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Category Archives: BioEngineering

Triple-Gene Announces Completion of Enrollment and Dosing in Phase 1 Trial of INXN4001, First Multigenic Investigational Therapeutic Candidate for…

"We are excited to have reached this important milestone in the clinical evaluation of INXN-4001 for treatment of end-stage heart failure," stated Amit Patel, MD, MS, Co-Founder and Medical Director of TripleGene. "Heart failure rarely results from a single genetic defect, and while single gene therapy approaches have been studied, these treatments may not fully address the causes of the disease. Our unique multigenic approach is designed to stimulate biological activity targeting multiple points in the disease progression pathway."

Triple-Gene's investigational therapy uses non-viral delivery of a constitutively expressed multigenic plasmid designed to express human S100A1, SDF-1, and VEGF165 gene products, which affect progenitor cell recruitment, angiogenesis, and calcium handling, respectively, and target the underlying molecular mechanisms of pathological myocardial remodeling. The plasmid therapy is delivered via RCSI which allows for cardiac-specific delivery to the ventricle.

"Heart failure is the leading cause of death worldwide and represents a significant and growing global health problem. Aside from heart transplant and LVAD, current treatment options for those patients with end-stage disease are limited," commented Timothy Henry, MD, FACC, MSCAI, Medical Director of the Carl and Edyth Lindner Center for Research and Education at The Christ Hospital and a member of the Triple-Gene Medical Advisory Board. "The INXN4001 investigational therapy represents a biologically-based method focused on repairing the multiple malfunctions of cardiomyocytes, and I look forward to seeing the results of this initial safety study and further exploring the promise of this innovative treatment approach."

Triple-Gene will present preliminary data from the Phase 1 study at theAmerican Heart Association Scientific Sessionsat the Pennsylvania Convention Center in Philadelphia. A poster titled "Safety of First in Human Triple-Gene Therapy Candidate for Heart Failure Patients" will be presented on Sunday, November 17thfrom 3:00 pm - 3:30 pm ETin Zone 4 of the Science and Technology Hall.

About the Phase 1 Trial of INXN-4001INXN-4001 is being evaluated in a Phase I open label study in adult patients with implanted Left Ventricular Assist Device (LVAD). The study is designed to investigate the safety and feasibility of supplemental cardiac expression of S100A1, SDF-1 and VEGF-165 from a single, multigenic plasmid delivered via Retrograde Coronary Sinus Infusion (RCSI) in stable patients implanted with a LVAD for mechanical support of end-stage heart failure. Twelve stable patients with an implanted LVAD were allocated into 2 cohorts (6 subjects each) to evaluate the safety and feasibility of infusing 80mg of INXN4001 in either a 40mL (Cohort 1) or 80mL (Cohort 2) volume. The primary endpoint of safety and feasibility is assessed at the 6-month endpoint. Daily activity data are also collected throughout the study using a wearable biosensor. Dosing on both Cohorts 1 and 2 has been completed, and patients continue follow-up per protocol.

About Triple-GeneTriple-Gene LLC is a clinical stage gene therapy company focused on advancing targeted, controllable, and multigenic gene therapies for the treatment of complex cardiovascular diseases. The Company's lead product is a non-viral investigational gene therapy candidate that drives expression of three candidate effector genes involved in heart failure. Triple-Gene is a majority owned subsidiary ofIntrexon Corporation(NASDAQ: XON) co-founded by Amit Patel, MD, MS, and Thomas D. Reed, PhD, Founder and Chief Science Officer of Intrexon. Learn more about Triple-Gene atwww.3GTx.com.

About Intrexon CorporationIntrexon Corporation (NASDAQ: XON) is Powering the Bioindustrial Revolution with Better DNAto create biologically-based products that improve the quality of life and the health of the planet through two operating units Intrexon Health and Intrexon Bioengineering. Intrexon Health is focused on addressing unmet medical needs through a diverse spectrum of therapeutic modalities, including gene and cell therapies, microbial bioproduction, and regenerative medicine. Intrexon Bioengineering seeks to address global challenges across food, agriculture, environmental, energy, and industrial fields by advancing biologically engineered solutions to improve sustainability and efficiency. Our integrated technology suite provides industrial-scale design and development of complex biological systems delivering unprecedented control, quality, function, and performance of living cells. We call our synthetic biology approach Better DNA, and we invite you to discover more atwww.dna.comor follow us on Twitter at@Intrexon, onFacebook, andLinkedIn.

TrademarksIntrexon, Powering the Bioindustrial Revolution with Better DNA,and Better DNA are trademarks of Intrexon and/or its affiliates. Other names may be trademarks of their respective owners.

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Triple-Gene Announces Completion of Enrollment and Dosing in Phase 1 Trial of INXN4001, First Multigenic Investigational Therapeutic Candidate for...

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Carson River restoration projects underway – The Record-Courier

From Alpine County to the Fort Churchill area, restoration projects are underway along the Carson River.

A group of project coordinators and other stakeholders gathered Wednesday to talk about keys to success and challenges. The seminar at Western Nevada College was hosted by the Carson Water Subconservancy District, the Carson River Coalition, and the Nevada Division of Environmental Protection.

All three of these projects had funding, which is often the biggest hurdle, said Kimra McAfee, executive director, Alpine Watershed Group.

McAfee described two completed projects in Hope Valley meadow conducted in 2010 and 2016, and a third project to begin construction next year.

The upcoming project entails two sites, one to reduce erosion, stabilize river banks, improve water quality and enhance aquatic habitat, and the other to shore up work done in one of the older projects.

Work at both sites is being funded through the state of California from fines levied against two ski resorts for clean water violations.

What is really special about this funding is it will go out four years and cover monitoring and adaptive management, said McAfee.

The challenges affect projects up and down the river: the variability of precipitation, typified by recent wet winters, which may only get worse with global warming, said McAfee, and slow and hard to grow vegetation.

We live in an area that can have both intense heat and frigid cold, said Francisca Mendive, Carson Valley Conservation District, which affects vegetative growth.

Mendive talked about a project near Cradlebaugh Bridge in Douglas Countys Johnson Lane area to reestablish and stabilize banks which are now cut in 10 to 15 foot verticals.

The project involves using willow cuttings and other bioengineering practices.

I want to debunk the scary term, bioengineering, she said. Property owners are not usually welcoming of a project, especially one with a big scary term like bioengineering.

A property owner helped draw attention to the need for work now underway years later in the Fort Churchill area.

Rob Holley, Dayton Valley Conservation District, showed a series of aerial photographs of the site starting in 1994 highlighting how the riverbed there has widened and receded, creating 15-foot vertical cut banks dangerously close to Fort Churchill Road and Buckland Ditch, which is used to irrigate 600 acres.

Its threatening infrastructure, said Holley.

The project is scheduled to start next summer.

We have a narrow window for construction. And the distance to the site adds to the cost, said Holley. This is time critical because it continues to erode.

A panel discussed funding, always the biggest obstacle.

Representatives from California and Nevada talked about federal grants available for projects to reduce non-point source pollution, so-called 319 money named for that section of the Clean Water Act.

States have some leeway how to administer the program and Birgit Widegren, Nevada Division of Environmental Protection, talked about Nevadas requirements.

The bad news is we get $1 million annually, said Widegren.

Thats in contrast to the $4 million annually allocated to California, but the Nevada program is somewhat less stringent and more inclusive, funding education programs as well, for example.

Ed James, general manager, CWSD, said the subconservancy district has roughly $300,000 to $400,000 annually to award projects. The money, which is not from the federal government, can also be used by recipients as matching funds for federal grants.

CWSD sends out a notice to apply in January and a panel reviews projects in March.

We usually get applications for five times what we have to give, said James. Our priority is construction.

And Jim Lawrence, deputy director, Nevada Department of Conservation and Natural Resources, talked about new funding to come from the state.

Assembly Bill 84 passed this year authorized up to $217 million in bonds for conservation and wildlife management, including $10 million for work on the Carson and Truckee rivers.

A staff position to oversee the program is slated to start in July, and regulations, which will outline key details, could be hammered out either before then or once the position is filled, said Lawrence.

This is going to be highly competitive, he said. As youre thinking about this program, what I think is most beneficial is telling a story, collectively telling a story for the Carson River corridor.

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Carson River restoration projects underway - The Record-Courier

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Innovation Shines at CREATIVATE – Lehigh University News

So Huffman decided to create a device that can be attached to a car and, at the touch of a button, control the vehicles noise level. As such, his company, Valvetronic Designs, was born: When the car is in loud mode, the valved exhaust system he created is open and the exhaust is able to flow right out of the car. However, in quiet mode, the exhaust is trapped within the device, preventing it from making any loud noises.

As Valvetronic Designs began to grow, Huffman sought the help of his roommate, Scott Gruninger 20.

I just brought this excellent man on because I'm terrible at managing my financials and such, Huffman said. So I need a man to help me with supply chain financials, and he's the guy.

Said Gruninger: Before I worked with Austin I had a 3-D printing business...And then, Austin and I, being roommates, we bounced ideas off of each other and he was like, Why don't you come do this full time with me? And I was like, Yeah!

Huffman is a supply chain management major, and Gruninger studies management consulting.

Were on track to do $30,000 in sales this month...We dont have any overheads because its just us, Huffman said. We outsource our manufacturing, and we build our supplies off site, so it doesn't cost us a bunch to make these systems, and it's just pushing marketing out. Getting my name out there is how we're going to [grow].

Lauren Farrell 13 combined two of her passions to create luxury handbags for sports fans.

At Lehigh, Farrell studied design and entrepreneurship.

I remember that the entrepreneurs had this passion that was totally contagious and I said, I have no idea what I want to do, but I want to be like them.

Farrells inspiration came about when she discovered a lack of fashionable bags on the market that met the strict regulations of sports centers. She decided to create a line of bags that met those restrictions, but still could appeal to the woman who loves fashion.

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UMD researchers are using a $2.8 million grant to study spinal cord injuries and dementia – The Diamondback

Professor Steven Jay in the lab where he oversees research on the link between spinal cord injuries and cognitive degeneration. (Joe Ryan/The Diamondback)

A team of University of Maryland and University of Maryland, Baltimore researchers is investigating the connection between dementia and spinal cord injuries with the help of a $2.8 million dollar grant awarded earlier this month from the National Institutes of Health.

The project a collaborative effort between the universitys bioengineering lab and the University of Maryland medical school, which is housed at the University of Maryland, Baltimore aims to examine a link between spinal cord injuries and degeneration in the brain.

The possible link has to do with particles called extracellular vesicles, which cells use for communication, said Steven Jay, an associate bioengineering professor working on the study.

[Read more: Police encounters can be risky for people with autism. A UMD lecturer thinks VR can help.]

After a spinal cord injury, the surrounding cells become inflamed and start to release the vesicles; they travel within the central nervous system and ultimately land in the brain. And now, researchers are looking to see if the spinal cord injury can lead to degeneration in the brain, said Alan Faden, a trauma professor at the medical school.

My mission, for now going [on] four years, is to develop the science that can be translated to humans to protect the brain and the spinal cord from secondary damage, Faden said. And to get people to recover faster and better.

Somehow, the particles affect the brain in a negative way, resulting in higher instances of dementia, said Jay, who is studying extracellular vesicles. But researchers want to figure out how.

The research team is working to understand the biological process surrounding the vesicles, Jay said. With an understanding of the biology, the team can study ways to reverse their negative impacts.

Instead of having them, you know, promote the problem, Jay said. Could we program them to actually solve the problem?

[Read more: A diverse group of UMD researchers is studying the link between brain and gut health]

Junfang Wu, an associate professor at the medical school, is leading the project. She said shes eager to explore the relationship with the vesicles, which she said could improve understanding of spinal cord injuries and their cognitive effects.

Thats the exciting part, Wu said.

Wu has been researching spinal cord injuries for years and said that the average age for spinal cord injuries has increased. These injuries, she said, often come from falls. And the risk of falls increases in older people, along with the risk of spinal cord injury, Wu said.

Still, Jay said, vesicles role in cell-to-cell communication was discovered a little over a decade ago. So though the researchers could identify a key player in spinal cord injuries, there is a long way to go before they discover something concrete and applicable.

Its following up on very recent knowledge, Jay said. So its not something thats going to be leading to therapy in two years or something like that.

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UMD researchers are using a $2.8 million grant to study spinal cord injuries and dementia - The Diamondback

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Plan your weekend around this year’s 41 North Film Festival | News, Sports, Jobs – Daily Mining Gazette

The film "Angelique's Isle" follows an Ojibway woman and her husband who are stranded on an island off Isle Royale. provided photo

I love all of them, of course, said Erin Smith, director of the 41 North Film Festival, when asked to name her favorites.

The film festival features 24 films this year, starting on Thursday morning and running through Sunday night. The event is free and open to the public, but tickets are required to track attendance and seating.

Smith was eventually able to narrow her favorites down, starting with Angeliques Isle, which shows Saturday evening.

A lot of people are familiar with the story, Smith said.

The film follows an Ojibway woman and her voyageur husband who are left for dead on an island off Isle Royale while working with copper speculators. One of the directors, Anishinaabe filmmaker Michelle Derosier, will be in attendance at the film festival for the showing.

A still from the movie "Picture Character". provided photo

Earlier in the day on Saturday, a grouping of films relating to environmental transformations will be shown. Anthropocene, Biggest Little Farm, and Honeyland all focus on the ways people and the environment are impacting each other.

Were actually having a little farmers market in the lobby, Smith said.

Honeyland will also be followed by a panel discussion with Kathleen Halvorsen, Victor Busov, and Melissa Hronkin, experts in beekeeping, forestry and biodiversity.

The last favorite Smith was allowed to name is a film shown on opening day, Human Nature. The film dives into the possibilities and perils of gene editing unlocked by CRISPR.

Its a very hot topic, Smith said.

"Angelique's Isle" is playing Saturday night. provided photo

Following the film, a panel discussion will be held with Paul Goetsch, Caryn Heldt, and Alexandra Morrison who have doctoral degrees in biochemistry, bioengineering, and philosophy, respectively.

Between films, the lobby of the Rozsa Center will play host to several local musicians and activities including cookie decorating and a costume contest.

Smith, as director, chooses the films for the festival each year, looking for ones that arent going to be shown at other venues in Copper Country. She said she tries to strike a balance between things that Michigan Tech students and community members will be interested in, as well as a balance between addressing serious issues and being entertaining.

Sometimes people will send me suggestions for films that end up being part of the program as well, she said.

Maiden, a film about the first competitive all-female sailboat crew showing on Sunday, was suggested and sponsored by the local yacht club.

The film "Human Nature" shows Thursday night. provided photo

Smith also took the chance to recognize others contribution to the festival.

We have some incredible volunteers, she said.

For tickets and more information, visit http://41northfilmfest.mtu.edu/2019/ or contact the Rozsa Center for the Performing Arts.

Schedule-

Thursday, Oct. 31, 2019

9:30 a.m. 10:30 a.m. Fortier / Acting

5 p.m. 6:30 p.m. American Factory

6:45 p.m. 7:30 p.m. Music in the Lobby

7:30 p.m. 9:30 p.m. Human Nature

9:30 p.m. 10 p.m. Music in the Lobby

10 p.m. 11:30 p.m. Scream Queen! My Nightmare on Elm Street

Friday, Nov. 1, 2019

3:30 p.m. 4:45 p.m. Shorts Program 1

5 p.m. 6:30pm Midnight Traveler

6:30 p.m. 7:30 p.m. Music in the Lobby

7:30 p.m. 9:15 p.m. Picture Character

9 p.m. 10:30 p.m. Music in the Lobby / Emoji Cookie Decorating

10:30 p.m. 12 a.m. Greener Grass

Saturday, Nov. 2, 2019

10:15 a.m. 11:15 a.m. Michelle Derosier and students

12 p.m. 1:30 p.m. Anthropocene

2 p.m. 3:30 p.m. Biggest Little Farm

4 p.m. 6 p.m. Honeyland

6 p.m. 7:30 p.m. Music in the Lobby

7:30 p.m. 9:45 p.m. Angeliques Isle

9:45 p.m. 10:30 p.m. Music in the Lobby

10:30 p.m. 12 a.m. Give Me Liberty

Sunday, Nov. 3, 2019

12 p.m. 1:30 p.m. Los Reyes

1:45 p.m. 3 p.m. Curtis Fortier

3:30 p.m. 5 p.m. The Bit Player

5:30 p.m. 6:30 p.m. Shorts Program

6:30 p.m. 7 p.m. Music in the Lobby

7 p.m. 9 p.m. Maiden

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BioMakerspace to open this coming IAP in Building 26 – The Tech

By Edwin SongOct. 31, 2019

A new biology makerspace, called the BioMakerspace, is scheduled to open this coming IAP in the basement of Building 26. The space, which is currently under construction, will be open to the entirety of the MIT community to be used for whatever the student users really want to use it for, said Justin Buck PhD 12 in an interview with The Tech. Buck is the manager of the BioMakerspace and is currently overseeing its construction.

Buck said the lab will have Biosafety Level 2 capabilities and contain all the basic equipment, including a tissue culture room, incubators, centrifuges, microscopes, pipettes, thermal cyclers, a refrigerator, a PCR, and common reagents.

Construction of the space is in the final stages and equipment is in the process of being obtained. We're really hoping to have a very large and active launch over IAP, said Buck. We plan to have workshops for folks to come and participate in if they don't have exposure to biology or would like to see and understand what its like to work with different projects.

MIT students looking to work in the lab must submit a project application and undergo lab training. Steve Wasserman, one of the biological engineering instructors who has helped run the biology makerspace program since its early stages, told The Tech in an interview that the lab has a stipulation that allows students to maintain intellectual property of the work they do in the lab and use it to launch their own companies.

A lounge next to the lab will provide a place to work and congregate. The idea that we're pursuing is that this facility will serve as the nucleus for a community that is interested in life science, Wasserman explained.

Construction began April 2019 after several years of what Buck described as a very successful pilot phase, which operated out of the bioengineering department teaching laboratories.

Wasserman said the idea for a biology makerspace program arose several years ago out of student demand. Students get all kinds of crazy ideas and they want to do them, and a lot of times there are barriers to doing them in various research labs around campus, Wasserman said, citing the lack of spaces with appropriate equipment, intellectual property rights, and supervision.

Past projects through the program range from therapeutic drug delivery to kombucha, mostly independent projects thought up by the students, Buck said. Thats what I think makes the space most unique and what it is. Perhaps its greatest value as an asset to the community is that it is open to any ideas.

Associate Provost Krystyn Van Vliet PhD 02 told The Tech in an interview that having an independent lab for the biology makerspace has several benefits over continued use of the bioengineering teaching labs, such as not having to risk disrupting classes being taught in the teaching labs and flexibility in regards to research groups and topics.

Teaching spaces have to have things set up just so, and they're not places where we tend to do projects that would mix different research groups, Van Vliet said. Creating a mechanism where students, postdocs, other kinds of research staff, and faculty can work on things together that might not be within the research interests and domain of the existing faculty is how new ideas get started.

Both Buck and Van Vliet mentioned the establishment of the biology makerspace as part of a campus-wide trend towards makerspaces. For example, Van Vliet pointed to the planned Project Manus community-wide makerspace that will be on the first floor of the Metropolitan Warehouse.

Buck said the BioMakerspace is the first space that is really enabled and focused on working with biology as a medium.

Construction of the BioMakerspace is conducted by Greene Construction and sponsored by the biological engineering and chemical engineering departments, along with a donor whose identity Buck declined to disclose.

Van Vliet said additional funds were provided by the MIT Committee for Renovation and Space Planning (CRSP), which she co-chairs. According to Van Vliet, CRSP was responsible for much of the behind-the-scenes work in arranging for the bio makerspace to be designed and built.

Along with Buck and Wasserman, several other biological and chemical engineering faculty and instructors, including the department heads, are closely involved in the project. Additionally, a BioMakers student groupis currently helping with planning and launching the itinerary of activities for IAP, Buck said.

Wasserman described student feedback as instrumental in the design of the layout and contents of the lab and lounge. In the more public areas, the windows are bigger. The windows get smaller and smaller as you go to more private places, Wasserman said. We asked in one of our surveys how open they wanted to space to be, ... and the students said that they didnt want to be in a fishbowl.

Students interested in the BioMakerspace are encouraged to contact Justin Buck at jbuck@mit.edu.

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