BYMEAGHAN CASEY

In the summer of 2014, the ALS Ice BucketChallenge took the world by storm, becoming aglobal social media phenomenon and raising morethan $220 million in the fight against ALS. Pat Quinn, who had been diagnosed with the

disease in March 2013 and launched the challengewith fellow patient and advocate Pete Frates, neverimagined it would take on such a life of its own.“It’s really amazing, the power of social media.

What resulted from it is the most amazing thing. Itcreated hope among patients who really didn’t haveany,” said Quinn, closing out MassBio’s PatientAdvocacy Summit on Oct. 27. He was joined at theevent by Andrew Frates, Pete’s brother.“August 2014 was a crazy, crazy month for my

family and I like to think of it as the first buildingblock in the end to ALS,” said Andrew, who hasbecome his brother’s caretaker.Both Quinn and Frates were diagnosed with

ALS before their 30th birthdays, and the pair—hailingfrom Yonkers, N.Y. and Beverly, Mass. respectively—came together in the hope of spreading greaterawareness of the disease. Their campaign elevatedALS to a topic that was discussed in the homes ofmillions of people. The funds raised are now beingused to support ALS care services, public policyinitiatives and research, including four collaborativeresearch initiatives to build understanding of the

SUMMIT HELPS BUILD PATIENT-CENTERED BIOTECHS

See SUMMIT Page 7

Vol. 9, No. 1 | A publication of the Massachusetts Biotechnology Council |Winter 2016

Page 3 LEVERAGING CONTRACT PARTNERSHIPS

POLICYMAKERS CONFRONTVALUE AND THE OPIOID CRISISAT ANNUAL BREAKFASTPages 4-5

PAT QUINN

HOPE FORCANCERTREATMENTPage 8

PHOTO: SEAN BROWNE

As we approach the opportunities andchallenges ahead of us in 2016, we do so underthe guidance of our new mission statement—toadvance Massachusetts’ leadership in the lifesciences to grow the industry, add value to thehealthcare system and improve patient lives.The statement is meant to bring focus to the

people at the heart of the work we all do: thepatients. We work to bring that mission to lifethrough programming like our second annualPatient Advocacy Summit, a sold-out event thatbrought industry leaders and patient advocatestogether to examine ways in which life sciencescompanies can more fully incorporate the patientvoice into the work they do throughout the drug

development cycle.Patient advocacy continued to be a theme at our

recent Policy Leadership Breakfast, where weaddressed issues touching the lives of residentsacross Massachusetts—with particular focus onboth drug access and abuse. MassBio has and willcontinue to convene stakeholders to discussalignment on drug cost and pricing topics, throughconversations and programming at our AnnualMeeting and events later in the year. We willcontinue to work with legislators such as AttorneyGeneral Maura Healey, who joined us at thebreakfast, and Gov. Charlie Baker, who will bejoining us in March for our Annual Meeting, tofind solutions.

Also at the policy level—by convening theBiotech Caucus, participating with the HealthPolicy Commission and serving on the ManagedCare Organization Task Force—we hope to ensurethe work our member companies are doing isincentivized and rewarded in any evolvinghealthcare system. It is critical in Massachusettsand beyond that we preserve and foster innovationso patients can receive the cures they sodesperately need.Thank you for your support and I wish you the

best as you continue to innovate. We will continueto champion for you.

Robert K. Coughlin is President & CEO of MassBio.

2 — MassBio NewsWINTER 2016

ROBERT K.COUGHLIN

Akrivis TechnologiesAleta

Artisan Healthcare ConsultingASOthera PharmaceuticalsBeat NB Cancer Foundation

BiognosysBioniqueBioSciKinBiovest

Bluefin BiomedicineBoston Clinical Trials

ChemPartnerCodiak BiosciencesCogitas Consulting

ColdstashContinuum HealthCommunications

Decibel TherapeuticsDEFINIENS, Inc

EH&EEuretos

Founders Science GroupGates Healthcare Communications

GnuBioHermeon Medical

Jana CareJuniper PharmaceuticalsKadmon Corporation

KinderPharmKiniksa PharmaceuticalsKSQ TherapeuticsLumioHealth

Microchips BiotechNanobiosymOptimal Wealth

PadlockPersonalisPreteoTheraProskauer Rose

Quad TechnologiesRaze TherapeuticsSlalom Consulting

STATStrategic Workspaces

SyreonThe Bowdoin Group

The Epilepsy FoundationTORQUE

Toxic ReportsTrevigenTrifermedUltivue

Vetter Pharma VittamedVL34

Waveguide CorporationWithumSmith+Brown

Xtuit

NEW MASSBIO MEMBERS

A PUBLICATION OF:Massachusetts Biotechnology Council

300 Technology SquareCambridge, MA 02139

617-674-5100 n www.massbio.org

Robert K. CoughlinPresident & CEO

Joseph FazioVP of Finance & Administration

John HallinanChief Business Officer

Sarah MacDonaldExecutive Vice President

Kendalle Burlin O’ConnellVP of Member Services

BOARD OF DIRECTORSGlenn Batchelder, ChairAbbie Celniker, Vice ChairMichael W. O’Hara, TreasurerDavid Lucchino, Clerk

Geoff MacKay, Immediate Past ChairMark R. Bamforth

Jim BurnsTim ClacksonRenee ConnollyGeoffrey CoxJeffrey Elton

Chris GarabedianScott Gillis

Steven P. GilmanRobert GottliebSam Guhan

Jose-Carlos Gutierrez-RamosJames HoyesEdwin Kania

Laurie Bartlett KeatingMelissa Bradford KlugMark Leuchtenberger

Liz LewisAntony Loebel

William McDermottLisa OlsenJohn OrloffMichael PelliniAdelene PerkinsDeanna PetersenAlice PomponioSteven Richter Rick RussellAmit Sachdev

Kathleen TregoningCharles Wilson

Lawrence Wittenberg PRODUCED BY:

GRANT COMMUNICATIONSCONSULTING GROUP

Boston/New York781-598-8200

gccg@grantgroup.comwww.grantgroup.com

VENTURE CAPITAL INVESTMENT

Venture investment in Massachusetts rose to an all-time high of $2 billion in 2015.

APPROACHING 2016 WITH A PATIENT-DRIVEN MISSION

MassBio NewsWINTER 2016 — 3

BY MEAGHAN CASEY

An increasing number of biopharma companies are turningto contract research and manufacturing partners for time andcost savings, as well as for access to scientific expertise.Worldwide, nearly a third of biopharma research dollars arespent on outsourcing services offered by CROs. That figurewas approximately $27 billion in 2015, and is projected tokeep growing. More than $17 million was spent in the CMOmarket last year. At the MassBio CRO/CMO Symposium, held on Nov. 13 at

the Renaissance Boston Waterfront Hotel, more than 300attendees gathered to focus on leveraging strategic partnershipsto accelerate drug development and maintain momentum inMassachusetts. The event is an annual one that shines aspotlight on the strength and success of the CRO and CMOcommunities in the Commonwealth. “We know that more companies are contracting services

out—or remaining virtual—in order to bring new products tomarket faster and more efficiently, and we know Massachusettscompanies can provide the full spectrum of services,” saidMassBio President & CEO Robert K. Coughlin. “We need to

talk about how to ensure our competitiveness well into thefuture.” Michael Martorelli, Director of Fairmount Partners, gave a

keynote presentation on piloting strategic options and stressedthe importance of maintaining focus in terms of nature andrange of services, geographic scope, types of prospectiveclients, desired rate of revenue growth and extent ofprofitability. A series of case studies, interactive panel discussions and

networking sessions followed, providing ample time to explorethe various techniques and approaches being used by bothvirtual and established companies. Panelists discussed trends inoutsourced biopharma and global and local options foroutsourcing—stressing that locally grown companies canprovide ease of access, operational efficiency and anunmatched level of technical expertise. “A lot of what we do is in the discovery phase, and time

matters,” said Jim Jersey, President of Agilux. “It’s importantto be adaptive and think about the advantages of proximity.” “If you’re looking for services that are capacity-oriented vs.

capability-oriented, you have different needs,” said RichlandTester, Senior Manager at Celgene. “If you just need the space,that’s one thing, but if you’re selecting a company for whatthey can do, you’re probably going to look more locally. Youmight have to regularly visit the biologists in the lab to build acloser relationship.” “You want to invest in quality,” said Jörg Holenz, Director

of Discovery and Preclinical Sciences at AstraZeneca. “Findthe niche CRO best suited to your project and leverage thatbrain power early on.”

Also at this year’s symposium, the amount of roundtablediscussions doubled, with 18 hot topics for attendees to choosefrom. In the closing keynote presentation, Willy Shih, professor at

Harvard Business School, spoke about the risks andopportunities for the Massachusetts cluster. “The choices you make today will influence the long-term

health of this cluster,” said Shih. “The easiest choices mightnot be the best ones. We’ve seen this in the electronicsindustry, the computer industry and the semiconductorindustry.”Shih talked about his time at Kodak—which for a long time

focused on film and outsourced manufacturing. Then theindustry went digital, forcing the company to refocus itspriorities back to the camera itself. Shih said the biotechnologyindustry also might need to refocus. “There’s a huge difference between being able to create a

couple milligrams in a lab and a year’s worth of supply,” saidShih. “We really need to strengthen biomanufacturing in thiscountry.” In an effort to improve and streamline access to local

resources, MassBio launched the Massachusetts CRO/CMOGateway in partnership with OnDeckBiotech. This onlineportal facilitates direct, real-time access to resources thataccelerate development timelines and reduce costs for clustermembers. For biotech and drug development companies, theportal combines resources to search for specific capabilities,detailed company profiles and tools to quickly executeconfidentiality agreements. Visit the MA CRO/CMO Gateway at www.MassBio.org.

Featuring Gov. Charlie BakerROYAL SONESTACAMBRIDGE, MA

SAVE THE DATE: MARCH 31- APRIL 1, 2016

Strategic partnerships acceleratedrug development and maintainmomentum in Massachusetts

SYMPOSIUM SHINES A SPOTLIGHT ON THESTRENGTH OF THE CRO AND CMO COMMUNITIES

Shikha Barman, CEO of Integral BioSystems, showcased hercompany's innovative drug delivery technology, OcuSurf.

MassBio President Robert K. Coughlin connected with TravisMcCready, President of the Mass. Life Sciences Center.

Michael Martorelli, Director of Fairmount Partners,delivered the keynote presentation.

Register today at www.MassBio.org.Sponsorship opportunities available:email Erica.Feinberg@MassBio.org

4 — MassBio News WINTER 2016 MassBio News WINTER 2016 — 5

BY MEAGHAN CASEY

MassBio’s 2016 Policy Leadership Breakfast tackled twoof the most pressing issues facing the Commonwealth—agrowing opioid addiction epidemic and a healthcare systemthat needs to find a balance among access, quality and cost.Attorney General Maura Healey opened the event, held on

Jan. 27 at the Omni Parker House, with a reminder that, onaverage, heroin and prescription drug abuse is claiming fourlives in the Commonwealth every day. “If we’re going to solve this crisis, we need to come

together,” said Healey. “We’re looking to make treatmentsmore available, intervene and educate people as early aspossible and get Narcan in the hands of more first responders.We need strong action and we need it now. I know it’s adifficult issue but there’s no greater collection of minds thanthose in this room.” Healey also addressed the challenge of treating diseases

such as hepatitis C, which is rising in conjunction withinjection drug abuse, when the approved drugs come with alist price upwards of $100,000.“There’s an infectious disease that’s rising, and there’s a

cure,” said Healey. “How do we get it into the hands of morepatients? Manufacturers and investors should rightly reaptheir financial reward, yet patients need access.” She has since sent a letter to Gilead urging for discounted

pricing on hepatitis C drugs Harvoni and Sovaldi for theCommonwealth’s public payers. Healey has made it clear sheintends to use her authority to shape the conversation andpublic policy, and hopes to work directly with the MassBiocommunity to do so.

“The work you’re doing is changing lives and building ahealthier, more promising future,” said Healey. “But thisbusiness is fragile. The reward is great, but so are the risksand setbacks. You need support for workforce developmentand the [Massachusetts] Life Sciences Center. My office ishere to work with you.”

During the panel discussion on innovation’s role in theopioid crisis, Marylou Sudders, Secretary of Health andHuman Services, shared her dream for a long-term solution—the development of a non-addictive painkiller, whichMassCONNECT graduate startup Blue Therapeutics iscurrently working on. Until that comes to fruition, companiesthroughout the Commonwealth, such as Alkermes, CollegiumPharmaceutical and kaléo are doing their part to createsafeguards to prevent and treatments to reverse overdoses, orto find ways to block opioid pleasure receptors.Michael Heffernan, Founder, President and CEO of

Collegium, spoke about his company’s technology platform,DETERx, which provides extended-release drug delivery,while safeguarding against common methods of abuse andtampering including crushing, chewing, heating and injecting.“Like seatbelts or airbags in a car, it’s not the solution, but

it’s part of the solution,” said Heffernan. “We all need tocollaborate to form a fully integrated approach to fightaddiction.” Closing the event, Blue Cross Blue Shield of Massachusetts

CEO Andrew Dreyfus and Sarah Emond, Chief OperatingOfficer of the Institute for Clinical and Economic Review,joined MassBio President & CEO Robert K. Coughlin todiscuss the pressures the healthcare system has faced andwhat to expect in the years ahead. “Personalized medicine is upon us and we’ll have to find a

way to fund it,” said Dreyfus. “There will have to be a certainlevel of caution and a focus on those treatments that we knowwill be effective.” Emond agreed her organization’s value framework will

have to be adjusted to measure one-time treatments such asgene therapy. Coughlin called for a Payer-Provider-IndustryTask Force to develop solutions and to bring those solutionsforward to policy makers.“We have a lot of work to do,” said Coughlin. “We want to

cure diseases with gene therapy and invent non-addictiveopioids. No one said it would be easy, but all of us,collectively, can solve these problems. We have to educateand engage every stakeholder to find a path forwardtogether.”

The 2016 Policy Leadership Breakfast addressed the growingopioid addiction and healthcare cost concerns

The work you’re doingis changing lives andbuilding a healthier,more promising future.

— Attorney General Maura Healey

“

Sarah Emond and Andrew Dreyfus joined MassBio President & CEO Robert K. Coughlin for a fireside chat.

WBUR HealthcareReporter MarthaBebinger moderateda panel.

Dr. Dennis Dimitri,president of theMassachusettsMedical Society,served as a panelmember.

Richard Pops and

Spencer

Williamson

shared their

companies’

perspectives on

the opioid crisis.

From left, Marylou Sudders, Dr. Dennis Dimitri, Michael Heffernan, Richard Pops and Spencer Williamson discussed the opioid crisis.

Attorney General MauraHealey opened the event.

MassBio President & CEO Robert K. Coughlin, center, joined representatives from Sunovion Pharmaceuticals at the Policy Breakfast.

TACKLING THE COMMONWEALTH'S BIGGEST CHALLENGES

”

PHOTOS: SEAN BROWNE

6 — MassBio NewsWINTER 2016

Why is MassBioEdundertaking a new Job

Trends initiative?

MassBio’s Impact 2020report identified achallenge for companiesas they grow and thrive inMassachusetts—data on

and access to the right workforce.MassBio’s job listings site reached an all-time high in 2015, averaging well over2,000 listings per day. Anecdotally,industry employers are reporting difficultyin hiring for a variety of positions. Suchreports raise concerns that ourtraditionally strong pipeline of highquality workers is under strain. It iscritical for us to better understand thenature of this challenge in growing andattracting the highly-talented workersneeded by the industry. That’s where theMassBioEd Foundation’s Job Trendsinitiative comes in.

What is the state of entry-level jobs for the life

sciences industry inMassachusetts?

In January, we publishedthe first Briefs of ourongoing Digest ofBiotechnology Job Trends.

The first series of Briefs focused onentry-level job trends which make up23% of all core job listings in theMassachusetts industry. The Briefsrevealed baseline skills that cut across alldegree levels, while also identifying thespecialized skill requirements of leadingjob categories at each degree level. Ofnote, while Associate’s degree level jobsincreased as a percentage of total coreindustry jobs since 2010, only about 300jobs, or 15% of all entry level job listings,are available to those who hold anAssociate’s degree or less.

Through this research, we discovered thatwe must uncover and articulateemployers’ expectations for entry levelcandidates and how academic programscan better align with industry needs.

What do you expect to seeout of this initiative in the

future? How cancompanies help?

Our goal is a completeunderstanding of industryjob trends and skillrequirements job seekers

need. Armed with this knowledge, we canguide industry in their development of joblistings and training programs, and engagehigher education so that degree programsalign with industry needs. It isunchartered territory and MassBioEdlooks forward to producing much neededguidance for academia and industry. Companies can help by designating

appropriate staff to serve on our SkillsAdvisory Group. It is this Group thatdrives the direction of our research.Ideally, members of the Skills AdvisoryGroup will possess a broad range ofknowledge about the skills required ofprospective job candidates. However, thisdoes not exclude experts who have deepknowledge in more narrow areas orhuman resources generalists with broadperspectives on candidates in the jobmarket. We hope to have a representationacross the sector, from novel drugdevelopment companies to contractresearch to biopharma manufacturing atboth small and large companies.

Save the date for MassBioEd’sConference on Job Trends on May 24,2016.

Visit www.MassBioEd.org or contactPeter.Abair@MassBio.org to learn moreand get involved.

Q

Q

Q

At the beginning of February, MassBiolaunched the new MassBio.org, designed togive the life sciences community new resources,new visibility opportunities and new ways toconnect. The sleek, responsive design not onlylooks better, it’s built better. Key features, likeMassBio’s member directory and events listing,are easier to access than ever before. Keeping the Patient at the Center MassBio’s new website will help MassBio

share stories from the people at the heart of thelife sciences industry: the patients. Read patientstories and learn more about MassBio membercompanies that are improving—and saving—lives around the world. Improved ResourcesIn the new Discover section of MassBio.org,

visitors can explore the wealth of resourcesMassBio offers including a crowdsourcedKnowledge Base for MassBio members,resources for entrepreneurs, funding

opportunities and MassBio’s policy work.Members can also access great savings throughthe MassBio Purchasing Consortium andMassBio Employee Rewards program. The new website also includes a new

MassBio Career Center with enhancedcapabilities for companies that are hiring andfor job-seekers themselves.Tell Your Story on MassBio.orgEvery MassBio member company has its

own page in the MassBio directory forincreased visibility within MassBio.org andthrough public searches. Members can sharetheir latest news, events and Knowledge Baseresources and write for our blog. All of thiscontent will connect to and enhance thecompany’s landing page.Discover the new MassBio.org today!

Contact communications@massbio.org withany questions, or if you have a patient story orblog post idea that you would like to share.

Q&AWITH

PETER ABAIR MEET THE NEW MASSBIO.ORG

EMPLOYEE REWARDS UPDATE

All employees of MassBio membercompanies can access discounts and dealson products, services, and experiences inthe Greater Boston area and beyond throughthe Employee Rewards program. Here arecompanies that have recently joined theprogram:Bar Boulud: Located at Mandarin

Oriental, Boston, Bar Boulud is a French-inspired bistro and wine bar frominternationally acclaimed chef DanielBoulud. MassBio members receive a 15%discount on any food item purchased duringdinner service from 5:30 p.m. until 10:30p.m. on Sunday through Wednesday.Beekman: Inspired by the Beekman 1802

Farm in Sharon Springs, NY, Beekman isone of the fastest growing lifestyle brandsin the country. MassBio members receive a20% discount on all products. Boston College Continuing Education

Program: BC Continuing Education offersa range of courses and a full certificateprogram that gives professionals an

understanding of the clinical researchlandscape. MassBio members receive 10%off Continuing Education clinical researchcourses. The Compass Tavern: Located in

Worcester, the Compass Tavern offers adelicious menu featuring New Englanddishes made from scratch. MassBiomembers receive 20% off of all appetizersand lunches Monday through Friday until4 p.m.The Lenox: Boasting a hallmark Back

Bay location, the Lenox is a family-owned,boutique hotel that expertly melds itscentury-old story with modern, luxuriousaccommodations. MassBio members cannow access discounted room rates.Wooberry: Worcester’s first yogurt shop

and winner of the Best of Worcester forfour years straight, Wooberry is offeringMassBio members 10% off of its gourmetice cream, frozen yogurt, and smoothies.Learn more and start saving at

www.MassBio.org.

UNLOCK THE SAVINGS

Executive Director of theMassBioEd Foundation

MassBio NewsWINTER 2016 — 7

disease, target new therapies, expedite clinicaltrials, and make DNA and RNA sequencingdata available to the entire ALS researchcommunity. “I think a big lesson is that advocacy starts

with the patient,” said Quinn. “If you’re notwilling to fight, who’s going to? I knew earlyon I would be proactive. You have to putyourself out there to make things happen.”Quinn’s lesson and the theme of building a

patient-centered biotech was consistentlyrepeated throughout the second annual summit,which brought industry leaders together withpatient advocates and other stakeholders toexamine ways in which life sciencescompanies can more fully incorporate thepatient voice into the work they do—not justapproaching regulatory applications or atcommercialization, but throughout the drugdevelopment cycle. Conversation circled around the importance

of the patient dialogue, particularly as medicineis becoming more personalized. “With rare diseases, the patients and their

families are really the ones who know thedisease better than anyone. Patient advocacy ispart of the fabric of what we do. I would cuteverything else before I would cut that. Havingsomeone remind us of our purpose is critical,”said Richard Peters, Head of Rare Disease atSanofi Genzyme. He joined bluebird bio ChiefFinancial Officer Jim DeTore to discuss theircompanies’ efforts to build, maintain andmeasure patient advocacy programs. “As rare patients, we realize we can’t sit

back and wait for an individual doctor orcompany to come up with a cure,” saidKathleen O’Sullivan-Fortin, board member ofALD Connect—an international, non-profitgroup of ALD patients, patient advocates andresearchers, who collaboratively educate,advocate, and conduct clinical research amongthose affected by X-linkedadrenoleukodystrophy (ALD).

O’Sullivan-Fortin, who has a 10-year-oldson with ALD, joined Michele Rhee ofbluebird bio to talk about collaborating toachieve impressive outcomes including ALDbeing added to the federal RecommendedUniform Screening Panel. The pair waspresented with MassBio’s CaringCollaborations Award. “It’s amazing what you can accomplish

when you don’t care who gets credit,” saidO’Sullivan-Fortin. “We’re trying to build a big,strong net to catch the cure. As a mom, itdoesn’t matter who finds a solution first. I hopeeveryone is wildly successful.”Another parent, Ron Suskind, a Pulitzer

Prize-winning journalist and best-sellingauthor, shared his experiences as caregiver tohis son, Owen, who was diagnosed withregressive autism at the age of three.Recognizing Owen’s affinity for Disneymovies, Suskind began channeling charactersto open up communication with his son. “Look through the eyes of the patient, it’s

amazing what you’ll learn,” said Suskind in anemotionally-charged keynote address.Capping off the day’s case studies, Victoria

Dibiaso of Sanofi and Dr. Steven Edelman ofTaking Control of Your Diabetes (TCOYD)talked about their work together to design aclinical trial that fits the individual needs ofeach patient.“We wanted a two-way conversation to

incorporate patient insights, reinvent researchmodels and make smarter, faster decisions,”said Dibiaso. “Based on feedback, we’ve beenable to simplify our prototype and reducedevelopment timelines. If we have the abilityto make someone’s life better, we have anobligation to do it fast and efficiently.”The day-long event, held at Google’s

Cambridge campus, also included paneldiscussions on how technology and patient dataare changing drug discovery and developmentand how companies can set clear expectationsfor the patient in the case of expanded access.

SUMMIT HELPS BUILD PATIENT-CENTERED BIOTECHS

Michele Rhee of bluebird bio andKathleen O’Sullivan-Fortin of ALDConnect were presented with theCaring Collaborations Award.

SUMMIT: from Page 1

LAABS, AURA BIOSCIENCES PROVIDE HOPE FOR FUTUREAura is in the process of

submitting an Investigational NewDrug (IND) application to the FDAand is advancing toward clinicaltesting. De los Pinos hopes to enroll24 patients in the trial and has 10hospitals throughout the U.S. linedup as sites. Dr. EvangelosGragoudas, Director of RetinaService at the Massachusetts Eyeand Ear Infirmary of HarvardMedical School, and Dr. CarolShields at the Wills Eye Hospital inPhiladelphia, are serving on theClinical Advisory Board and helpingto guide the clinical development.Dr. Ivana Kim, Co-Director of theOcular Melanoma Center at Mass.

Eye and Ear, will also participate asone of the investigators in the trial. “It has the potential to be

transformative,” said Kim. “Themain benefit would be eliminatingsome of the side effects of radiationwe’re seeing now, such as damage tothe retina or optic nerve, that lead tovision loss. I would also love to seeit developed into a diagnostic tool, totest borderline lesions that could betreated early. That would be a bigstep forward.”As a survivor, Laabs is

encouraged by the progress beingmade and is hopeful he, too, canplay a role. Following his experiencewith ocular melanoma, he foundedthe Rare Cancer ResearchFoundation (RCRF) to advance

efforts to develop new and bettertherapies to treat rare cancerpatients.Laabs, who built a successful

career in global renewable energydevelopment and solar productdistribution, sold his clean energyfinance company, Climate Bridge—which had become one of the largestplayers in the global carbonmarkets—to embark on this, adifferent kind of socially innovativeproject.“In remission, I was left with

questions of what to do next—reallywhere I could make the greatestpositive impact as possible over thecourse of my lifetime,” said Laabs.“I spent a lot of time thinking,‘should I work directly in ocular

melanoma?’ But all these cancershave common needs, and I realizedthat I don’t want anyone dying ofcancer, no matter what type. So Icame to the conclusion that the bestuse of my time and talent would beto connect with groups doing reallyimportant work and help them to dothat work more efficiently.” Working with more than 200

foundations and medical researchorganizations, RCRF administers theresearch infrastructure and enablesresearchers to focus on novel,incremental innovation. Thefoundation offers solutions forpatient registries, tissue samples, celllines, animal models, and genomesequencing, available to researchersthrough a single, virtual, integrated

platform. The hope is that it willradically accelerate the time-to-market for new, life-savingtherapies. At the same time,researchers can reallocate time,money and attention to novel drugdevelopment efforts and bringingnew therapies to market. “We’re a supporting player,

supplying the tools and the buildingblocks,” said Laabs. “The mostimportant thing is improving patientquality of life and longevity. To seebetter patient outcomes, it’snecessary to bring together differentgroups to solve problems that notone individual group could solve onits own. It’s that collaboration that Iwould want to see in my owntreatment.”

PATIENT: from Page 8

Journalist Ron Suskind spoke about hisrole as caregiver to his son.

Richard Peters of Sanofi Genzyme andJim DeTore of bluebird bio discussedthe business case for patient advocacy.

MassBio NewsWINTER 2016— 8

300 TECHNOLOGY SQUARE, CAMBRIDGE, MA 02139

PRESORT-STD U.S. POSTAGE

PAIDCAMBRIDGE, MA 02142

PERMIT NO. 981

Laabs' foundation and Aura Biosciences advance efforts todevelop new and better therapies to treat rare cancer patients

A PATIENT’S DESIRE TO HELP

BYMEAGHAN CASEY

Four years ago, Memphis native MarkLaabs’ future was bright. He was 28,working in China as chief operating officerof a company dedicated to fighting climatechange, and living out his calling to followhis “personal North Star.” A single moment completely shifted

where that North Star was leading him. While in a meeting in Beijing, Laabs lost

vision in his right eye. Nearly 48 hours andfive consultations later, he was sitting withthe dean of ophthalmology at a majormedical center, being told it was ocularmelanoma. A rare cancer, ocular melanoma is

diagnosed in about 2,500 adults every yearin the U.S. Although produced from thesame cells, called melanocytes, ocularmelanoma is different from skin cancer. Itdevelops in the uvea, or uveal tract, of theeye. In approximately half of thosediagnosed, the tumor can aggressivelygrow and spread to other parts of the body,becoming fatal.As Laabs quickly discovered, there have

been no targeted therapies available andthe cancer is usually treated with aninvasive radioactive plaque placed againstthe exterior of the eye near the tumor.Known as brachytherapy or plaquetherapy, this treatment can require multiplesurgeries and can lead to cataracts,retinopathy and loss of vision. Thealternative to radiation is surgery toremove the eye. “I was essentially a biohazard for eight

days while the radioactive plaque was onmy eye and at the end of that process, thetumor was dead,” said Laabs, who hadflown back to the U.S. for treatment inPhiladelphia. The treatment left him inremission, but he was unable to regainmost of the vision in his right eye. Aura Biosciences, a Cambridge-based

biotech company developing highly tumor-targeted breakthrough therapies for rarecancers, is hoping to improve the optionsfor future ocular melanoma patients. Thenew class of therapies would potentiallytarget and destroy cancer cells selectively,while leaving surrounding tissueunharmed—an approach Aura calls

molecular surgery. It would effectivelytransform the treatment of ocularmelanoma into a routine outpatientprocedure. “By enabling physicians to treat cancer

more selectively, effectively and safelythan they can do today, we aim toeliminate the need for risky proceduresthat carry significant morbidity andoften do little to improve a patient’soverall survival,” said Aura’sFounder and CEO Elisabet de losPinos. “When you’re focusing on arare disease and thinking about thelimited and highly invasive alternativesout there, it creates an incredible senseof purpose. Everything is aligning at themoment and, if successful, the endproduct will be extremely meaningful forpatients.”In May, Aura was granted Orphan

Disease Designation by the FDA for itslead product candidate, AU-011. Thecompany’s pre-clinical research,‘Evaluating the in vivo efficacy of a first-in-class drug for the treatment of primaryuveal melanoma,’ was delivered thatsame month by McGill University HealthCentre researchers at the 2015Association for Research in Vision andOphthalmology (ARVO) AnnualMeeting. “Aura’s novel approach has the

potential to dramatically improve theoutcomes, and hope, for patients with

ocular melanoma,” said Grant Allen, Co-Founder and Chairman of the OcularMelanoma Foundation. “The OrphanDrug Designation for AU-011 is a hugestep forward, enabling Aura to potentiallybring this drug to patients in an expeditedmanner.” AU-011 consists of a viral-like

particle drug conjugate that bindsselectively to cancer cells in the eyeand upon activation with anophthalmic laser, the smallmolecule selectively destroysthe membrane of cancer cells,killing them without damagingthe adjacent retina.

Elisabet de los Pinos is CEO ofAura Biosciences.

PATIENT PROFILE

See PATIENT Page 7

Ocular melanoma survivor MarkLaabs founded the Rare

Cancer Research Foundation.