Intellia Therapeutics Demonstrates Sustained and Durable Genome Editing with CRISPR/Cas9 in One-Year Animal Study

On September 26, 2017 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR/Cas9 technology, reported the full 12-month data of its completed long-term mouse study, demonstrating robust and durable in vivo genome editing post single-dose, intravenous administration using its proprietary lipid nanoparticle (LNP) delivery system (Press release, Intellia Therapeutics, SEP 26, 2017, View Source [SID1234520654]).

The data presented at the 13th Annual Meeting of the Oligonucleotide Therapeutics Society in Bordeaux, France, demonstrated robust editing and durability:

Achieved and maintained approximately 97 percent reduction in serum TTR protein levels through one-year, following a single dose
Attained approximately seventy percent editing at the target DNA site in the liver through one year
The transient nature of LNP delivery was confirmed with 99 percent clearance of mRNA within 10 hours and of sgRNA within 72 hours in the liver
The genome editing treatment was well tolerated with no adverse events during the course of the 12-month study
“These results show the long-term persistence of edited TTR genes in the mouse liver, indicating the potential for sustained efficacy and tolerability of our CRISPR/Cas9 platform as we move to higher species,” said David Morrissey, Ph.D., senior vice president, Platform and Delivery Technology. “These data validate Intellia’s approach as we remain on track with our non-human primate studies, which will permit us to designate our development candidate and perform IND-enabling activities in 2018 for the development of potential therapies in humans.”

Pieris Pharmaceuticals to Present at the LEERINK Partners Roundtable Series on Thursday, September 28 at 9:30 AM ET

On September 26, 2017 Pieris Pharmaceuticals, Inc. (NASDAQ: PIRS), a clinical-stage biotechnology company advancing novel biotherapeutics through its proprietary Anticalin technology platform for cancer, respiratory and other diseases, reported that President and CEO Stephen Yoder will present a corporate overview and meet with investors at the LEERINK Partners Roundtable Series on Rare Disease and Immuno-Oncology (Press release, Pieris Pharmaceuticals, SEP 26, 2017, View Source [SID1234520652]). The conference will take place September 27-28 at the Lotte New York Palace, New York, NY.

Mr. Yoder is scheduled to present on Thursday, September 28 at 9:30 AM ET. The presentation will be webcast live and can be accessed on the day of his presentation and for 90 days thereafter via this link.

CEO Jeffery Bacha Discusses DelMar’s VOL-083 Clinical Trials and the Major Milestones Ahead

On September 26, 2017 Jeffery Bacha, CEO of DelMar Pharmaceuticals Inc. (NASDAQ:DMPI) interviews on the Uptick Network Stock Day Podcast with host Everett Jolly. Mr. Bacha discusses DelMar’s VOL-083 phase 3 clinical trial chemotherapy that has recently launched and how this trial has progressed since its launch (Press release, DelMar Pharmaceuticals, SEP 26, 2017, View Source [SID1234520651]). Delmar recently completed a financing, and Mr. Bacha explains how this financing benefits the company and supports DelMar’s clinical trials. Furthermore, Mr. Bacha discusses the major milestones for the future of DelMar and what the company is looking to accomplish with its research and clinical trials.

In closing, CEO Jeffery Bacha states, “There is a wonderful story about a fellow who is a glioblastoma patient and he was treated the Monday, Tuesday and Wednesday prior to the weekend that he and his wife joined us on what’s called The Ride to Concur Cancer. He rode over 250 kilometers from Vancouver British Columbia to Seattle and he’s on our drug and he’s tolerating it very well,” Mr. Bacha continues, “It is amazing to be a part of that with him. Just knowing that this is someone that we are hopefully going to be able to help and being a part of that puts this into perspective of how important this is and we are looking to do that for many more patients.”

To listen to the full interview please click here or the following link: View Source

Autolus Secures US$80 million Series C Funding

On September 26, 2017 UCL Business spinout company, Autolus Limited, a clinical-stage biopharmaceutical company focused on the development and commercialisation of next-generation engineered T-cell therapies, reported that it closed on a US$80 million (£59 million) Series C financing (Press release, UCLB, SEP 26, 2017, View Source [SID1234520650]).

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New investors Cormorant Asset Management, Nextech Invest and others joined founding investor Syncona Ltd, Woodford Investment Management and Arix Bioscience in this Series C round.

The funds will enable Autolus to establish clinical proof of concept for three programs: AUTO2 in multiple myeloma, AUTO3 in diffuse large B cell lymphoma and paediatric acute lymphoblastic leukaemia and AUTO4 in T-cell lymphoma. In addition, building on its advanced cell programming technologies Autolus plans to advance its pre-clinical pipeline products for solid tumour indications, and will set up the infrastructure required for bringing a CAR-T cell therapy rapidly and successfully to market.

Dr Christian Itin, Chairman and CEO of Autolus, commented:
"We welcome our new shareholders and the continued support of our existing investors, and are looking forward to delivering on the exciting promise of our growing pipeline of engineered T cell product candidates. Since our inception three years ago, Autolus has made substantial progress with two dual targeting programs in three clinical studies, a novel program for T-cell lymphoma’s clinic ready and a unique suite of cell programming technologies established for use in haematological and solid cancers. With the new financing we are well on our way to building a premier fully integrated Oncology Company that harnesses the unique power of T cells to combat cancer."

Bihua Chen, CEO Cormorant Asset Management, commented:
"The upcoming commercial launches of the first generation of CAR-T products herald an important advance in the treatment of cancer. We are excited to be part of a company that is poised to be at the forefront of the next revolution in this field. We are impressed by Autolus’ advanced cell programming and manufacturing capabilities, and a team which we believe has the ability to deliver the full potential of these potentially life-changing therapies in both haematological and solid cancers."

Dr. Martin Murphy, CEO Syncona Ltd., commented:
"Autolus is delivering on world class science with a mission to build a leading oncology business. As a founding shareholder of Autolus we are excited about the remarkable potential of T-cell therapies for the treatment of patients with cancer. Unlocking that potential requires deep innovation in all areas, from cell programming to manufacturing and supply chain, in order to deliver these potentially transformative treatments to patients. Success in this space requires building a new type of biopharma business, which we are excited to be part of."

Joe Anderson, CEO of Arix Bioscience, commented:
"CAR-T is a proven new approach and represents a substantial advance in the treatment of cancer. Autolus has the science and the team to develop the next generation of CAR-T therapies, with a differentiated technology and approach that has the potential to transform patients’ lives. Arix is privileged to be part of this and I look forward to working with my colleagues on the Autolus Board and our co-investors to help achieve these goals."

The Series C follows the £70 million Series A and B financings.

OncoSec to Present at the Cantor Fitzgerald Global Healthcare Conference

On September 26, 2017 OncoSec Medical Incorporated (“OncoSec”) (NASDAQ:ONCS), a company developing DNA-based intratumoral cancer immunotherapies, reported that Punit Dhillon, CEO and President of OncoSec, will present at the Cantor Fitzgerald Global Healthcare Conference on Wednesday, September 27, 2017 at 4:35 PM ET / 1:35 PM PT (Press release, OncoSec Medical, SEP 26, 2017, View Source [SID1234520649]). The conference will take place at the Intercontinental New York Barclay Hotel.