As California’s legislature heads into its break for the summer, returning on Aug. 21, the top three bills of concern to the life sciences industry all face the potential for amendments before reaching their respective final floor votes. | READ MORE »
Legislative Update From DC: FDA User Fees; ACA Repeal & Replace; Briefing On Cost & Value | READ MORE »
IPAB Will Not Be Triggered Until 2021, Medicare Trustees Report | READ MORE »
South San Francisco Life Sciences Cluster Sees Increased Development | READ MORE »
CLSA Member Boehringer Ingelheim expansion will bring nearly 300 new high-wage and highly-skilled positions to the City of Fremont. | READ MORE »
Biotech Primer article: Meds For Thinner Blood Can Equal Fewer Clots, But How?| READ MORE »
California Life Sciences Association (CLSA) is the state’s largest and most influential life sciences advocacy and business leadership organization. With offices in Sacramento, San Diego, South San Francisco, Los Angeles and Washington DC, CLSA works closely with industry, government, academia and others to shape public policy, improve access to innovative technologies and grow California’s life sciences economy. CLSA serves biotechnology, pharmaceutical, medical device and diagnostics companies, research universities and institutes, investors and service providers throughout the Golden State. CLSA was founded in 2015 when the Bay Area Bioscience Association (BayBio) and the California Healthcare Institute (CHI) merged. Visit CLSA at www.califesciences.org, and follow us on Twitter @CALifeSciences, Facebook, LinkedIn, and YouTube.
Dr. Robert Califf at CLSA Evening with Thought Leaders, San Diego Legislative Roundtable & Tour, July New Members, Giants of Science Deadline, New ScaleMatrix Member Benefits
By: Sara Radcliffe
CLSA President & CEO
CLSA – California Life Sciences Association
July 27, 2017
CLSA to Feature Dr. Robert Califf at Evening with Thought Leaders
Join us on Aug. 24at the Sofitel Los Angeles at Beverly Hills to hear Dr. Robert Califf – former FDA Commissioner and current leader with Duke Health, Duke University, and Verily Life Sciences – discuss trends and topics ranging from therapeutic and diagnostic innovations to health policy. Register here.
CLSA Hosts San Diego Legislative Tour & Discussion with Key Life Sciences Organizations
On July 14, our state advocacy team partnered with Organovo, Takeda and Sanford Burnham Prebys to educate members of the California Legislative Technology and Innovation Caucus on key areas of interest to our community.
We were thrilled to have participation from Assemblymember Kevin Mullin (Chair, Select Committee on Biotechnology), Assemblymember Todd Gloria, and staff from the offices of Assemblymembers Kevin Mullin, Evan Low, Rocky Chavez and Marie Waldron’s office. Organovo’s CEO, Taylor Crouch, began the tour by educating our attendees about cutting edge three-dimensional bioprinting and fabrication of living human tissue that can function like native tissues. Next CLSA took the group on a tour of Takeda’s San Diego facility and hosted a roundtable luncheon where CLSA staff and member companies briefed legislators about ongoing issues of concern to the life sciences sector. And finally, a tour of the Sanford Burnham Prebys Medical Discovery Institute (SBP) provided a comprehensive overview of biological research and the drug discovery process. The Institute’s CEO, Dr. Perry Nisen, educated the legislators about the work SBP is doing on cancer research, neuroscience, immunity, and metabolic disorders. Learn more here.
CLSA Announces Strategic Partnership with San Diego’s ScaleMatrix
In our ongoing efforts to advance life sciences innovation, we are excited to announce our new strategic partnership with ScaleMatrix, a unique hybrid-cloud services provider with a 14,000-square-foot life-sciences incubator and facility in San Diego.
ScaleMatrix’s incubator is tailor-made for early-stage companies in the fields of genomics, molecular diagnostics and bioinformatics. Their facility houses both a CLIA-certified genomics laboratory and a state-of-the-art datacenter. CLSA member companies can also leverage ScaleMatrix capabilities to address challenges with compliance, data center resources and scalable service delivery. Learn more here.
CLSI and BioLabs Host San Diego CARB-X Event
CLSI brought together a key segment of the San Diego community earlier this month at BioLabs San Diego for a CARB-X panel and networking event. Ninety-three attendees heard from Neil Abdollahian, Chief Business Officer of Cidara Therapeutics, and Zachary Zimmerman, CEO of Forge Therapeutics, two of the first CARB-X funded companies, in a panel moderated by Heather Shane, Executive Director of CLSI’s west coast CARB-X accelerator. To date, 18 companies have been awarded up to an aggregate of $41.6M in awards and up to an additional $52.6M in milestone-based options from the five-year $455M international public private CARB-X partnership devoted to developing products to combat antimicrobial resistance.
CLSA Welcomes 12 New Members in July
Please join us in welcoming the 12 new CLSA members who joined us in the last month:
Allele Biotechnology & Pharmaceuticals, BioAmp Diagnostics, DNA Electronics, EBD Group, Encoded Genomics, Encompass Bioscience, Encyclogen, Genea Biocells, Mantra Bio, MaxCyte Inc., Maxterial, and MITRx. Our membership portfolio grows ever more robust with the addition of these organizations, which add significantly to the expertise and resources available in the CLSA network.
Are You Coming to Our Open House & Anniversary Celebrations?
Did you miss the sold-out Open House Celebration in South San Francisco? It’s not too late to join CLSA to celebrate the second anniversary of our CHI and BayBio merger at our Open House & Anniversary Celebration in San Diego next week. Join us for fun, networking, and these highlights:
- Wine & Beer Tastings from Local Breweries & Wineries
- Food Trucks & Gourmet Catering
- Music/Local Bands
- Power Purchasing Partner Programs
- Photo Booths
If you haven’t been to a CLSA networking event yet, register now – we’re almost full!
Open House & Anniversary Celebration
– San Diego, August 3
JLABS, 3210 Merryfield Road, San Diego, CA
Final Week to Participate in CLSA Giants of Science Grant Contest
Do you know of a high school in the Bay Area that could benefit from a $20,000 science program grant? Thanks to our generous sponsors (Alexandria Real Estate, Celgene, Genentech, Gilead, Illumina, Nektar, Sutro Biopharma, Theravance Biopharma and Wareham Development) CLSA and CLSI are giving away three (3) $20,000 science grants during the 2017 Giants baseball season. The deadline to apply is July 31 – for more details or to submit an application, visit http://www.knbr.com/giantsofscience.
Don’t Miss Out – Register for the 2017 MedTech Conference
September 25-27 – San Jose, CA
Join CLSA and more than 2,500 attendees at The MedTech Conference powered by AdvaMed to network, gain access to capital and share insights in San Jose, Calif. from September 25-27. The conference features world-class plenary speakers, networking, and business development. Take advantage of our exclusive CLSA discount to claim your spot for The MedTech Conference today. Register Now at http://hubs.ly/H06K-H30.
President & CEO
California Life Sciences Association
PS – Have newsworthy items to submit for consideration for our monthly CLSA Bulletin? Feel free to send those tips to Elizabeth Gibson (EGibson@califesciences.org), Senior Director of Marketing.
California Legislature Heads to Summer Recess with Major Bills in Flux
July 24, 2017
As California’s legislature heads into its break for the summer, returning on Aug. 21, the top three bills of concern to the life sciences industry all face the potential for amendments before reaching their respective final floor votes. Those priority bills are Senate Bill 17 (Hernandez), SB 790 (McGuire) and Assembly Bill 265 (Wood).
SB 17 would require, among many other things, industry to provide a 60-day advance notice of any price increase on a drug to all California public agency purchasers, health insurers, and pharmacy benefit managers (PBM) if that drug’s price increased 10 percent or more cumulatively over the previous two calendar years. On the date of the increase, the drug’s manufacturer would have to submit a host of information to the state, including proprietary information like expected marketing budgets for the drug. This information would then be posted publicly by the state in a manner that allows identification of the individual drugs.
SB 17 is currently before the Assembly Appropriations Committee. CLSA and our partner trade associations are pushing for amendments to make the bill less administratively burdensome and costly for the industry, as well as requesting the same confidentiality protections granted to insurers under the bill.
Click here to add your voice and urge legislators to reject SB 17.
SB 790 would restrict all “gifts,” as defined, from manufacturers to physicians unless it falls under one of the many, often confusing, exceptions under the bill. For instance, while meals provided as part of a third-party’s conference may be allowable under certain circumstances, meals as part of a group educational dinner would be subject to a limit of $250 per person, per year. Furthermore, while many expenses associated with research activities, including clinical trials, are permitted, things like equipment loans are not. It is also unclear the extent to which the distribution of investigational new drugs are permitted, as only FDA approved drugs are covered in the bill.
CLSA has been the lead for the life sciences sector in lobbying against SB 790, and the bill is now eligible to be taken up on the Assembly floor, which is the last stop before reaching the Governor’s desk. In addition to lobbying in the Capitol, CLSA has spearheaded an aggressive letter-writing campaign that has resulted in roughly 400 letters from member company employees being sent to the Assembly.
We anticipate that the bill will be taken up on the floor shortly after the legislature returns from recess, though, prior to that vote, we also anticipate the bill will be heavily amended to specify the activities it aims to restrict, as opposed to its current broad restriction with a laundry list of exceptions.
Click here to add your voice and urge legislators to reject SB 790.
AB 265 would prohibit a drug manufacturer from offering in California “any discount, rebate, product voucher, or other reduction in an individual’s out-of-pocket expenses, including, but not limited to, a copayment or deductible” if a lower cost and therapeutically equivalent brand or generic drug is “available.” The bill has gone through several rounds of amendments and now excludes biologics and instances where a patient has received a prior authorization or step therapy exception.
The bill is currently eligible to be taken up on the Senate floor, and we anticipate a few substantive amendments to the bill prior to that vote.
CLSA will continue to fight vigorously on behalf of California’s life sciences industry and to protect our ability to discover new treatments and cures for patients. Questions? Please contact Brett Johnson, CLSA’s Senior Director, Policy and Regulatory Affairs (Bjohnson@califesciences.org).
Legislative Update from DC: FDA User Fees; ACA Repeal & Replace; Briefing on Cost & Value
July 23, 2017
On July 12, the House of Representatives passed the FDA Reauthorization Act of 2017 (FDARA), the legislation needed to renew the FDA’s prescription drug (PDUFA) and medical device (MDUFA) user fee programs. The bill was passed by voice vote under suspension of the rules, and now awaits action in the Senate. The Senate leadership announced that they will stay in session and delay August Recess until the 3rd week of August, allowing them additional time to vote on the reauthorization bill. The FDA’s current authority to collect user fees expires on Sept. 30, however, the FDA has announced that they will not be issuing pink slips (reduction in force notices – or, RIFs) due to the high degree of confidence that the legislation will be enacted before the authorization lapse. CLSA is pleased that the user fee package passed by the House includes several provisions of importance to the life sciences sector, including a provision to improve medical device facility inspections. We continue to urge our California senators to support the bill when it is brought before the Senate for a vote.
ACA Repeal & Replace
Although the House of Representatives passed a bill to repeal and replace the Affordable Care Act (ACA) several months ago, the Senate continues to rework its own version of such legislation to garner enough support to pass, amidst a divided Republican conference. As of press time, the Senate has passed procedural motions allowing the Better Care Reconciliation Act (BCRA) to come to the floor, with an anticipated 20+ hours of debate underway. Notably, although all previous drafts of repeal and replace legislation have included a full repeal of the medical device tax, one proposal currently under consideration in the Senate does not include such a provision. At least two amendments have been filed to support a repeal of the medical device tax. CLSA remains supportive of a full repeal of the medical device tax and is communicating with our California delegation to underscore our support for a swift and full repeal of the medical device tax.
Congressional Briefing: The Cost and Value of Prescription Medicines
On July 13, CLSA and 8 other state and regional life sciences associations hosted a congressional briefing on the cost and value of prescription medicines in the healthcare system. Speakers included Aaron Vandervelde (Berkeley Research Group), Mark Fendrick, MD (University of Michigan), and Jeanne Haggerty (BIO). The discussion was focused around the complexities surrounding the current drug pricing ecosystem, and explored factors for consideration in moving toward a value-based healthcare system.
CLSA’s Protect Access and Innovation public affairs campaign offers additional resources to help illustrate the value of medical innovation, including an infographic on The Complicated Way We Pay for Medicine.
Questions? Please contact Adam Lotspike, CLSA’s Associate Director of Federal Government Relations (Alotspike@califesciences.org).
South San Francisco Life Sciences Cluster Sees Increased Development
July 21, 2017
The City of South San Francisco continues its economic and population growth at a rapid pace. Construction sites are numerous and further development plans are under consideration. There are currently 5,300,000 square feet of research and development (R&D) and office space in the construction pipeline, along with 530 new housing units. New projects include another 450,000 square feet of office/R&D and another 2,100 housing units.
In early July, a joint study session was held by the South San Francisco City Council and the City Planning Commission on these current and new proposals for further development. One such new proposal includes the first ever building of residential housing in the specifically zoned life sciences cluster east of Highway 101. This would involve the addition of 1,200 units of residential housing to be built. As originally intended, the Oyster Point Development Plan, which was approved by the SSF City Council in 2011, was to upgrade the Oyster Point area of SSF’s life sciences cluster to include more R&D and office development only. This housing concept is being considered as a proposed amendment to that Plan.
CLSA provided testimony at the public study session as part of our continued close work with City Hall to address the growth and success of the cluster. CLSA’s longstanding policy is generally to support housing near transit corridors and Oyster Point does not fit that policy. Additionally, as this proposed housing development at Oyster Point would be located directly in the core of our industrial area, CLSA offered our thoughts on its potential impact on the life sciences cluster as a whole, local traffic conditions, transportation options for employees in SSF, and the lack affordable housing throughout the Bay Area.
For more than 25 years, CLSA, and its forebear BayBio, has housed our own offices in the life sciences cluster in South San Francisco. Our mission has always been to see this cluster on the eastern side of Highway 101 succeed and we will continue to advocate for policies to support that.
For more information on the public hearing, the development project, and for any questions, please contact Reese Isbell, CLSA’s Director of Local Government and Community Relations (Risbell@califesciences.org).
IPAB Will Not Be Triggered Until 2021, Medicare Trustees Report
July 20, 2017
On July 13,Medicare Trustees released their annual report predicting that, due to slower than expected growth in Medicare spending, the Independent Payment Advisory Board (IPAB) will not be triggered until 2021.
As you may know, IPAB was first proposed as part of the Affordable Care Act, and has remained a controversial entity with bipartisan opposition in Congress. According to last year’s report, IPAB was expected to be triggered this year, and since the Board had never been formed, HHS Secretary Price would have been solely responsible for finding over a billion dollars’ worth of cuts to Medicare for FY 2018 alone.
CLSA opposed the creation of the IPAB when it was proposed as part of the ACA, and we have been advocating for its repeal ever since. We are strongly supportive of legislation championed by California Rep. Raul Ruiz, MD (D-Palm Springs) – H.R. 849, the Protecting Seniors’ Access to Medicare Act – and have worked to secure 20 additional bipartisan members of the California Congressional Delegation as cosponsors of the bill: Reps. Nanette Barragán (D-Carson), Ami Bera, M.D. (D-Sacramento), Julia Brownley (D-Thousand Oaks), Ken Calvert (R-Corona), Salud Carbajal (D-Santa Barbara), Tony Cárdenas (D-San Fernando Valley), Paul Cook (R-Yucaipa), Jeff Denham (R-Modesto), Duncan Hunter (R-El Cajon), Darrell Issa (R-Vista), Steve Knight (R-Santa Clarita), Doug LaMalfa (R-Oroville), Ted Lieu (D-Santa Monica), Devin Nunes (R-Fresno), Scott Peters (D-San Diego), Ed Royce (R-Brea), and Linda Sánchez (D-Cerritos), Norma Torres (D-Pomona), David Valadao (R-Hanford) and Mimi Walters (R-Irvine).
CLSA’s letter of endorsement is available here, and our grassroots advocacy “Take Action” campaign is online here. We will continue to urge our California delegation to support the swift and full repeal of IPAB. Questions? Please contact Adam Lotspike, CLSA’s Associate Director of Federal Government Relations (Alotspike@califesciences.org).
CLSA Helps Educate Assemblymembers with Life Sciences Company Tours
July 19, 2017
On July 14, 2017, CLSA partnered with Organovo, Takeda and Sanford Burnham Prebys to host a tour for members of the California Legislative Technology and Innovation Caucus.
Assemblymember Kevin Mullin (Chair, Select Committee on Biotechnology), Assemblymember Todd Gloria, staff from the offices of Assemblymembers Kevin Mullin, Evan Low, Rocky Chavez and Marie Waldron’s office were part of a group of legislators and legislative staff to join the tour.
The tour started at Organovo, where CEO, Taylor Crouch, educated the attendees about cutting edge three-dimensional bioprinting and fabrication of living human tissue that can function like native tissues. This breakthrough technology is groundbreaking, with applications ranging from speeding up the drug development process to the treatment of organ diseases.
After Organovo, CLSA took the legislators and staff on a tour of Takeda’s San Diego facility and hosted a roundtable luncheon for CLSA members and the legislators. As part of the discussion CLSA staff and member companies briefed legislators about ongoing issues of concern to the life sciences sector. Some of the issues discussed included:
- Mandatory reporting of drug prices and related proprietary information (Senate Bill 17),
- Physician education and scientific information sharing on pharmaceuticals (Senate Bill 790),
- Manufacturing and R & D tax credits,
- Sharps and pharmaceutical waste take back, particularly the state preemption of local programs,
- Science, technology, engineering, and math (STEM) education,
- Precision medicine funding,
- Connected medical devices,
- Continuous glucose monitors, and
- Affordable housing and transportation concerns.
Both Mullin and Gloria addressed the audience and pledged to provide support to advance innovation in the life sciences sector. As a native of South San Francisco, Assemblymember Mullin has close ties to the biotech cluster in that city, and is keenly aware of the value our industry provides to the state in terms of employment, economic growth, and innovation.
Assemblymember Gloria acknowledged that the biotech sector is one of the hallmarks of Assembly District 78 (San Diego region), and committed to continuing his efforts to better understand our challenges and opportunities.
Lastly, the tour of the Sanford Burnham Prebys Medical Discovery
Institute (SBP) provided a comprehensive overview of biological research and the drug discovery process. The Institute’s CEO, Dr. Perry Nisen, educated the legislators about the work SBP is doing on cancer research, neuroscience, immunity, and metabolic disorders.
This event provided a great opportunity for our members to showcase their facilities and to have a robust discussion with the legislators about the current issues facing our industry. CLSA will continue to create similar opportunities throughout the rest of 2017 and beyond to build effective relationships with elected officials at all levels of government.
For more information on CLSA’s tours and roundtables, please contact Manny Heer, Director of Alliance Development & Advocacy (firstname.lastname@example.org).
CLSA Member Boehringer Ingelheim Breaks Ground on $217 Million Expansion of Fremont, California Manufacturing Facility
Expansion will triple capacity, add nearly 300 new jobs
Fremont, CA, July 21, 2017 – Boehringer Ingelheim announced a $217 million investment to upgrade and expand their Fremont manufacturing facility. This investment will bring nearly 300 new high-wage and highly-skilled positions to the City of Fremont. The site was recently awarded $25.5 million in two separate tax credits from the state, making them the largest award to a pharmaceutical company. This significant expansion will enable the company to increase its manufacturing capacity by one-third.
“The Bay Area is the largest biotech cluster in the world. We are happy to have made Fremont our home and are grateful for the support we have received from the community and the State of California,” said Jens Vogel, President and CEO of Boehringer Ingelheim Fremont, Inc. “We have made a commitment to the region and are excited to continue to invest in the Bay Area and expand our workforce to help increase patient access to highquality medicines.”
“The State Treasurer’s Office is excited to play a role in helping Boehringer Ingelheim expand and create nearly 300 permanent, high-paying jobs right here in California,” said State Treasurer John Chiang. “The incentives my office provides ensure that California will retain and create jobs, bolster the economy and clean the environment, today and into the future.”
Boehringer Ingelheim is a research focused company who invests nearly 20% of all net sales back into research and development – that equates to nearly 20 cents on every dollar. The Fremont facility focuses on manufacturing medicines for immunology, rheumatology and oncology.
About the Company
Boehringer Ingelheim Fremont, Inc., based in Fremont, CA, is a human biologics manufacturing facility. With a diverse team of over 500 people, we cover biopharmaceutical development and manufacturing from small scale to commercial, from drug substance manufacturing to fill/finish, packaging and medical device assembly. Everything we do, we do to help patients – through the reliable supply of high quality medicines and being on the cutting edge of technology and innovation.
Founded in 1885, Boehringer Ingelheim is the largest family-owned and privately-held pharmaceutical company in the world with approximately 55,000 employees worldwide that focuses on both human and animal health. This allows the company to focus long-term and invest heavily in research and development in both human and animal medicine with its 16 facilities in the United States.
Learn more about Boehringer Ingelheim Fremont Inc., visit us at: https://www.boehringer-ingelheim.us/our-focus/biopharma/fremont Twitter: #BoehringerUS ### Media Contact: Erin Crew Erin.email@example.com 203-798-5564
Cloudian, ScaleMatrix and OnRamp BioInformatics Cut Storage Costs and Speed Human Genomic Data Analysis
July 18, 2017 by ScaleMatrix Marketing Team
The original press release can be viewed here
Life-sciences ecosystem cuts storage costs by 50 percent, reduces complexity and drastically speeds data analysis of DNA sequencing and interpretation
San Mateo, California – July 18, 2017 – Cloudian, Inc., a global leader in hybrid-cloud object storage systems, today announced it is working with ScaleMatrix and OnRamp BioInformatics to improve efficiencies of data analysis and storage for life sciences companies focused on human genomics.
ScaleMatrix, a hybrid-cloud services provider, operates a 14,000-square-foot life-sciences incubator that couples a CLIA-certified genomics laboratory with a state-of-the-art datacenter powering Cloudian object storage and OnRamp BioInformatics genomic analysis software. The incubator helps reduce the barriers to entry for early-stage companies in the fields of genomics, molecular diagnostics and bioinformatics. Using an on-site Illumina sequencer at the incubator, researchers and biologists can generate genomic data then use genomic software from OnRamp BioInformatics to rapidly process and interpret data in ScaleMatrix’s GPU-enabled high-density compute farm and efficiently store valuable insights and data to Cloudian object storage.
The Cloudian deployment enables researchers to store, tag, track and retrieve large amounts of data intelligently and cost-effectively, while the OnRamp BioInformatics software streamlines and simplifies genomic data analysis so that biologists, clinicians and drug developers can unlock valuable insights.
“Our cradle-to-grave experience helps break down major barriers in life sciences, which involve massive data storage and analysis requirements that often aren’t within reach of start-ups,” said Chris Orlando, co-founder of ScaleMatrix. “Cloudian’s petabyte-scalable storage is the ideal platform to quickly and cost-effectively accommodate the data volumes inherent in genomic research. OnRamp BioInformatics completes the solution with an intuitive user experience, workflow automation and comprehensive data and analysis tracking to increase storage efficiency and research productivity.”
By 2025, 40 exabytes of data will be generated annually by DNA sequencers as two billion genomes are sequenced. One human genome generates 250 gigabytes of raw data, which can expand by a factor of from three to five as it is processed by any of the 11,000 open-source applications currently available for genome analysis. This data creates incredible management challenges for the future of precision medicine. Cloudian scale-out storage allows labs to store information at 70 percent less cost than with traditional on-premises storage; it also eliminates the variable charges of public cloud storage.
“DNA sequencing is important for our future and for human longevity, but the cost of storing and analyzing genomic data is a significant hurdle,” said Tim Wesselman, CEO of OnRamp Bioinformatics. “Too often, storage costs and data analysis complexity become bottlenecks to scientific breakthroughs by obscuring insights or even by forcing researchers to delete data that may have real value. Unlike public cloud storage, Cloudian has no access charges and easily scales to genomic proportions, so researchers can stay within their budgets and retain the data they need.”
“Object storage is ideal for the scale and management of the large amounts of data produced in the life sciences, and particularly genomics,” said Michael Tso, CEO of Cloudian. “We’re helping power developments in human genomics that can ultimately lead to new discoveries in health, drug development and longevity.”
MaxCyte and National Institute of Allergy and Infectious Diseases to Collaborate on Research for Ultra-Rare Disease Therapy
– Researchers to explore development of new therapies for X-linked chronic granulomatous disease (CGD) using MaxCyte’s gene-correction platform
Maryland, USA – 06 June 2017 – MaxCyte, Inc. today announces it has entered into a Cooperative Research and Development Agreement (“CRADA”) with the National Institutes of Health’s (“NIH”) National Institute of Allergy and Infectious Diseases (“NIAID”) to develop treatments for X-linked chronic granulomatous disease (“CGD”) using next-generation gene correction leveraging CRISPR/Cas9 and MaxCyte’s Flow Electroporation™ Platform.
CGD is an inherited genetic disorder that impairs the function of the immune system and leads to ongoing and severe bacterial infections. The disease affects approximately 1 in 250,000* people worldwide and is currently only treatable through high-risk treatments, such as allogeneic bone marrow transplantation.
NIAID will conduct pre-clinical research evaluating the effectiveness and safety of CRISPR-Cas9 gene editing on models of CGD by “correcting” the faulty gene that causes the disease. MaxCyte will supply mRNA molecules and focus on leveraging its Flow Electroporation™ Platform to develop robust and scalable processes that result in a clinically meaningful correction of mutated gene sequences.
Doug Doerfler, President & CEO of MaxCyte, said: “We are delighted to continue our collaboration with NIAID, one of the world’s leading infectious disease institutes, which is leveraging MaxCyte’s expertise in developing a new generation of genome editing therapy for CGD patients. We believe that this work will validate the use of our platform for developing gene-editing therapies via rapid, cost-effective manufacturing. This agreement, along with recent data announced from a research effort between MaxCyte and NHLBI of NIH in sickle cell disease, further demonstrates our commitment to deliver new therapies to patients where there is an extremely high unmet medical need.”
The MaxCyte/NIAID CRADA marks the latest step in the collaboration between the Company and the NIAID to advance new treatments for CGD, and reflects MaxCyte platform’s ability to be used in multiple fields of indication. MaxCyte received Maryland Stem Cell Research Fund grants in 2015 and 2017 to pursue its collaboration with the NIAID to develop preclinical processes and clinical-scale protocols for CGD and other rare diseases. MaxCyte also presented data generated in the collaboration at the 2015 American Society of Gene and Cell Therapy (ASGCT) annual meeting on the ability of genome editing in hematopoietic stem cells to restore oxidase activity. Earlier this year, MaxCyte shared data from the collaboration at the ASGCT’s annual meeting highlighting the achievement of therapeutic levels of gene correction in hematopoietic stem cells obtained from CGD patients.
MaxCyte is a US-based global company dedicated to driving the acceleration of the discovery, development, manufacturing and commercialization of next-generation, cell-based medicines. The Company provides its patented, high-performance cell engineering platform to biopharmaceutical partners engaged in drug discovery and development, biomanufacturing, and cell therapy, including gene editing and immuno-oncology. With its robust delivery platform, MaxCyte’s team of scientific experts helps its partners to unlock their product potential and solve problems. This platform allows for the engineering of nearly all cell types, including human primary cells, with any molecule, at any scale. It also provides a high degree of consistency and minimal cell disturbance, thereby facilitating rapid, large-scale, clinical and commercial grade cell engineering in a non-viral system and with low-toxicity concerns. The Company’s cell-engineering platform is FDA-accredited, providing MaxCyte’s customers and partners with an established regulatory path to commercialize cell-based medicines. MaxCyte is also developing CARMA, its proprietary, breakthrough platform in immuno-oncology, to rapidly manufacture CAR therapies for a broad range of cancer indications, including solid tumors where existing CAR-T approaches face significant challenges. For more information, visit http://www.maxcyte.com/
CARB-X Brings the San Diego AMR Community Together
On Tuesday July 18, CARB-X, one of the world’s largest public private partnerships focused on combating antimicrobial resistance (AMR), held a standing-room only event at BioLabs in San Diego. The purpose of the event was to: (1) bring together the antimicrobial resistance community in the greater San Diego area; (2) provide information about the mission and model of CARB-X; and (3) feature Forge Therapeutics and Cidara Therapeutics, the two San Diego companies that won CARB-X awards of up to $15M together in the first round of funding.
Ninety-four people were in attendance, including experienced life science executives, life science startups and pre-formation companies, as well as well-known academic leaders in the field from UCSD, Scripps and the California Institute for Biomedical Research. Companies and researchers represented a wide range of AMR fields, such as phage therapy, diagnostics, traditional small molecules and biologics, and were joined by partners in several industry-academic collaborations.
Heather Shane, Executive Director of CLSI’s west coast CARB-X accelerator, opened the program with an overview of CARB-X and qualifications for accessing its non-dilutive funding. Ms. Shane then moderated a panel featuring Neil Abdollahian, Chief Business Officer of Cidara Therapeutics, and Zachary Zimmerman, CEO of Forge Therapeutics, followed by an active exchange and networking reception with attendees. “This was a great event! Very lively discussions with people interested in the AMR space,” posted Karen Joy Shaw, Chief Scientific Officer at Amyplyx Pharmaceuticals, and leader in the AMR community.
CLSI Partner NexGeneGirls featured in first Genentech “Communities” Video Series
NexGeneGirls founder and Scientific Researcher at Genentech, Marlena Jackson, is featured in the first video in Genentech’s “Communities” series: What Does a Scientist Look Like? The series is intended to highlight remarkable employee services to the community. While an employee of Genentech, Ms. Jackson founded NexGeneGirls in 2011 to provide opportunities in STEM fields to young females of color in underserved communities. Since then, she has served over 300 girls through workshops, trainings, mentoring, and internships by providing a multi-generational teaching and substantive, hands-on science experience for African American female high school students. Watch the video here.
CLSI formally partnered with NexGeneGirls in October of 2016 to develop the 2017 NexGeneGirls Bootcamp and Summer Internship Academy. Over the spring, the program provided hands-on laboratory skills training taught by students in City College of San Francisco’s Stem Cell program to six high school females, helping them to master basic laboratory techniques such as pipetting, general biosafety, DNA extraction, and cloning and plasmid maps.
This summer, the girls interned three days per week in labs at UCSF, The Gladstone Institutes, and San Francisco State University. On the other two days, they provided after-school science workshops for middle school students at the Boys & Girls Club of San Francisco, and participated in enrichment seminars and field trips with professional women from industry.
The program will culminate with an Awards Celebration on July 27 during which the NexGeneGirls will present scientific posters on their internship research projects. The Honorable Edwin M. Lee, Mayor of San Francisco, will give remarks at the celebration.
NexGeneGirls was also a recent winner of the SPARK Igniting Global Change competition, led by a philanthropic network of young professionals who invest in innovative, grassroots, women-led organizations that are transforming communities.
About Marlena Jackson
A native of the Bayview, Marlena Jackson is the founder of NexGeneGirls, a grassroots organization committed to empowering young girls of color by providing access to careers in science, technology, engineering, and math (STEM). Aware of the asthma and breast cancer prevalent in her community, Ms. Jackson worked her way through City College of San Francisco’s Bridge to Bioscience program and San Francisco State University, and is currently a scientist at Genentech, one of the world’s leading biotech organizations. Marlena founded NexGeneGirls to develop science competency for young females of color from Bayview Hunter’s Point (BVHP) and other underserved communities, opening doors to rewarding and lucrative STEM careers. She has run the program on top of her full-time job for over four years.
In 2016, Ms. Jackson was selected as a Change Maker at The White House United State of Women Summit in Washington, D.C. She was also a featured speaker at Mayor Ed Lee’s Bay Area Women’s Summit that same year. She has been honored by Senator Mark Leno, Mayor Edwin Lee, the San Francisco Board of Supervisors, and the Commission on the Status of Women and Girls as one of the “Women Inspiring Innovation through Imagination: Celebrating Women in STEM.” A proud mother of two daughters, Marlena has also been honored by the Sun Reporter as one of the “The Talented 25” who are working to make the lives of others better.
To learn more about NexGeneGirls, contact Marlena Jackson at firstname.lastname@example.org.
Tax-Deductible Equipment, Supply & Consumable Donations
Northern California companies can make tax deductible donations of equipment, supplies and consumables – giving them a second life in cash-starved classrooms – through the Bio-Link Depot, which is moving to Oakland on September 1, 2017. The Depot accepts high caliber equipment and supplies from donating entities and makes them available to over 300 teachers and over 95,000 students in Northern California high schools, community colleges, and universities.
As biotech companies restructure, move or upgrade their equipment and supplies, the Depot is a compelling alternative to the auction house or landfill. Interested donors can arrange with the Depot to have their items picked up regardless of size or quantity. The Depot ensures that Donor’s contributions are known by recipients and thanks contributing companies on its website.
To make a donation or learn more about the Depot, contact Daniel Michael (510) 686-3411.
Biotech Primer article: Meds for Thinner Blood Can Equal Fewer Clots, But How?
STOPPING A BIG PROBLEM: BLOOD CLOTS
July 13, 2017
Original article can be found here.
MEDS FOR THINNER BLOOD CAN EQUAL FEWER CLOTS, BUT HOW?
The FDA’s recent approval of Portola Pharmaceuticals’ (South San Francisco, CA) new blood thinner drug Bevyxxa paved the way for the prevention of blood clots in patients hospitalized for conditions such as heart failure, stroke, and pulmonary disease. The medical term for blood clot is venous thromboembolism (VTE), but if we take it apart:
- “venous”means relating to a vein or the veins.
- “thrombo” is a blood clot.
- “embolism”involves the lodging of an embolus, a blockage-causing piece of material, inside of a blood vessel.
Hospitalized patients are at high risk for VTE because of their restricted mobility — not being able to move causes blood to pool and collect in the body. An especially dangerous type of VTE is deep vein thrombosis — blockage of a vein that is deep within the body, as opposed to near the surface of the body. If a portion of a deep vein thrombosis breaks off, it may travel to the lungs, causing a potentially fatal pulmonary embolism.
An estimated 24 million people are hospitalized annually due to VTE, so let’s find out how blood clotting is activated and learn the science of Bevyxxa.
A CLOT IN THE DARK
In healthy people, blood clotting is activated when tissue or a blood vessel is damaged, and involves specialized blood cells known as platelets — also known as thrombocytes. Either type of aforementioned damage results in activating platelets, which then form an initial “plug” at the site of injury. At the same time, proteins known as clotting factors are also activated. Clotting factors work together to produce a protein called fibrin, which is a fiber-like protein that forms a network of strands that, together with the platelets, form a clot at the site of injury. Clot formation in response to injury prevents excessive bleeding and enables healing to begin.
VTE occurs when blood clots form in the absence of an injury. These clots may break free and migrate to another part of the body, where it may interfere with blood circulation and impair organ function. If this occurs in a major organ such as the lungs, brain, or heart, critical injury or death may result. The clots may also grow to a size large enough to block the flow of blood in the blood vessel in which it originally developed. Risk factors for VTE may be acquired (including older age, major surgery, prolonged immobilization, certain type of cancers, pregnancy and hormonal contraceptives) or inherited.
MECHANISM OF ACTION: BEVYXXA
Bevyxxa and other drugs that prevent the formation of blood clots belong to a class of drugs called anticoagulants which thin the blood. Bevyxxa works by directly inhibiting one of the key clotting factors, Factor Xa. This differs from older anticoagulants such as warfarin that works by inhibiting Vitamin K, which is required for complete activation of clotting factors.
Some key benefits of direct Factor Xa inhibition include faster onset, less interaction with other medicines or certain foods, and fewer bleeding events observed during clinical trials, leading to a better safety profile. Bevyxxa is the first oral Factor Xa inhibitor to be approved, and has been approved for use for up to 42 days. These attributes mean Bevyxxa can be prescribed to a patient to continue taking the anticoagulant after release from the hospital.
Patients were selected for treatment with Bevyxxa based on increased levels of “D-dimers” in their blood. D-dimers are degradation products of fibrin, the key protein component of blood clots. When our body breaks down blood clots, D-dimers are produced. Thus, having higher than normal blood levels of D-dimers is a sign that higher levels of blood clots are present.
AN ANTICOAGULANT U-TURN
Anticoagulant drugs can be life-saving; however their inhibitory effects may need to be reversed due to major bleeding, or in the case of an emergency surgery. Portola’s andexanet alfa, currently in late stage development, reverses Factor Xa inhibition. Andexanet alfa works by irreversibly binding Bevyxxa, preventing it from binding clotting Factor Xa. If Bevyxxa can’t interact with and inhibit Factor Xa, it no longer prevents blood clotting.
COCKTAIL FODDER: BLOOD THINNERS IN THE WILD
Ever wonder how mosquitos and ticks are able to keep the blood flowing from their point of attack until they’ve had their fill? It turns out that their saliva contains a natural anticoagulant which prevents platelets from being activated. Fortunately, the effect is only temporary and localized to the site of the insect bite.
Foods that we eat may also impact blood clotting ability. For example, foods high in vitamin E such as almonds and hazelnuts, as well as spices such as cayenne pepper, garlic, ginger, and onion have some natural anticoagulant effects, while foods high in vitamin K such as leafy green vegetables, egg yolk, and soybeans may promote coagulation. For most healthy people, the relatively small amounts of these foods consumed in a normal diet would not have a significant impact on blood clotting; however, those on anticoagulant medicines may want to consult their physician about any possible dietary impact on their medicine’s efficacy.