Mary Beth DeLena

The Friedreich's Ataxia Research Alliance provides patient registry services. The private company is based in Downingtown, PA.

Imbria Pharmaceuticals, Inc. is a clinical stage biopharmaceutical company developing novel therapies that restore cellular energy deficits. It focuses on restoring or improving the cell?s ability to produce energy where energy imbalances are at the root of disease, including cardiovascular diseases and inborn errors of metabolism. The company was founded by Andrew Levin and David-Alexandre C. Gros in 2018 and is headquartered in Boston, MA.

RA Capital Management LP (Private Equity) (RA Capital) is a private equity & venture capital subsidiary of RA Capital Management LP founded by Peter Kolchinsky in 2004. The firm is headquartered in Boston, MA.

Nayan Therapeutics, Inc. operates as a biotechnology company. It focuses on small molecule development for oscular disorders involving rod degeneration. The company is headquartered in Medfield, MA.

Anadys Pharmaceuticals, Inc. develops and manufactures novel small molecule medicines. It engages in developing novel medicines in the areas of hepatitis C and oncology. The firm develops ANA598, a direct-acting antiviral for the treatment of hepatitis C. The company was founded by Stelios B. Papadopoulos and Kleanthis G. Xanthopoulos in 1992 and is headquartered in Little Falls, NJ.

New England Venture Capital Association, Inc. is a non-profit organization which operates as a catalyst, and advocate, and a resource for the innovation economy. The company is headquartered in Cambridge, MA.

Ventus Therapeutics, Inc. operates as a biopharmaceutical company. It focuses in developing novel small molecule medicines that target the innate immune system to treat autoimmune diseases, inflammatory diseases and cancer. The company was founded by Hao Wu, Richard Flavell, Judy Lieberman, Thomas Tuschl, and Feng Shao and is headquartered in Montreal, Canada.

Sionna Therapeutics, Inc. is a clinical-stage life sciences company located in Natick, MA. The company is dedicated to developing highly effective and differentiated treatments for cystic fibrosis (CF) by normalizing the function of CFTR, the key protein associated with disease progression in CF. Sionna is advancing a pipeline of small molecules engineered to correct the protein defects caused by ?F508, the most common mutation that affects the CFTR protein. The company has a first-in-class portfolio of programs directly targeting correction of ?F508-CFTR, the key and unique mechanism to enable full restoration of ?F508-CFTR function, and complementary programs targeting ICL4 and more. The CEO of the company is Michael Cloonan.

Bright Peak Therapeutics AG engages in the development of engineered cytokines for immuno-oncology indications. The company is headquartered in Basel, Switzerland.

Santi Therapeutics, Inc. provides pharmaceutical services. The company is headquartered in Boston, MA.

Be Biopharma, Inc. develops engineered B cells as a new category of cellular medicines. The company was founded by David Steinberg, Aleks Radovic-Moreno and Lea Hachigian and is headquartered in Cambridge, MA.

Caraway Therapeutics, Inc. provides heathcare services. The company was founded by Timothy Harris, Wade Harper ,Ivan Dikic ,Tony Hyman and is headquartered in Cambridge, MA.

Ventus Therapeutics U.S., Inc. operates as a biotechnology company. The company is headquartered in Natick, MA.

KalVista Pharmaceuticals Ltd. develops novel plasma kallikrein inhibitors. It also engages in the development of serine protease inhibitors and novel plasma kallikrein inhibitors represent a, approach to the treatment of DME, a leading cause of adult visual loss, plasma kallikrein inhibitors target a distinct molecular pathway and as such have the potential to offer those patients an effective treatment option. The firm has a clinical and advanced pre-clinical product pipeline targeting both intravitreal injection and oral administration, and its plasma kallikrein inhibitors can be applied to additional indications in ophthalmology. The company was founded by Lloyd Paul Aiello, Andrew Crockett, Robert Haigh and Edward P. Feener in 2011 and is headquartered in Porton Down, the United Kingdom.

SV Health Investors LLC is a Venture Capital firm, a subsidiary of SV Health Investors LLP founded in 2006. SV Health Investors LLC is headquartered in Boston.

Atlas Venture Advisors Inc (Atlas Venture Advisors) is a venture capital firm founded in 1980 by Christopher Julian Spray. The firm is headquartered in Cambridge, Massachusetts.

Part of Bright Peak Therapeutics AG, Bright Peak Therapeutics, Inc. develops immunotherapies for the treatment of cancer and autoimmune diseases. The company is based in San Diego, CA. The company was founded by Vijaya R. Pattabiraman, Jeffrey Bode. The CEO is Fredrik Wiklund.

MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research. The Association also provides comprehensive health care and support services, advocacy and education. MDA combats neuromuscular diseases through programs of worldwide research, comprehensive medical and support services, and far-reaching professional and public health education. With national headquarters in Tucson, MDA has more than 200 offices across the country, sponsors some 200 hospital-affiliated clinics and supports more than 330 research projects around the world. MDA supports more research on neuromuscular diseases than any other private-sector organization in the world. MDA scientists are in the forefront of gene therapy research and have uncovered the genetic defects responsible for several forms of muscular dystrophy, Charcot-Marie-Tooth disease, a form of amyotrophic lateral sclerosis (ALS, or Lou Gehrig's disease), childhood spinal muscular atrophy and several other neuromuscular conditions. The Association's comprehensive services program includes diagnostic and follow-up medical consultations, flu shots, support groups, MDA summer camps for youngsters, a medical equipment program, assistance with equipment repairs and resource referral. Through its national advocacy program, MDA works to make life better for people with muscular dystrophy and related muscle diseases by providing representation in matters of public policy and research advancement, nationally and internationally; and facilitating active involvement in these areas by the people it serves. MDA was created in 1950 by a group of adults with muscular dystrophy, parents of children with muscular dystrophy and a physician-scientist studying the disorder. Since its earliest days, it has been energized by its number-one volunteer and national chairman, entertainer Jerry Lewis.

Critical Path Institute is a nonprofit organization established in 2005 as a public and private partnership with the FDA's Critical Path Initiative program. The non-profit company is based in Tucson, AZ, and operates from Ireland with additional staff in multiple other locations. The company's mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. The company orchestrates the development of drug development tools through an innovative, collaborative approach to the sharing of data and expertise, and accelerate the pace and reduce the costs of medical product development by building consensus among the participating institutions. The company was founded in 2005 by Raymond L. Woosley, Glenn C. Rice. Daniel M. Jorgensen has been the CEO of the company since 2022.

Founded in 1898, Northeastern University is a private research university located in the heart of Boston. Northeastern teaches worldwide experiential learning, urban engagement, and interdisciplinary research that meets global and societal needs. The University offers a range of undergraduate and graduate programs leading to degrees through the doctorate in nine colleges and schools.

Part of Beckman Research Institute of The City of Hope, Oligonucleotide Therapeutics Society, Inc. is a society that envisions a new era of oligonucleotide drugs that would change the landscape of therapeutic modalities. The company is based in San Diego, CA. Over 100 treatments are in development for common conditions, including cancer and Alzheimer’s disease, as well as rare diseases. The society offers various awards to support the accomplishments of students, postdocs, and young investigators.