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CF Organizations

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CF Organizations
& Support

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FAQs

FAQs

How can I get involved in genetic medicine research?

If you want to get involved in genetic medicine research, here are some ways to get started:

  • Explore Clinical Trials
    • ClinicalTrials.gov: This is a comprehensive global database of clinical studies where you can search for trials specifically by filtering for “cystic fibrosis” and other relevant keywords.
    • Cystic Fibrosis Foundation’s Clinical Trial Finder: This platform allows you to search for clinical trials based on their location and the type of research you’re interested in. It often includes studies on genetic therapies, gene editing, and mRNA treatments.
  • Consult with Your CF Care Team
    • Speak with your physician or care team. They can provide personalized guidance based on your medical history and trials that might be a good fit.
    • CF clinics are often informed about upcoming or ongoing research, and they can help connect patients with appropriate clinical trials.
  • Join Patient Registries
    • Emily’s Entourage’s Patient Database: Emily’s Entourage maintains a global patient database of individuals with CF who do not benefit from existing CFTR modulator therapies due to ineligible genetic mutations, side effects from CFTR modulators, or suboptimal response to CFTR modulators. The database, called Emily’s Entourage CF Clinical Trial Connect (CTC), allows these individuals to contribute their data and become informed of relevant clinical research and trials. It also accelerates the development of lifesaving therapies by offering faster, more targeted clinical trial recruitment.
  • Connect with Research Institutions or Companies
    • Reach out to biotech companies focused on genetic therapies for CF. For instance, companies working on mRNA treatments may be recruiting for early-stage trials. ReCode Therapeutics and other biopharma companies developing genetic therapies for CF may offer avenues for participation. Contact [email protected] to learn more about a Phase 1b study evaluating RCT2100, an investigational inhaled mRNA therapy for people with CF who have mutations in the CFTR gene that do not respond to CFTR modulators.
  • Advocacy Groups
    • Cystic Fibrosis Research Community: There are advocacy groups and communities that engage in research partnerships and collaborations. These groups often provide information about the latest treatments and clinical trials and offer resources on how to get involved.

What genetic therapies are available for those who can’t take CFTR modulators?

For people with CF who cannot take CFTR modulators, either because their mutations don’t respond to these therapies or they experience adverse effects, there are emerging genetic therapies under development that may provide alternative options. These therapies aim to address the root cause of CF.

Many of these genetic therapies are still in clinical or preclinical stages, so they are not yet approved or widely available as treatments. However, they offer hope for patients who cannot benefit from CFTR modulators, particularly for those with rare mutations like nonsense mutations.

  • mRNA-Based Therapies: One of the most promising therapies under development is messenger RNA (mRNA) therapy, which works by delivering synthetic messenger mRNA to the body’s cells, instructing them to produce the CFTR protein, which is faulty or missing in people with CF. ReCode Therapeutics is developing RCT2100, an inhaled mRNA therapy designed to deliver mRNA directly to the lungs to help cells create a functional CFTR protein, targeting the root cause of the disease rather than just managing its symptoms.
  • Gene Editing Therapies: CRISPR/Cas9 Gene Editing: This technology allows scientists to “cut and paste” sections of DNA to correct the underlying mutation that causes CF. Several biotech companies and research institutions are exploring CRISPR-based gene editing to directly repair or replace faulty CFTR genes in the patient’s cells.
  • Gene Therapy (Viral Vectors): Gene therapy involves using viral vectors, such as Adeno-associated Virus (AAV) vectors to deliver a functional copy of the CFTR gene to a patient’s lung cells. This therapy aims to provide a lasting solution by enabling cells to produce functional CFTR protein.
  • Stem Cell Therapy: Researchers are also exploring the use of stem cell therapies to treat CF. These therapies would involve replacing defective cells in the lungs with genetically corrected stem cells that can differentiate into healthy, functional lung tissue capable of producing CFTR protein.
  • Oligonucleotide-Based Therapies: Antisense Oligonucleotides (ASOs) are small synthetic strands of nucleic acids that can bind to RNA and modulate its function. For CF, ASOs may be designed to correct splicing mutations or other types of genetic defects that cause CFTR protein dysfunction.

Anyone interested in genetic therapies for CF should consider participating in clinical trials, as these are investigational therapies and not yet FDA-approved. You can search trial databases (e.g., ClinicalTrials.gov) or consult with your healthcare provider for ongoing studies in your area. Connecting with advocacy organizations like Emily’s Entourage and the Cystic Fibrosis Foundation can also help you stay informed about trial opportunities and advancements.

Are there any known side effects of genetic therapies?

Genetic therapies for CF, while promising, can have potential side effects. Common risks include immune reactions, which can lead to inflammation or flu-like symptoms. Long-term side effects are still being studied, as these treatments are relatively new and their lasting impact is not yet fully understood.

What are the most important concepts for people with CF to understand when discussing genetic therapies?

  • Treatments aim to address the root cause of the disease by correcting or compensating for the defective CFTR gene rather than just managing symptoms. Genetic therapies, such as mRNA treatments or gene editing, are in clinical trials but hold promise for patients, especially those who don’t benefit from current CFTR modulators.
  • It’s important to stay informed about new research and talk with your doctor or care team about any clinical trials or investigational therapies that might be appropriate for your specific CF mutation and health condition.

What mutations are eligible for the currently enrolling genetic therapy trials?

To find out which CF mutations are eligible for currently enrolling genetic therapy trials, follow these steps:

  • Explore Clinical Trials
    • ClinicalTrials.gov: This is a comprehensive global database of clinical studies where you can search for trials specifically by filtering for “cystic fibrosis” and other relevant keywords.
    • Cystic Fibrosis Foundation’s Clinical Trial Finder: This platform allows you to search for clinical trials based on their location and the type of research you’re interested in. It often includes studies on genetic therapies, gene editing, and mRNA treatments.
  • Consult with Your CF Care Team
    • Speak with your physician or care team. They can provide personalized guidance based on your medical history and trials that might be a good fit.
    • CF clinics are often informed about upcoming or ongoing research, and they can help connect patients with appropriate clinical trials.
  • Join Patient Registries
    • Emily’s Entourage’s Patient Registry: Emily’s Entourage maintains a global patient registry of CF individuals who are genetically ineligible for CFTR modulators or do not benefit from existing modulator therapies. The registry allows these individuals to contribute their data and become informed of relevant clinical research and trials. It also accelerates the development of lifesaving therapies by offering faster, more targeted clinical trial recruitment.
  • Connect with Research Institutions or Companies
    • Reach out to biotech companies focused on genetic therapies for CF. For instance, companies working on mRNA treatments may be recruiting for early-stage trials. ReCode Therapeutics and other biopharma companies developing genetic therapies for CF may offer avenues for participation. Contact [email protected] to learn more about a Phase 1b study evaluating RCT2100, an investigational inhaled mRNA therapy for people with CF who have mutations in the CFTR gene that do not respond to CFTR modulators.
  • Advocacy Groups
    • Cystic Fibrosis Research Community: There are advocacy groups and communities that engage in research partnerships and collaborations. These groups often provide information about the latest treatments and clinical trials and offer resources on how to get involved.

Would some participants receive a placebo in a genetic therapy trial?

  • In some cases, participants in a CF clinical trial may receive a placebo. This is common in trials to assess the efficacy and safety of the treatment being studied.
  • In a placebo-controlled trial, participants are typically divided into two groups: one receiving the investigational therapy and the other receiving a placebo, which has no therapeutic effect. This allows researchers to compare outcomes between the two groups to determine whether the genetic therapy is effective in improving CF symptoms or addressing the underlying cause of the disease.

It is important to note:

  • Not all trials use placebos—some use open-label designs where all participants receive the treatment, especially in early-phase or compassionate-use studies.
  • Placebo-controlled trials are designed with safety in mind, and participants receive close monitoring.
  • Before joining any trial, it’s important to discuss the design with your doctor or care team, including whether placebos are involved, so you can make an informed decision.

When would genetic therapies be available for compassionate use?

  • Investigational therapies may become available for compassionate use when a patient with a serious or life-threatening condition, such as cystic fibrosis, has no other treatment options and cannot participate in a clinical trial.
  • Compassionate use, also known as expanded access, allows eligible patients to access experimental treatments that are still in development. To qualify, a doctor must request the therapy on behalf of the patient, and the company developing the drug and regulatory authorities, like the FDA, must approve the request.
  • While compassionate use offers hope for those in need, it’s typically limited to exceptional cases where no alternatives exist.

How effective would mRNA or gene therapy be compared to Trikafta?

  • The effectiveness of mRNA or gene therapy for cystic fibrosis compared to Trikafta is still unknown, as there is no head-to-head data comparing them.
  • Trikafta, a highly effective CFTR modulator, has significantly improved outcomes for many patients with common CF mutations. However, mRNA and gene therapies aim to address the root cause of CF by enabling the production of functional CFTR protein in a broader range of mutations, potentially offering a more comprehensive solution, particularly for patients who cannot benefit from Trikafta. The full potential of these therapies will only become clear as clinical trials progress.

Will clinical trials for genetic therapies accept participants with lower lung function?

  • Whether clinical trials for genetic therapies accept participants with lower lung function depends on the specific trial’s eligibility criteria. Some trials may set a minimum lung function threshold, typically measured by FEV1 (forced expiratory volume), to ensure patient safety and the trial’s ability to accurately measure outcomes.
  • However, some studies, especially those aimed at more severe cases of CF, may accept participants with lower lung function to assess the therapy’s effectiveness in a broader range of patients. It’s important to review each trial’s requirements and consult with your healthcare provider to determine if you’re eligible.

How will genetic therapies impact people who are post-transplant?

  • Genetic therapies, such as mRNA or gene editing, are primarily designed to correct the underlying cause of CF by enabling cells to produce functional CFTR protein. For people with CF who are post-transplant, the impact of genetic therapies may be limited, as lung transplants replace the damaged tissue with donor lungs that do not have the CF mutation.
  • However, these therapies could potentially benefit other organs affected by CF, such as the pancreas or liver, or help prevent complications in other parts of the body. It’s essential for post-transplant patients to consult their care team, as genetic therapies are still experimental, and their use in this population may require further investigation.

Dean J. Mitchell

Chairman, Board of Directors

Mr. Mitchell currently serves as chairman of the Board of Directors of Praxis Precision Medicines, Inc., an appointment he has held since 2020, and as a board director for Theravance Biopharma, Inc. (since 2014) and Precigen (since 2009). Mr. Mitchell previously served as board chairman of Kinnate Biopharma, Inc. until its sale in 2024 and as board director for ImmunoGen, Inc. until its sale in 2024. He served as executive board chairman for Covis Pharma Holdings until its sale in 2020 and board chairman of PaxVax Corporation until its sale in 2018.

Mr. Mitchell previously held the position of president and chief executive officer at Lux Biosciences, Inc., a biotechnology company focusing on the treatment of ophthalmic diseases, at Alpharma, Inc., a publicly traded specialty pharmaceutical company until its acquisition by King Pharmaceuticals, Inc., and at Guilford Pharmaceuticals, Inc., a publicly traded pharmaceutical company focused on oncology and acute care, until its acquisition by MGI Pharma Inc. He previously held executive roles at Bristol Myers Squibb and GlaxoSmithKline. Mr. Mitchell earned a B.A. from City University London and a B.Sc. in biology from Coventry University.

Trisha Millican
ReCode Therapeutics

Ms. Millican has over 20 years of experience in the life science industry including debt and equity financings, mergers and acquisitions, company formation, licensing transactions, co-development/promotional arrangements, and commercial product launches. Currently, Ms. Millican is a strategic advisor to various life science companies and serves on the board of Life Science Cares, San Diego. Most recently Ms. Millican served as the chief financial officer of Metacrine, Inc. where she led finance and investor relations and raised over $250 million in equity and debt financings including an initial public offering. Prior to Metacrine, Ms. Millican served as the senior vice president of finance at Seragon, a private biotechnology company focused on developing selective estrogen receptor degraders (SERDs) targeting hormone-dependent cancers, which was acquired by Genentech in August 2014.

From July 2012 to August 2013, she served as vice president of finance at Aragon, a discovery-stage small molecule company focused on therapeutics for the treatment of hormone-resistant cancers, which was acquired by Johnson & Johnson in August 2013. From September 2009 to July 2012, Ms. Millican served in various senior financial management roles at Zogenix, Inc., a pharmaceutical company developing and commercializing innovative central nervous system therapies for people living with serious and life-threatening rare central nervous system disorders and medical conditions.

At the beginning of her professional career, Ms. Millican spent five years with the public accounting firm, Deloitte LLP. She holds a bachelor’s degree in Accountancy from the University of San Diego and is a certified public accountant in the state of California.

Justin Hanes, Ph.D.

Scientific Advisory Board Member
Dr. Hanes is the Lewis J. Ort professor of ophthalmology at the Wilmer Eye Institute at Johns Hopkins University, with secondary appointments in chemical & biomolecular engineering, environmental health sciences, neurosurgery and oncology. A globally recognized leader of research at the interface of nanotechnology and medicine, Dr. Hanes focuses his research on the delivery of genetic medicines, characterization of mucus and design, and testing of nanoparticles that can penetrate mucus barriers. His lab recently discovered methods to make drug- and gene-loaded particles that efficiently penetrate mucus barriers, which may allow for more effective therapies for eye diseases.

Elliott Sigal, M.D., Ph.D.

Scientific Advisor

Dr. Sigal currently serves as co-chair of the scientific advisory board for Amgen, Inc. and as a senior advisor to New Enterprise Associates, Inc. He is a member of the Board of Directors for Adaptimmune Therapeutics, Vir Biotechnology, Surface Oncology, Tessera Therapeutics and Affinia Therapeutics. He is a former executive vice president of Bristol Myers Squibb where he also served as chief scientific officer and president of research and development from 2004 until 2013. Under his leadership, BMS brought 14 new medicines to market and became a leading immuno-oncology company. In 2012, SCRIP Intelligence named Dr. Sigal the best R&D chief in the pharmaceutical industry.

Eric Olson, Ph.D.

Scientific Advisory Board Member
Dr. Olson is the founding chair of the department of molecular biology at the University of Texas Southwestern Medical Center. He also directs the Hamon Center for Regenerative Science and Medicine and the Wellstone Center for Muscular Dystrophy Research. He holds the Robert A. Welch Distinguished Chair, the Pogue Distinguished Chair and the Annie and Willie Nelson Professorship. Dr. Olson and his team discovered many of the genes that control heart and muscle development and disease. His most recent work has provided a new strategy for correction of Duchenne muscular dystrophy-causing mutations using CRISPR gene editing.

Carrolee Barlow, M.D., Ph.D.

Scientific Advisory Board Member

Dr. Barlow was most recently the chief medical officer of ESCAPE Bio, Inc. Prior to joining ESCAPE Bio, she served as chief executive officer of the Parkinson’s Institute and Clinical Center. Dr. Barlow was also formerly acting chief medical officer at Amicus Therapeutics and was a co-founder, chief scientific officer and chief medical officer of BrainCells, Inc. She also served as director of molecular neuroscience at Merck Research Laboratories and was a professor at the Salk Institute. Dr. Barlow is an expert in neuroscience and neurodegeneration, the treatment of rare and neurological diseases and clinical development for new therapies.

Rafael Amado, M.D.

Scientific Advisory Board Member

Dr. Amado serves as president, head of global oncology research and development at Zai Labs. He most recently served as executive vice president of research and development at Allogene Therapeutics, Inc., and previously, president of research and development at Adaptimmune Therapeutics PLC, after serving as its chief medical officer. He has held several roles of increasing responsibility at GSK, most recently as senior vice president and head of oncology research and development. Dr. Amado has built a career leading the development of a number of breakthrough therapies in hematology and oncology, with a focus on immunotherapies and CAR-T drug discovery and development.

Daniel J. Siegwart, Ph.D.

Chair of the Scientific Advisory Board
Dr. Siegwart is the W. Ray Wallace Distinguished Chair in Molecular Oncology Research and associate professor in the Department of Biochemistry at the University of Texas Southwestern Medical Center and is a co-founder of ReCode Therapeutics. Dr. Siegwart has been a pioneer in the design of delivery technologies for genetic medicines. ReCode’s SORT LNP platform, developed by Dr. Siegwart and his lab, was described by Nature as one of the “Seven Technologies to Watch in 2022.” In 2016, his lab reported the first non-viral system for in vivo CRISPR/Cas gene editing. Dr. Siegwart and his research team are focused on solving challenges in disease therapy and diagnosis, with a focus on the development of new materials that can deliver genetic medicines to treat cancer and genetic diseases.

Alan Colowick, M.D., M.P.H.

Matrix Capital Management Company

Alan Colowick is Senior Managing Director on the Technology-Life Sciences team at Matrix Capital Management Company, LP and its affiliate, AyurMaya Capital Management Company, LP. He currently also serves on the BODs of Acelyrin Inc, Alumis Therapeutics, Solve Therapeutics, Personalis, Inc., AC Immune SA, Harpoon Therapeutics, Inc., InCarda Therapeutics, Inc., XyloCor Therapeutics, and Teon Therapeutics, Inc. Prior to joining Matrix, Dr. Colowick was a Partner at Sofinnova Investments, where he was Chairman of the BOD of two portfolio companies and helped lead their successful acquisitions (Velos Bio acquired by Merck for $2.75B and Principia Inc acquired by Sanofi for $3.7B).

Prior to that, Dr. Colowick had an 18 year career in biotech including significant operating and executive roles at Amgen; Gloucester Pharmaceuticals, where he was CEO and led its acquisition by Celgene: and Celgene, where he was President of EMEA. Dr. Colowick received his MD from Stanford University and completed fellowship training in Hematology and Oncology at the Dana Farber Cancer Institute. He also holds an MPH from Harvard University.

Rakhshita Dhar, M.S.

Leaps by Bayer

Rakhshita Dhar is Senior Director of Venture Investments Health at Leaps by Bayer. She joined the team in 2021 and is responsible for search & evaluation and healthcare focused investment for Leaps. She currently sits on the board of Deka Biosciences, Gro Bio, AffiniT therapeutics, Bloom science and Edifice health.

Prior to joining Leaps, Rakhshita was Director of Business Development at Roche Pharmaceuticals where she led collaborations with Dyno Therapeutics and Rheos Biosciences. She also spent a few years at MassBio developing an accelerator program for life-science start-ups.

She got her Undergraduate degree in Biochemistry from Mumbai University and her Masters in Molecular Biology from Georgetown University.

Shehnaaz Suliman, M.D., M.B.A., M.Phil.

ReCode Therapeutics

Dr. Suliman is a physician, drug developer and dealmaker with over 25 years of experience building and transforming small and large biopharmaceutical companies.

Prior to becoming CEO of ReCode Therapeutics in January 2022, she served as president and chief operating officer of Alector, a leading immuno-neurology and immuno-oncology company where she played a critical role growing, scaling, raising capital and advancing a late-stage development pipeline.

Prior to Alector, she served as senior vice president, corporate development and strategy at Theravance Biopharma from 2017 to 2019, where she helped deliver a $1B profit sharing partnership with Janssen for the company’s lead JAK inhibitor program.

Prior to Theravance, Dr. Suliman was vice president and global head, immunology, infectious diseases and specialty care at Roche from 2015 to 2017. Dr. Suliman worked for Genentech as a group leader and project team leader in the R&D Portfolio Management and Operations Group from 2010 to 2015, where she oversaw an early development portfolio of more than 30 programs across multiple therapeutic areas. She held various management roles of increasing responsibility at Gilead Sciences, Inc. between 2005 and 2010 and played a significant role in forward-integrating Gilead into new therapeutic areas through M&A. Prior to Gilead, Dr. Suliman was an investment banker with Lehman Brothers and Petkevich & Partners, advising numerous public and private companies on buy- and sell-side transactions.

She was named one of the 2017 Fiercest Women in Life Sciences and as one of the National Diversity Council’s Power 50 in 2021 (Most Influential Women in Business). Dr. Suliman serves as an independent director on the Board of Directors of Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) and 10x Genomics (NASDAQ: TXG). Dr. Suliman received her M.D. (MB, ChB) from the University of Cape Town Medical School, South Africa, and holds an MBA, with distinction, and M.Phil. degrees from Oxford University, where she was a Rhodes Scholar.

Peter Thompson, M.D.

OrbiMed Advisors
Dr. Thompson is a partner on the private equity team at OrbiMed Advisors LLC. With over 25 years of industry experience, he has held executive positions at Becton-Dickinson and Chiron, co-founded and was CEO of Trubion Pharmaceuticals, co-founded Corvus Pharmaceuticals, Cleave Biosciences, Silverback Therapeutics and serves as a director on several company boards. Dr. Thompson is an Ernst & Young Entrepreneur of the Year awardee, an Affiliate Professor of Neurosurgery at the University of Washington, an inventor on numerous patents and a board-certified internist and oncologist. He was on staff at the National Cancer Institute following his internal medicine training at Yale University.

Oleg Nodelman

EcoR1 Capital

Mr. Nodelman is the Founder and Portfolio Manager of EcoR1 Capital LLC, a biotech-focused investment advisory firm established in 2013, which invests in companies at all stages of research and development.

With twenty years of experience in biotech investing, Mr. Nodelman has expertise in all aspects of investment management and deep roots in the biotech and scientific communities. Before founding EcoR1, Mr. Nodelman was a portfolio manager at BVF Partners, one of the first hedge funds dedicated to the biotechnology sector. He currently serves as a Board Member for three publicly traded companies: Prothena (NASDAQ: PRTA), a clinical-stage neuroscience company, AnaptysBio (NASDAQ: ANAB), a clinical-stage biotechnology company focused on emerging immune control mechanisms applicable to inflammation and immuno-oncology indications, and Nuvation Bio (NYSE: NUVB), a clinical-stage biopharmaceutical company tackling unmet needs in oncology.

Mr. Nodelman has a Bachelor of Science in Foreign Service with a concentration in Science and Technology from Georgetown University.

Helen Kim, M.B.A.

Vida Ventures

Ms. Kim is a Managing Director of Vida Ventures, a life sciences focused venture capital firm.

Most recently, Ms. Kim was a Partner at The Column Group. Previously, she served as Executive Vice President of Business Development at Kite Pharma, Inc., where she led business and corporate development initiatives including selling Kite Pharma to Gilead in 2017. Previously, Ms. Kim served as Strategic Advisor of NGM Biopharmaceuticals, Inc. from January 2012 through November 2014; served as Chief Business Officer at NGM Biopharmaceuticals, Inc. from August 2009 to January 2012; served as Chief Executive Officer and President of Kosan Biosciences where she restructured and successfully assisted in selling the company to Bristol-Myers Squibb in 2008.

She received a B.S. in chemical engineering from Northwestern University and an M.B.A. from the University of Chicago. Ms. Kim is currently a director for Assembly Biosciences, Applied Molecular Transport, Exicure, Inc., A2 Biotherapeutics, Neogene and PACT.

Ed Hurwitz, J.D., M.B.A.

MPM Capital

Mr. Hurwitz, an accomplished biotech executive and investor, is a Managing Director at MPM Capital and serves as Chairman of MPM portfolio companies BioIntervene and Rekindle Therapeutics, and on the board of Dyne Therapeutics.

Prior to MPM, he was founder and Managing Director of Precision BioVentures, where he founded and seeded start-up biotechnology companies, including Viewpoint Therapeutics. As a director at Alta Partners, he led 12 successful investments including Applied Genetic Technologies Corporation, Avid Radiopharmaceuticals (acquired by Lilly), Calistoga Pharmaceuticals (acquired by Gilead), Cara Therapeutics, FoldRx Pharmaceuticals (acquired by Pfizer), MacroGenics and Taligen Therapeutics (acquired by Alexion).

Over the course of his career, he has been a Senior Vice President and CFO of Affymetrix, a microarray pioneer, and a biotech research analyst for Robertson Stephens & Company and Smith Barney Shearson. He also practiced law at Cooley Godward LLP.

He earned his J.D. and M.B.A. degrees from the University of California, Berkeley’s Boalt School of Law and Haas School of Business, and his B.A. in molecular biology from Cornell University.

Rana Al-Hallaq, Ph.D.

Pfizer Ventures

Dr. Al-Hallaq is Executive Director and Partner at Pfizer Ventures.

Dr. Al-Hallaq leverages her preclinical, clinical, and business development experience to assess, invest in, and manage equity investments for Pfizer Ventures. She currently has responsibility for Pfizer’s investments in Mitokinin, Inc. (San Francisco, CA), Autifony Therapeutics Ltd. (Stevenage, UK), Blade Therapeutics (South San Francisco, CA), RefleXion Medical (Hayward, CA), Montis (Leuven, Belgium), Biograph55 (San Francisco, CA), Vivet Therapeutics (Paris, France), and Arkuda (Cambridge, MA) among others.

Prior to her current role, Dr. Al-Hallaq was a Transactionalist in Worldwide Business Development at Pfizer where she was responsible for negotiating and transacting licenses, acquisitions, and partnerships across therapeutic areas. Dr. Al-Hallaq joined Pfizer in 2015 as an Early Candidate Clinical Lead where she advised early clinical programs in CNS to ensure alignment with business strategies. Prior to joining Pfizer, she held roles at Allergan (formerly Actavis, formerly Forest Laboratories), first in Clinical Development Psychiatry as scientific and operational lead on Phase 2 and Phase 3 studies investigating novel treatments for Major Depressive Disorder and schizophrenia, and later in Business Development where she assessed and executed on a number of acquisitions and licenses across therapeutic areas. She began her training as a research fellow at the National Institutes of Health (NIH).

Dr. Al-Hallaq graduated Summa Cum Laude and Phi Beta Kappa with a B.A. in Biology from Hamilton College and holds a Ph.D. in Neuroscience from Georgetown University Medical Center.