On April 30, 2018 PharmaCyte Biotech, Inc. (OTCQB: PMCB), a clinical stage biotechnology company focused on developing targeted cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology, Cell-in-a-Box, reported the successful results of long-term studies on the type of cells that will be encapsulated using its Cell-in-a-Box technology and then combined with low doses of the cancer prodrug ifosfamide for the treatment of patients with locally advanced, non-metastatic, inoperable pancreatic cancer (LAPC) (Press release, PharmaCyte Biotech, APR 30, 2018, View Source [SID1234525857]).

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These studies, which have taken over three years to complete, are needed to develop the comprehensive dossier of information concerning the genetically engineered human cells that are encapsulated for use in PharmaCyte’s therapy for LAPC. This work is required by the U.S. Food and Drug Administration ("FDA") to be included in PharmaCyte’s Investigational New Drug Application (IND) PharmaCyte is in the process of preparing for submission to the FDA.

The long-term studies were designed to demonstrate that the cells did not alter their properties after long periods in cell culture. The successful nature of these studies ensured that the properties of the product will not change over time and that product manufactured now will be the same as product manufactured in the future.

Kenneth L Waggoner, Chief Executive Officer of PharmaCyte, said, "We are delighted with the results of these long-term studies. They are crucial to demonstrating the long-term functionality and other characteristics of our genetically engineered cells, the ‘Active Pharmaceutical Ingredient (API),’ we plan to encapsulate as part of our therapy for LAPC. We have shown the API continues to function as designed over lengthy periods of time. We now have a large dossier of data to reference in our IND that documents each of the important characteristics of our API."

The characteristics of the cells that were tested included their genetic composition, their metabolic activity and the amount of the active enzyme (cytochrome P450 2B1) produced that converts the inactive prodrug, ifosfamide, to its active cancer-killing form. Importantly, the studies also showed that the cells were stable in the absence of the use of "selection agents," meaning that the cytochrome P450 gene that had been introduced into the cells was stably integrated in the genome of the cells.

On April 30, 2018 Portola Pharmaceuticals, Inc. (NASDAQ:PTLA) reported that it will host a webcast and conference call to discuss the Company’s financial results for the quarter ended March 31, 2018, and provide a general business overview on Wednesday, May 9, 2018, at 4:30 p.m. ET (1:30 p.m. PT) (Press release, Portola Pharmaceuticals, APR 30, 2018, View Source;p=RssLanding&cat=news&id=2345725 [SID1234525859]).

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Conference Call Details
The live conference call on Wednesday, May 9, 2018, at 4:30 p.m. ET, can be accessed by phone by calling (844) 452-6828 from the United States and Canada or 1 (765) 507-2588 internationally and using the passcode 7068059. The webcast can be accessed live on the Investor Relations section of the Company’s website at View Source It will be archived for 30 days following the call.

On April 30, 2018 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, reported eight oral and poster presentations at the upcoming American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2018 Annual Meeting being held May 16 -19, 2018 in Chicago, IL (Press release, Rocket Pharmaceuticals, APR 30, 2018, View Source;p=RssLanding&cat=news&id=2345662 [SID1234526550]).

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"Rocket has a robust pipeline of five innovative gene therapy programs across lentiviral vector (LVV) and adeno-associated viral vector (AAV) platforms, and we are pleased that our programs to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD) are featured at ASGCT (Free ASGCT Whitepaper)," said Gaurav Shah, Chief Executive Officer and President of Rocket. "Our most advanced program to treat FA is in a Phase 1/2 trial for which updated data will be presented during the Presidential Symposium. In children with FA, FANCA gene mutations enable chromosomal abnormalities that frequently lead to bone marrow failure, acute myeloid leukemia and death, with relatively toxic bone marrow transplant regimens as a principal therapy. We are hopeful that our FANCA-focused LVV gene therapy has the potential to enable broadly-applicable prevention of bone marrow failure, leading to safer and transformative outcomes."

Jonathan Schwartz, M.D., Chief Medical Officer of Rocket, added, "Our second most advanced program is our LVV gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I), which is expected to enter the clinic early next year. We are focused on the most severe form of LAD-I in which approximately 61-75% of children do not survive past the age of 2 due to life threatening infections. Based on our research, a modest correction of the defective gene encoding for the CD18 receptor can enable survival to adulthood. Both FA and LAD-I are examples of Rocket’s focus on exploring definitive therapies for patients with clearly defined monogenic diseases by targeting the underlying genetic deficit; these are disorders where a modest number of gene-corrected stem cells can make a meaningful difference for patients otherwise facing very limited treatment options. "

Oral Presentations:

Title: Engraftment and Phenotypic Correction of Hematopoietic Stem Cells in Non-Conditioned Fanconi Anemia Patients Treated with Ex Vivo Gene Therapy
Session: 330 Presidential Symposium & Presentation of the Top Abstracts
Presenter: Juan Bueren, Ph.D. Head of the Hematopoietic Innovative Therapies Division, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) / CIBER-Rare Diseases / IIS-Fundación Jiménez Díaz, and principal investigator of the RP-L102 trial.
Date: Friday, May 18, 2018
Session Time: 1:15 p.m. – 3:15 p.m. Central Time
Presentation Time: 2:45 p.m. – 3:00 p.m. Central Time
Location: International Ballroom North & South

Title: Immunotoxin-Based Conditioning Facilitates Autologous Hematopoietic Stem Cell Engraftment and Multi-Lineage Development in a Fanconi Anemia Mouse Model
Session: 115 Hematopoietic Cell Therapies
Presenter: Meera Srikanthan, M.D., Seattle Children’s Hospital; Fred Hutchinson Cancer Research Center
Date: Wednesday, May 16, 2018
Session Time: 10:30 a.m. – 12:00 p.m. Central Time
Presentation Time: 10:45 a.m. – 11:00 a.m. Central Time
Location: Salon A-5

Title: Gene Editing in Fanconi Anemia Hematopoietic Stem and Progenitor Cells
Session: 302 Advances in Genome Editing in HSCs – Organized by the Hematologic and Immunologic Gene and Cell Therapy Committee
Presenter: Paula Rio, Ph.D., Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT)
Date: Friday, May 18, 2018
Session Time: 8:00 a.m. -10:00 a.m. Central Time
Presentation Time: 9:00 a.m. – 9:30 a.m. Central Time
Location: International Ballroom North

Poster Presentations:

Title: Improvements in the Transduction Conditions of Human Hematopoietic Progenitors with the CPcoRPKW-17 Therapeutic Lentiviral Vector to be Used in a Pyruvate Kinase Deficiency Gene Therapy Clinical Trial
Session: Hematologic & Immunologic Diseases I
Date: Wednesday, May 16, 2018
Time: 5:30 p.m. – 7:30 p.m. Central Time
Location: Stevens Salon C, D

Title: Leukocyte Adhesion Deficiency I: A Closer Step to a Gene Therapy Clinical Trial
Session: Hematologic & Immunologic Diseases I
Date: Wednesday, May 16, 2018
Time: 5:30 p.m. – 7:30 p.m. Central Time
Location: Stevens Salon C, D

Title: Pairs of Guide RNAs Mediate Precise Deletions on the PKLR Gene via Non Homologous End Joining Generating a Human Hematopoietic Progenitor Model of Pyruvate Kinase Deficiency
Session: Hematologic & Immunologic Diseases II
Date: Thursday, May 17, 2018
Time: 5:15 p.m. – 7:15 p.m. Central Time
Location: Stevens Salon C, D

Title: Leukocyte Adhesion Deficiency-I: A Comprehensive Review of Published Cases
Session: Hematologic & Immunologic Diseases III
Date: Friday, May 18, 2018
Time: 5:45 p.m. – 7:45 p.m. Central Time
Location: Stevens Salon C, D

Title: Towards the Gene Therapy Clinical Trial for Pyrivate Kinase Deficiency
Session: Hematologic & Immunologic Diseases III
Date: Friday, May 18, 2018
Time: 5:45 p.m. – 7:45 p.m. Central Time
Location: Stevens Salon C, D

Zai Laboratory has filed a 20-F – Annual and transition report of foreign private issuers [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 20-F, Zai Laboratory, 2018, APR 30, 2018, View Source [SID1234527579]).

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On April 30, 2018 Triumvira Immunologics, Inc., a privately held biopharmaceutical company developing a novel platform for engineering T cells to attack multiple types of cancers, reported that it will present an abstract at the International Society for Cellular Therapy Annual Meeting (ISCT) in Montreal, and a company overview at the Bloom Burton & Co. Healthcare Investor Conference in Toronto, both on Thursday, May 3 (Press release, Triumvira Immunologics, APR 30, 2018, View Source [SID1234525860]).

At the ISCT, Dr. Chris Helsen, Director of Platform Development, will be presenting during the "Cancer Immunotherapy" breakout plenary on May 3, 11:00am – 12:15pm ET. The abstract is titled T cells engineered with T-Cell Antigen Coupler (TAC) receptors display robust efficacy against solid and liquid tumor xenografts in the absence of any toxicity.

At the Bloom Burton & Co. conference, President and Chief Executive Officer Dr. Paul Lammers will provide an overview of Triumvira and the improvements the company is making over current therapies with its novel immuno-oncology platform T Cell-Antigen Coupler (TAC). Dr. Lammers will also be giving updates on Triumvira’s lead drug, CD19-TAC01, which is targeted to enter clinical development in Q1 2019 for the treatment of patients with CD19 positive B-cell malignancies. Dr. Lammers will be presenting May 3, at 3:00pm ET and will be available for private meetings during the day.

About the ISCT
The International Society of Cellular Therapy is a global society of clinicians, regulators, technologists, and industry partners with a shared vision to translate cellular therapy into safe and effective therapies to improve patients’ lives. ISCT Members gain access to an influential global community of peers, experts, and organizations invested in cell therapy. ISCT offers a unique collaboration between academia, regulatory bodies, and industry partners in cell therapy translation.

About the Bloom Burton & Co. Conference
The Bloom Burton & Co. Healthcare Investor Conference brings together U.S., Canadian and international investors who are interested in the latest developments in the Canadian healthcare sector. Attendees will have an opportunity to obtain corporate updates from the premier Canadian publicly traded and private companies through presentation and private meetings.