Sean Parker and a collection of investors that includes Beth Seidenbergs new venture investment operation based in LA.Drazan a J&J Innovation vet with a long record of entrepreneurial endeavors exited the stage in 2018 when his last mission ended as he stepped aside as president of Grail. It wasnt long, though, before he was helping out with a business plan for ArsenalBio that revolved around the work of a large group of interconnected scientists supported by the Parker Institute for Cancer Immunology.The biotech started by putting together an arsenal of technologies aimed at making cell therapies for cancer much, much better than the rather crude first-generation drugs that hit the market from Novartis and Kite.Their drugs have become the baseline against which all others are being measured.The technology set were developing is independent of the chassis, Drazan tells me. It doesnt have to be autologous (extracted from the patient) or allogeneic (off the shelf). It doesnt have to be a T cell, it could be a B cell. But they are starting out on the autologous side, where they have the most knowledge and insight into manufacturing techniques.It also doesnt have to be close to the clinic.Drazan expects the biotech will be working its way through preclinical operations for a few years, with enough money from the $85 million launch round to get into humans.By todays superheated fundraising standards, thats not a huge amount of cash. Lyell, another cell therapy 2.0 startup we featured last week, raised $600 million in a year, including a big chunk of cash from GlaxoSmithKline. Drazan is interested in dealmaking as well, but he also knows he has the cash necessary to support the company for a good run a key part of what it takes to bring together a stellar team of top players.

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Sean Parker helps create a CRISPRed cell therapy 2.0 play and he's got a high-profile set of leaders on the team - Endpoints News

Although administration of anti-CD19 chimeric antigen receptor (CAR)-T cell therapy takes place at authorized treatment centers, community oncologists still play an important role, particularly in the recognition of eligible patients and the management of adverse effects of the treatment.

Arecent piece in The Oncologist detailed this crucial element of CAR-Tadministration and highlighted key aspects of CAR-T cell indications andeligibility for community oncology providers.1

Tomaximize the chances of a patient receiving CAR-T cell therapy, communityoncologists should refer patients early and broadly, as the time of referralto CAR-T cell infusion can take 4 to 6 weeks.

Mostbroadly, patients with relapsed or refractory large B-cell lymphoma who havefailed on 2 or more prior therapies can be referred. Patients who have failedor relapsed after first-line immunochemotherapy may also be eligible.

Patientswho progress on first-line therapy should be referred directly to academiccenters whenever possible for management because high rates of relapse areobserved with second-line treatments, the authors wrote. Academic centers areequipped to facilitate a smooth and rapid transition to the next line oftherapy, especially CAR-T cell therapy, if patients are already receivingtreatment there, which may be particularly important for patients with rapidlyprogressing disease.

Aspart of this process, community oncologists should be aware of which centers intheir state offer CAR-T cell therapy.

Communityoncologists also play an important role in postinfusion care. Patients treatedwith CAR-T cell therapy are advised to carry a wallet card with them at alltimes that defines symptoms that could indicate a serious adverse event forwhich to seek medical attention. Any patient in response that does notexperience a serious adverse event after a 4- to 8-week stay returns home.

Thesepatients can experience prolonged hypogammaglobulinemia and B-cell aplasia, andsome patients may require supportive care with IVIG. Prolonged cytopenias canalso occur. Because the treatment causes immunosuppression, patients are atongoing risk for serious infections after discharge as well.

Coordinationand communication between the local oncologist and CAR-T cell treatment oncologistare important during the months after patients return home from their minimum4-week stay near the treatment center, the authors wrote. After this period,the authorized treatment center, in coordination with the local oncologist, mayhave patient follow-ups every 2 weeks until month 3, then decreasing infrequency to 6 months and 12 months after CAR T-cell infusion, then yearlyuntil 5 years after CAR T-cell infusion, the authors wrote.

Reference

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Community Oncologist: A Key Player in CAR-T Cell Therapy - Cancer Therapy Advisor

More News17 Oct 2019 | 10:17 PM

Bhubaneshwar, Oct 17 (UNI) CII EXCON 2019 - South Asias largest construction equipmenttrade fair is scheduled to be held at, Bengaluru from December 10-14 next.

Kolkata, Oct 17 (UNI) Microsofts M12, Mayfield and Pivotal Ventures on Thursday announced he second global Female Founders Competition to accelerate funding for women entrepreneurs developing business-to-business software-as-a-service and deeptech solutions.

Shillong, Oct 17 (UNI) Meghalaya Chief Minister Conrad K Sangma on Thursday exudedconfidence that United Democratic Party (UDP) candidate Balajiedkupar Synrem, will winthe by-election to the Shella assembly constituency.

Patna, Oct 17 (UNI) RJD vice president Shivanand Tiwari today admitted that NDA was able to maintain its lead in arithmetic votes as the opposition parties had yet to give a direction to their politics.

Kolkata, Oct 17 (UNI) To mark the centenary of the formation of the Indian Communist Party (ICP) as an emigre unit in Tashkent by the Second World Congress of the Communist Third International in 1920, the CPI(M) on Thursday kickstarted its year-long celebration at the Netaji Indoor Stadium here.

Read more:
Stem cell therapy brings hope for the treatment of neurological disorders- Dr.Bansod - United News of India