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.

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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.

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

CAMBRIDGE, Mass., Oct. 16, 2019 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo, announced one oral presentation and four poster presentations were accepted for the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) taking place October 22-25, 2019, in Barcelona, Spain.

Intellias data includes important updates about the companys programs and platform development activities:

Oral Presentation:

In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression

Intellia will present data on its alpha-1 antitrypsin deficiency (AATD) program, which uses a modular hybrid delivery system combining lipid nanoparticle (LNP) encapsulated CRISPR/Cas9 with an adeno-associated virus (AAV) donor DNA template. Intellias gene knockout approach eliminates the production of the faulty PiZ variant of the protein, while targeted insertion of a wild-type gene copy facilitates production of a functional circulating protein. This builds on Intellias similar approach for targeted gene insertion of Factor 9, which achieved increased levels of circulating human Factor IX protein through two months in non-human primates and sustained through 12 months in mice.

Presenter: Anthony Forget, Ph.D.Abstract number: OR48Session 5b: New delivery systems and technologiesPresentation date/time: Friday, October 25, 2019, 11:30 a.m. 1:30 p.m. CETLocation: Room 113-115

Poster Presentations:

In Silico, Biochemical and Cell-Based Integrative Genomics Identifies Precise CRISPR/Cas9 Targets for Human Therapeutics

This poster presentation will highlight Intellias approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Researchers demonstrated that potential off-target editing profiles discovered through empirical data from biochemical approaches were the most sensitive and accurate.

Presenter: Daniel OConnell, Ph.D.Poster ID Number: P655Date: Wednesday, October 23, 2019

Generation of a Library of WT1-Specific T Cell Receptors (TCR) for TCR Gene Edited T Cell Therapy of Acute Leukemia

This poster presentation focuses on Intellias ongoing research collaboration with IRCCS Ospedale San Raffaele to develop CRISPR/Cas9-edited T cell therapies to address intractable cancers, such as acute myeloid leukemia (AML). Researchers have successfully established a protocol enabling consistent and efficient tumor-specific TCR isolation and characterization from healthy donors. Based on these results, Intellia has selected multiple lead TCRs, which are undergoing development candidate evaluation.

Presenter: Erica Carnevale, Ph.D., Ospedale San RaffaelePoster ID Number: P111Date: Wednesday, October 23, 2019

Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In-Locus Insertion Combined with Endogenous TCR Knockout

This poster presentation focuses on the companys T cell engineering technology, which is being applied in its Wilms Tumor 1 (WT1) lead ex vivo program. Intellia has identified an efficient CRISPR/Cas9-mediated process that inserts tumor-specific TCRs with high yield into the TRAC locus. Simultaneous knockout of the TRBC1 and TRBC2 loci substantially eliminates production of the endogenous T cell receptors.

Presenter: Birgit Schultes, Ph.D.Poster ID Number: P162Date: Thursday, October 24, 2019

CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria

This poster presentation will demonstrate the effects of independent CRISPR/Cas9-mediated knockout of each of two target genes involved in oxalate formation, lactate dehydrogenase A (LDHA) and hydroxyacid oxidase 1 (HAO1), to address primary hyperoxaluria type 1 (PH1).

Presenter: Sean Burns, M.D.Poster ID Number: P552Date: Thursday, October 24, 2019

About Intellia Therapeutics

Intellia Therapeuticsis a leading genome editing company focused on developing curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more aboutIntellia Therapeuticsand CRISPR/Cas9 atintelliatx.comand follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements ofIntellia Therapeutics, Inc.(Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its planned submission of an IND application for NTLA-2001 in mid-2020; its plans to generate preclinical and other data necessary to nominate a first engineered cell therapy development candidate for its AML program by the end of 2019; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program and otherin vivoandex vivoprograms; develop our proprietary LNP/AAV hybrid delivery system to advance our complex genome editing capabilities, such as gene insertion; its presentation of additional data at upcoming scientific conferences regarding CRISPR-mediated, targeted transgene insertion in the liver of NHPs, using F9 as a model gene, via the Companys proprietary LNP-AAV delivery technology, and other preclinical data by the end of 2019; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR and AML programs, in any future studies, including human clinical trials; its ability to develop otherin vivoorex vivocell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; the impact of its collaborations on its development programs, including but not limited to its collaboration withRegeneron Pharmaceuticals, Inc. or Ospedale San Raffaele; statements regarding the timing of regulatory filings regarding its development programs; and the ability to fund operations into the second half of 2021.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain our intellectual property position, including through our arbitration proceedings against Caribou; risks related to Intellias relationship with third parties, including our licensors; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations withNovartisor Regeneron or its otherex vivocollaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with theSecurities and Exchange Commission. All information in this press release is as of the date of the release, andIntellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Media:Jennifer Mound SmoterSenior Vice PresidentExternal Affairs & Communications+1 857-706-1071jenn.smoter@intelliatx.com

Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com

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Intellia Therapeutics Announces Presentations at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) - BioSpace

Results of an ex vivo study evaluating the phenotypic and growth characteristics of T cells collected by leukapheresis from cohorts of patients with newly diagnosed or relapsed/refractory multiple myeloma support use of chimeric antigen receptor (CAR)-T therapy earlier in course of the disease. The hypothesis-generating findings from this study were published in Blood Advances.

While CAR-T therapy targeted against the B-cellmaturation antigen (BCMA) has been associated with promising results inpatients with multiple myeloma, nearly all of the patients responding to thisapproach eventually develop progressive disease. Hence, strategies to optimizepatient selection for CAR-T therapy in the setting of multiple myeloma arebeing actively pursued.

Theratio of CD4 to CD8 T cells and/or the frequency of the CD81 CD45RO2 CD271 T-cell memory phenotype were usedin this study as surrogates for the clinical effectiveness of CAR-T therapysince previous studies ofCAR-T therapy in patients with chronic lymphocyticleukemia and multiple myeloma showed that of all baseline patient- anddisease-related characteristics considered, clinical response to CAR-T therapywas associated only with this T-cell ratio and/or the frequency of this subsetof memory T cells in the premanufacturing leukapheresis product.

Twocohorts of patients where compared in this study: 38 patients with newly diagnosedmultiple myeloma who had participated in clinical trials of induction therapy andon whom leukapheresis was performed before consolidation therapy and autologousstem cell transplantation (ASCT); and 25 patients with relapsed/refractorymultiple myeloma enrolled in a phase 1 clinical trial of anti-BCMA CAR-Ttherapy and on whom leukapheresis was performed during a washout period shortlyfollowing study enrollment.

Inboth patient cohorts, leukapheresis samples were exposed ex vivo to anti-CD3and anti-CD28 monoclonal antibodies covalently linked to magnetic beads toprovide stimulatory/costimulatory signals for T-cell proliferation and theexpansion of functional T cells.

The 2 patient cohorts were similar with respect to median age (ie, 55 years; 58 years [relapsed/refractory]), although the time from multiple myeloma diagnosis was 222 days for those treated with induction therapy and 4.6 years for those with relapsed/refractory disease.

Inaddition, differences in the median number of prior lines of therapy (1 vs 7),and bone marrow cellularity occupied by myeloma plasma cells (13% vs 65%) wereobserved when the former and latter cohorts were compared at the time thatleukapheresis was performed.

Akey finding from this study was a significantly higher frequency of T cellswith the CD81 CD45RO2CD271 T-cell memory phenotype(43.9% vs 29.0%; P =.001), as well asa significantly higher median CD4/CD8 ratio (2.6 vs 0.87; P <.0001) in the postinduction versus the relapsed/refractorypatient cohort.

Inaddition, the CD4/CD8 ratio was also significantly higher in the postinductioncohort compared with responders to anti-BCMA CAR-T therapy from the relapsed/refractorycohort (2.6 vs 1.3; P= .0009); however,while higher in the postinduction cohort, the difference in the frequency of Tcells with the CD81 CD45RO2CD271 T-cell memory phenotypewas not statistically significant when these 2 groups were compared.

Regardingcapacity for ex vivo proliferation during manufacturing, significantly highernumbers of population doubling by day 9 (PD9) were observed for thepostinduction cohort compared with either the overall relapsed/refractorycohort or the group of responders within the relapsed/refractory cohort.

Ourresults suggest that CAR T cells manufactured from leukapheresis samplesobtained after response to induction therapy would be, on average, moreclinically effective than those obtained from heavily relapsed/refractorymultiple myeloma patients, the study authors concluded.

Reference

Garfall AL, Dancy EK, Cohen AD, et al. T-cell phenotypes associated with effective CAR T-cell therapy in postinduction vs relapsed multiple myeloma. Blood Adv. 2019;3:2812-2815.

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CAR-T Therapy May Be More Effective When Administered Earlier in the Multiple Myeloma Treatment Continuum - Cancer Therapy Advisor

CAMBRIDGE, Mass. Sanofi is accelerating nascent efforts in gene therapy, aiming to use its expertise in vaccines to catch up in a competitive field that's well ahead of the French pharma.

The company has prioritized gene therapy programs amid a broader effort to boost internal R&D speed and impact, said John Reed, Sanofi's head of research and development, in a Wednesday interview at Sanofi's Cambridge office.

"When I joined, I saw that we were dabbling in gene therapy and decided that we need to get more serious about gene therapy if we are going to continue to be impactful in that space," said Reed, who came over to Sanofi from Roche last July.

In particular, the company is retrofitting one of its vaccine facilities near Lyon, France, to produce GMP-grade adeno-associated viral vectors, or AAVs. Reed said he expects the plant to be operational in about a year.

The new R&D chief is steering the company away from areas for which it's historically been known, including, most notably, cardiovascular disease and diabetes. Sanofi is largely exiting cardiovascular R&D and is cutting spending in half on diabetes R&D, Reed said.

While vaccines make up a comparatively smaller portion of Sanofi's revenues, Reed noted the company's decades-long expertise in producing inactivated viruses could translate well to gene therapy. Reed was recently in Lyon to discuss the budget and headcount requirements for the change, he said.

"We have an opportunity to really leverage those competencies around vaccines for the gene therapy area," Reed said. "We are looking at how we can use that as a competitive advantage to be players in that space."

Several of Sanofi's pharma peers have bet heavily on gene therapy, investing in manufacturing and snapping up biotech leaders through multi-billion dollar acquisitions, such as by Novartis for AveXis and Roche for Spark Therapeutics.

Smaller companies like BioMarin Pharmaceutical, meanwhile, hold sizable leads in therapeutic areas that Sanofi hopes to play a larger role in, like hemophilia.

Reed acknowledged an acquisition "could be an accelerator" in establishing Sanofi's presence in cell and gene therapy.

"We flirt with those things all the time," he said, when asked about his openness to a deal like those for AveXis and Spark. "It's a bit challenging to point your finger at any one gene therapy company and say that solves all our problems."

"It's been really tough to pull the trigger on something like that," he added. "In the interim, we've been establishing the capabilities more internally."

How much it would be willing to pay, or afford, is another question. Under former CEO Olivier Brandicourt, the company last year targeted roughly 20 billion euros in acquisitions, a budget largely consumed by deals for Bioverativ and Ablynx in the blood disease space.

The company's first AAV-delivered gene therapy recently entered the clinic for a form of a rare eye disease called Leber congenital amaurosis, Reed added.

Two gene-edited cell therapies are in Phase 1/2 testing via a collaboration with Sangamo Therapeutics. Other programs remain preclinical as the group works on establishing GMP manufacturing capabilities.

All of this is taking place against a backdrop of change for Sanofi research and development teams.

Reed is working to narrow the company's focus to advance only first- or potentially best-in-class therapies, a bar that led Sanofi to cut several dozen programs from its pipeline earlier this year.

Reed has also restructured employee's incentives, taking away bonuses for starting projects and replacing them with an emphasis on starting first-in-human studies, a milestone Sanofi usually reaches slower than industry leaders.

"I don't want to reward people for starting projects, I want to reward them for finishing projects," he said. "We have too many projects."

Part of that's involved reducing bureaucracy and streamlining decision-making, moving from 33 committees that interact with R&D teams to three. Reed's given decision-making authority to team leaders for each molecule, calling them CEOs of their drug candidate.

Even before Reed came on board, productivity had begun to improve from a nadir in 2014, when Sanofi's entire organization produced only two clinical candidates that year. Now, Sanofi is delivering about six per year and, with the 2018 acquisitions of Bioverativ and Ablynx, should reach eight or nine per year.

Still, of the last 10 drugs Sanofi has won approvals for, only one was an internal project, Reed said. For the company's next 10 assets, Reed expects six or seven to have been internally developed.

As Reed re-focuses, Sanofi has exited or restructured partnerships this year with Regeneron, Alnylam Pharmaceuticals and Lexicon Pharmaceuticals.

Paring down the pipeline and restructuring deals also speaks to Sanofi's R&D budget, which the company expects to keep flat for the next few years. The pharma spends about half what companies with larger revenues like Pfizer, Novartis and Roche do.

Reed says the ultimate goal is to bring about 12 programs into clinical development each year, and growing internal R&D to the point where it's responsible for the majority of those candidates progressing.

"With the resources we have, that would be industry competitive," he added.

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Sanofi investing in gene therapy as R&D focus turns toward rare disease - BioPharma Dive

CAMBRIDGE, Mass., Oct. 16, 2019 (GLOBE NEWSWIRE) -- LogicBio Therapeutics Inc. (Nasdaq:LOGC), a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients, today announced upcoming presentations at the European Society of Gene and Cell Therapy (ESGCT) 27th Annual Congress, held in Barcelona, Spain, October 22-25, 2019.

We are thrilled to be presenting positive data on our Next Generation Capsid Development Program on the anniversary of our collaboration with Childrens Medical Research Institute of Australia, a leader in gene therapy, childhood cancer, embryology and neurological diseases. The goal of the collaboration is to develop novel, synthetic adeno-associated virus (AAV) capsids which are highly tropic for human tissues and optimized for manufacturing. These data give us further confidence that we can improve the performance of current AAV vectors, expanding our pipeline and strengthening our GeneRide platform, said Fred Chereau, CEO of LogicBio. Further, we are pleased to present additional preclinical data further supporting the durability of expression, compared to canonical gene therapy, in one of our GeneRide platform programs and to have been invited to speak on AAV manufacturing.

Panel PresentationTitle: AAV manufacturing: critical parameters influencing vector quality attributesPresenter: Matthias Hebben, Ph.D., VP, Technology Development, LogicBio Therapeutics (INV36)Session: 1d ATMP manufacturingSession date/time: October 23, 2019, 8:30-10:30 a.m. CEST

Poster PresentationsTitle: AAV development program: towards next generation of livertropic AAV variants (P025)Session date/time: October 23rd, 2019, 1:00-3:00 p.m. CEST

Title: Durability of factor IX expression in mice treated neonatally with a nuclease-free, promoterless, AAV-based gene therapy, GeneRide (P423)Session date/time: October 23rd, 2019, 1:00-3:00 p.m. CEST

Additional information on the meeting can be found on the ESGCT website: https://www.esgct.eu/home.aspx

About LogicBio TherapeuticsLogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients with significant unmet medical needs using GeneRide, its proprietary technology platform. GeneRide enables the site-specific integration of a therapeutic transgene in a nuclease-free and promoterless approach by relying on the native process of homologous recombination to drive potential lifelong expression. Headquartered in Cambridge, Mass., LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families.

For more information, please visit http://www.logicbio.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the federal securities laws. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on April 1, 2019 with the SEC, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contacts

Brian LuqueAssociate Director, Investor Relationsbluque@logicbio.com951-206-1200

Stephanie SimonTen Bridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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LogicBio Therapeutics to Present New Data on Next Generation Capsid Development Program and GeneRide Platform Program at the European Society of Gene...

LOUISVILLE, Ky. Cancer patients in Louisville and throughout the region soon will have access to some of the most advanced immunotherapy treatments available.

Louisville resident Thomas E. Dunbar has pledged $1 million to the University of Louisville to create a specialized center to provide chimeric antigen receptor positive T (CAR T) cell therapies to patients at the U of L James Graham Brown Cancer Center and other centers in the Midwest.

The new program will be named the Dunbar CAR T-Cell Program.

This gift will allow both kids and adults to be treated right here in Kentucky with the most innovative cell-based immunotherapy being developed, said Jason Chesney, M.D., Ph.D., director of the U of L Brown Cancer Center.

In CAR T-cell therapies, immune cells are extracted from the patients own blood and then are genetically modified to fight cancer. The modified cells are infused back into the patient where they fight the cancer and create long-term immunity to its recurrence.

In addition to dramatic treatment results, CAR T-cell immunotherapy leads to fewer toxic side effects than traditional chemotherapy.

Patients who have been treated with all the conventional therapies who then underwent treatment in clinical trials with CAR T cells had dramatic response rates. Eighty-three percent of kids in the original trial who had lethal, terminal B-cell acute lymphoblastic leukemia responded to this therapy, Chesney said.

The Dunbar CAR T-Cell Program will include laboratories for manufacturing the CAR T cells and will administer both FDA-approved and clinical-trial therapies to adult and pediatric cancer patients.

The goal is for the facilities to be fully functional and receiving patients by Sept. 30, 2020.

Tom Dunbars son, Evan, lost his battle to cancer with neuroblastoma in 2001 at the age of 6. In 2009, Wally Dunbar, Tom Dunbars father, lost his battle with melanoma.

Donor Tom Dunbar with his son, Evan

U of L Brown Cancer Center

This year, Toms physician wife, Stephanie Altobellis, M.D., helped identify his own cancer.

Kentucky is at ground zero, with the nations highest rates of cancer diagnosis and death, Tom Dunbar said. Its completely unacceptable. We have to lead the charge right here where the need is the greatest and we can do the most good. We need treatments that are not toxic. Watching our loved ones miserable with pain, often just from the treatments, and yet still die in front of us simply cant be the best that we can do.

To learn more about how CAR T-cell treatment works visit: uoflbrowncancercenter.org

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Dunbar CAR T-Cell Program brings advanced immunotherapy to cancer patients - WHAS11.com