SAN CARLOS, CA, March 4, 2024 -- Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, announced the presentation of summary findings from its ongoing Phase 2 clinical study (GLM101-002) in adult patients with PMM2-CDG at the Rare Disease Day 2024 Symposium, CDG Scientific and Family Conference, that took place March 1-3 in San Diego.
Glycomine Announces Encouraging Efficacy Data from Ongoing Phase 2 Clinical Study in PMM2-CDG
Aura Biosciences Announces First Patient Dosed in Global Phase 3 CoMpass Trial
BOSTON--Dec. 7, 2023-- Aura Biosciences Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the first patient has been dosed in the global Phase 3 CoMpass trial evaluating the safety and efficacy of bel-sar for the first-line treatment of adult patients with early-stage choroidal melanoma.
Minoryx announces enrollment of first patients with cerebral Adrenoleukodystrophy (cALD) in US Phase 3 clinical trial, CALYX
Mataró, Barcelona, Spain, November 16, 2023 - Minoryx Therapeutics, a registration stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces the first patients have been enrolled in its US Phase 3 clinical trial (CALYX) of lead candidate leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD), an orphan indication with no alternative therapeutic options.
Tune Therapeutics Reveals Epigenetic Editing Program Targeting Hepatitis B Virus
DURHAM, N.C. & SEATTLE-- November 13, 2023--Pioneering epigenome editing company Tune Therapeutics has announced it is working on a new and potentially curative approach to treating chronic HBV infection. The program was unveiled by Dr. Ed Gane, Professor of Medicine at the University of Auckland and world-renowned authority on Hepatitis B Virus (HBV) at the American Association for the Study of Liver Diseases (AASLD) conference in Boston, MA on November 11th. The program, described by Gane at a session on emerging HBV treatments, aims to utilize TEMPO –Tune’s proprietary ‘genetic tuning’ platform – to achieve functional cure through the permanent, epigenetic repression of viral activity.
Aura Biosciences Receives FDA Agreement Under Special Protocol Assessment (SPA) for CoMpass Phase 3 Clinical Trial of Belzupacap Sarotalocan (Bel-sar) in Early-stage Choroidal Melanoma
BOSTON--Aura Biosciences, Inc. (NASDAQ: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced it has received agreement from the U.S. Food and Drug Administration (FDA) under an SPA for the design and planned analysis of CoMpass, the Company’s global Phase 3 clinical trial of bel-sar for the first-line treatment of adult patients with early-stage choroidal melanoma (CM). The Company also announced the presentation of positive Phase 2 safety and efficacy data of bel-sar with 90% of patients at twelve months of follow-up evaluating two key clinical endpoints: tumor control and visual acuity preservation using suprachoroidal (SC) route of administration for the first-line treatment of adult patients with early-stage CM. The results were presented at the American Academy of Ophthalmology (AAO) 2023 Annual Meeting in San Francisco, California.
Nick Pappas Joins Pappas Capital as Managing Partner
RESEARCH TRIANGLE PARK, N.C., Sept. 19, 2023 -- Pappas Capital, a leading investor in next-generation life science and technology companies, announced today that Nick Pappas (no relation) has joined the firm as a Managing Partner of Pappas Ventures, the firm's life science venture business. In his role, Nick will oversee Pappas Ventures and lead fundraising and investment efforts for its current and future venture funds. Previously, Nick served as a Managing Partner and Head of Investments at Philips Ventures, where he led a global investment program that was recognized as a leader in corporate venture capital.
Us2.ai Partners with Leading Academic Medical Center to Collaborate on AI-Enabled Echocardiography Software
SINGAPORE--September 13, 2023--Us2.ai, a Singapore-based MedTech firm, has partnered with Duke University to co-develop and commercialize AI tools for echocardiography, to enable earlier detection, improved diagnosis and more efficient management of heart disease.
Mirum Pharmaceuticals Enters Agreement to Acquire Bile Acid Product Portfolio for the Treatment of Rare Liver Diseases from Travere Therapeutics
FOSTER CITY, Calif. & SAN DIEGO--July 18, 2023--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) and Travere Therapeutics, Inc. (NASDAQ: TVTX) today announced that they have entered into a definitive agreement for the sale of Travere’s bile acid product portfolio that includes Cholbam® (cholic acid) and Chenodal® (chenodiol), two medications addressing rare diseases in high-need settings.
Veralox Secures $24MM Financing to Advance Development of First-in-Class Therapies for Immune-Mediated Diseases
FREDERICK, MD., June 20, 2023 -- Veralox Therapeutics, a clinical-stage biotechnology company developing a new class of therapies targeting the 12-lipoxygenase (12-LOX) pathway to address some of medicine’s most persistent and serious immune-mediated diseases, today announced the appointment of Jonathan Mow as the company’s new chief executive officer.
Mr. Mow’s appointment comes as Veralox secured $24 million in funding to advance VLX-1005 through a Phase 2a proof-of-concept study evaluating its impact on heparin-induced thromobcytopenia (HIT), a life-threatening rare disease caused by an aberrant immune response to heparin exposure. The investment round included new investors Pappas Capital and NYBC Ventures and existing investors Hatteras Venture Partners, Sanofi Ventures, JDRF T1D Fund and Genesys Capital, amongst others. In conjunction with the financing, the company welcomes Peter Young of Pappas Capital as a director and Meg Wood of NYBC Ventures as an observer.
Minoryx gains FDA approval to initiate a Phase 3 clinical trial in patients with cerebral Adrenoleukodystrophy
Mataró, Barcelona, Spain, May 31, 2023 - Minoryx Therapeutics, a registration stage biotech company focused on the development of therapies for orphan central nervous system (CNS) disorders, today announces the US Food and Drug Administration (FDA) approval of its Phase 3 clinical trial (CALYX) of lead candidate leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD).
The CALYX protocol has received both FDA and central IRB approval. Significant preparations for the trial commencement have been completed and patient recruitment is expected to start by the end of Q2 2023 with results anticipated by late 2025.
