Esophageal Atresia

Dive Brief:Roche is deepening its commitment to neuroscience, agreeing to a licensing deal with Ionis to develop two preclinical RNA medicines for Alzheimers and Huntingtons diseases, Ionis said Wednesday.Per deal terms, Roche is paying $60 million upfront for rights to the undisclosed drugs, and will assume responsibility for developing them once they are ready for human trials. Ionis could receive further payments if clinical, regulatory and commercial milestones for the drugs are met.Roche has invested heavily in Alzheimers drug development, advancing two antibody therapies into Phase 3 testing only to find they didnt slow disease progression. The Swiss drugmaker has fallen behind rivals Eisai, Biogen and Eli Lilly, which have benefited from recent clinical development successes.Dive Insight:Roche and Ionis have worked together for years. The two are currently collaborating on tominersen, a type of drug called an antisense oligonucleotide that blocks the expression of a protein mutated in Huntingtons disease. That drug is in a Phase 2 trial measuring biologic signs of disease as well as patient function.They also have partnered on an antisense drug Roche is testing in kidney disease and Ionis is studying as a treatment for an age-related eye disorder.In Alzheimers, Roche was among the early innovators to develop antibody drugs capable of blocking a protein called amyloid that accumulates in the brains of people with the disease. Roche hit clinical setbacks, as did Lilly, Biogen and Eisai.Now, however, Eisais Biogen-partnered drug Leqembi is approved in the U.S. and Lillys donanemab is under Food and Drug Administration review.Roche hasnt fully ended its anti-amyloid drug work yet, continuing tests of one called crenezumab in people who have an inherited form of Alzheimers. Its also still studying a formulation of the twice-failed drug gantenerumab thats designed to better reach into the brain.Other mid-stage assets include two antibody drugs that target another protein implicated in Alzheimers disease, called tau. The company recently disclosed Phase 1 data from a drug targeting another protein known as gamma secretase thats related to amyloid buildup.Huntingtons disease has proven nearly as difficult as Alzheimers to treat, with approved medicines so far only able to reduce involuntary movements. Ionis and Roche are among several companies seeking to help patients by blocking mutant proteins, although their drug tominersen has had setbacks, too.The Ionis deal signals Roche wants to keep stocking its pipeline with new approaches for treating Alzheimers and Huntingtons. Ionis demonstrated that it can successfully develop a drug for a degenerative neurological disorder when Biogen gained FDA approval for Qalsody, an Ionis-invented treatment for amyotrophic lateral sclerosis. '

HOLLISTON, Mass., Aug. 21, 2023 /PRNewswire/ -- Harvard Apparatus Regenerative Technology, Inc. (OTCQB: HRGN) ("Harvard Apparatus Regenerative Technology" or the "Company"), a clinical-stage biotechnology company developing the technology to regenerate organs inside the body to treat severe diseases, today announced the Approval of an Orphan Disease Application for its lead product, the Cellspan Esophageal Implant by the European Medicines Agency (EMA). The EMA is the centralized regulatory agency for the review and approval of new medicines in the European Union as well as Iceland, Norway and Liechtenstein. The Orphan Disease Designation (ODD) was approved to treat Esophageal Atresia, a congenital disorder where children are born with an incomplete, underdeveloped esophagus. The World-Wide incidence of Esophageal Atresia is estimated to be 1/2500 to 1/4000 live births. The designation provides Harvard Apparatus Regenerative Technology with exclusive marketing rights, as well as additional benefits related to clinical protocol development and Scientific Advice. Specifically, access to the Centralized Authorization procedure allows companies to make a single application to the European Medicines Agency, resulting in a single opinion and a single decision from the European Commission, valid in all EU Member States. Sponsor's may also have access via orphan designation to conditional approval, which is conducted under the centralized procedure. Authorized orphan medicines benefit from ten years of protection from market competition with similar medicines with similar indications once they are approved. This period of protection is extended by two years for medicines that also have complied with an agreed pediatric investigation plan granted at the time of review of the orphan medicine designation. Although the Agency does not offer research grants for sponsors of orphan medicines, funding opportunities are available from the European Commission as well as other sources, including Horizon Europe and the E-Rare ERA-NET program for rare diseases. Mr Jerry He, CEO, commented, "the ODD will expedite our efforts to bring Cellspan Esophageal Implant to the European market". Dr. William Fodor, CSO, commented, "the approval of the ODD application further supports our position as a leading developer of tissue engineered organ replacement products designed to replace, restore and regenerate new tissues in congenital organ deficiencies, as well as in tubular organs that are damaged by disease or injury." About Harvard Apparatus Regenerative Technology, Inc. We are a clinical-stage biotechnology company developing regenerative-medicine treatments for disorders of the gastro-intestinal system and other organs resulting from cancer, trauma or birth defects. Our technology is based on our proprietary cell-therapy platform that uses a patient's own stem cells to regenerate and restore function to damaged organs. We believe that our technology represents a next-generation solution for restoring organ function because it allows the patient to regenerate their own organ, thus eliminating the need for human donor or animal transplants, the sacrifice of another of the patient's own organs or permanent artificial implants. We conducted the world's first successful regeneration of the esophagus in a patient with esophageal cancer in August 2017. This surgery was performed by Dr. Dennis Wigle, Chair of Thoracic Surgery at the Mayo Clinic. The results were published in the Journal of Thoracic Oncology Clinical and Research Reports in August 2021. The procedure demonstrated that our technology was able to successfully regenerate esophageal tissue, including the mucosal lining, to restore the integrity, continuity and functionality of the esophageal tube. HRGN has 13 issued U.S. patents, 2 issued in China, 1 issued in Japan, 2 issued in Europe, 2 U.S. orphan-drug designations which can provide seven years of market exclusivity in the US market after market approval from the FDA and 1 EMA orphan drug designation, which can provide ten years of market exclusivity in the European market after market approval from the EMA. For more information, please visit and connect with the Company on LinkedIn. Forward-Looking Statements Some of the statements in this press release are "forward-looking" and are made pursuant to the safe harbor provision of the Private Securities Litigation Reform Act of 1995. These "forward-looking" statements in this press release include, but are not limited to, statements relating to the capabilities and performance of our products and product candidates; development expectations and regulatory approval of any of the Company's products, by the U.S. Food and Drug Administration, the European Medicines Agency or otherwise, which expectations or approvals may not be achieved or obtained on a timely basis or at all; and success with respect to any collaborations, clinical trials and other development and commercialization efforts of the Company's products, which such success may not be achieved or obtained on a timely basis or at all. These statements involve risks and uncertainties that may cause results to differ materially from the statements set forth in this press release, including, among other things, the Company's inability to obtain needed funds in the immediate future; the Company's ability to obtain and maintain regulatory approval for its products; plus other factors described under the heading "Item 1A. Risk Factors" in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2022 or described in the Company's other public filings. The Company's results may also be affected by factors of which the Company is not currently aware. The forward-looking statements in this press release speak only as of the date of this press release. The Company expressly disclaims any obligation or undertaking to release publicly any updates or revisions to such statements to reflect any change in its expectations with regard thereto or any changes in the events, conditions or circumstances on which any such statement is based. Investor Relations Contact Joseph Damasio Chief Financial Officer 774-233-7330 [email protected] SOURCE Harvard Apparatus Regenerative Technology, Inc.

HOLLISTON, Mass., Jan. 25, 2021 /PRNewswire/ -- Biostage, Inc. (OTCQB: BSTG) ("Biostage" or the "Company"), a bioengineering company developing next-generation esophageal implants, today announced that it appointed Herman Sanchez as an independent director to its Board of Directors. Mr. Sanchez has been working in the life sciences industry for over 20 years in various positions including designing and running randomized trial research, optimizing of clinical administration of health services, and working as a strategic consultant to the life sciences industry. He is currently a Senior Partner helping run Trinity Life Sciences' strategy consulting business. He joined Trinity over a decade ago and has worked closely with clients to support strategic decision making across the product lifecycle. In his work consulting for pharmaceutical/biotech and medical device companies he has covered several diseases/therapeutic areas including oncology, rare and ultra-rare diseases, cell therapies, cardiovascular, diabetes, alcohol abuse/dependence, neurological, orthopedic, and renal diseases. He has been published in peer-reviewed publications on various topics including renal disease, patient epidemiology, medication adherence, suicidal ideation, minority patient recruiting, alcohol use/abuse and depression/anxiety treatment. Mr. Sanchez, prior to working in the life sciences industry, earned an MBA from the Tuck School of Business at Dartmouth and an AB in Psychology from Harvard University. "We are very excited to announce the addition of the seasoned life sciences industry expert to Biostage," said Jason Chen, Chairman of Biostage. "The guidance and expertise from Mr. Sanchez will be valuable as we continue the development of our Cellspan™ platform and new product pipeline. On behalf of Biostage's Board of Directors and management team, I sincerely welcome Mr. Sanchez to our Board of Directors at this important moment of the Company. We look forward to Herman's engagement and guidance to bring our innovative treatment option to the potential patients with unmet medical needs and future commercialization." Mr. Sanchez commented, "I am excited to join the Board of Directors of Biostage and collaborate with the Board and executive team. The company has been working with a novel cell therapy to solve a critical unmet need for patients, especially kids. I hope my experience will help guide the company in developing and delivering this innovative technology to the market." About Biostage, Inc. Biostage is a bioengineering company that is developing next-generation esophageal implants. The Company's Cellspan™ technology combines a proprietary, biocompatible scaffold with a patient's own cells to create an esophageal implant that could potentially be used to treat pediatric esophageal atresia and other conditions that affect the esophagus. The Company's esophageal implant leverages the body's inherent capacity to heal itself as it is a "living tube" that facilitates regeneration of esophageal tissue and triggers a positive host response resulting in a tissue-engineered neo-conduit that restores continuity of the esophagus. These implants have the potential to dramatically improve the quality of life for children and adults. At Biostage, we believe the future of medicine has been inside us all along. For more information, please visit and connect with the Company on Twitter and LinkedIn. Forward-Looking Statements Some of the statements in this press release are "forward-looking" and are made pursuant to the safe harbor provision of the Private Securities Litigation Reform Act of 1995. These "forward-looking" statements in this press release include, but are not limited to, statements relating to our development expectations and regulatory approval of the Company's products, including those utilizing its Cellspan™ and Cellframe™ technology, which expectations or approvals may not be achieved or obtained on a timely basis or at all; or success with respect to any collaborations, clinical trials and other development and commercialization efforts of the Company's products, which such success may not be achieved or obtained on a timely basis or at all. These statements involve risks and uncertainties that may cause results to differ materially from the statements set forth in this press release, including, among other things, the Company's inability to obtain needed funds in the immediate future; the Company's ability to obtain and maintain regulatory approval for its products; plus other factors described under the heading "Item 1A. Risk Factors" in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2019 or described in the Company's other public filings. The Company's results may also be affected by factors of which the Company is not currently aware. The forward-looking statements in this press release speak only as of the date of this press release. The Company expressly disclaims any obligation or undertaking to release publicly any updates or revisions to such statements to reflect any change in its expectations with regard thereto or any changes in the events, conditions or circumstances on which any such statement is based. Investor Relations Contact Shunfu Hu Vice President of Business Development and Operations 774-233-7300 shu@biostage.com Company Codes: OTC-QB:BSTG, OTC-PINK:BSTG