Rapport Therapeutics’ IPO came with a concurrent private placement that boosted the biotech’s cash haul. The new capital will support clinical development of a lead drug with a novel approach to focal epilepsy, a form of the disorder that affects 1.8 million people in the U.S.

The post Rapport’s Stock Market Debut Reaps $154M as Novel Epilepsy Med Heads for Mid-Stage Test appeared first on MedCity News.

Rapport Therapeutics’ lead program could offer more targeted epilepsy treatment compared to currently available anti-seizure medications, which introduce many side effects. The IPO will support plans to develop the oral small molecule in focal epilepsy as well as peripheral neuropathic pain and bipolar disorder.

The post CNS-Focused Rapport Therapeutics Preps IPO to Back Phase 2-Ready Epilepsy Drug appeared first on MedCity News.

The FDA approved X4 Pharmaceuticals drug Xolremdi for treating WHIM syndrome. The molecule, which addresses the underlying cause of this rare immunodeficiency, was licensed from Sanofi’s Genzyme subsidiary.

The post Drug Licensed from Sanofi Becomes First FDA-Approved Therapy for Ultra-Rare Primary Immunodeficiency appeared first on MedCity News.

Vertex Pharmaceuticals gains a Phase 3-ready Alpine Immune Sciences drug with potential applications in a wide range of immunological disorders. The lead indication for the Alpine drug, povetacicept, is the chronic kidney disease IgA nephropathy.

The post Vertex Pharma Bolsters Immunology Profile With $4.9B Alpine Immune Sciences Acquisition appeared first on MedCity News.

The FDA approved Vertex Pharmaceuticals’ gene therapy Casgevy for treating beta thalassemia, an inherited blood disease that leads to low levels of functioning hemoglobin. Last month, the one-time treatment won its first FDA nod for treating sickle cell disease.

The post Vertex’s CRISPR Gene Therapy Lands Another FDA Nod in a Rare Blood Disease appeared first on MedCity News.

Roche is paying $50 million up front to begin a drug R&D alliance with Monte Rosa Therapeutics, a biotech whose molecular glue technology could address targets previously deemed undruggable. It’s Roche’s second such deal in the past month.

The post Roche Deal Aims to Get Molecular Glues to Stick to Elusive Cancer, Neuro Targets appeared first on MedCity News.

Precede Biosciences’ liquid biopsy platform yields insight into the genes and pathways in diseased tissue. Based on Dana-Farber Cancer Institute research, the startup aims to support biopharma drug R&D as it also develops the technology for clinical care applications.

The post Liquid Biopsy Startup Precede Sets Out to Make Precision Medicine More Precise appeared first on MedCity News.

Novo Nordisk is paying Valo Health $60 million up front to gain three preclinical cardiovascular disease programs. Milestone payments for those programs and others covered under the artificial intelligence drug discovery pact could reach up to $2.7 billion.

The post Novo Nordisk Turns to Flagship’s Valo Health for AI-Driven Cardio Drug R&D appeared first on MedCity News.

Magnet Biomedicine builds on decades of molecular glue research from its scientific co-founder, Harvard University Professor Stuart Schreiber. The startup is developing drugs that take these glues beyond the biotech industry’s current focus on targeted protein degradation.

The post Startup Magnet Biomedicine Attracts $50M to Expand Scope of Molecular Glue Drugs appeared first on MedCity News.

Intarcia Therapeutics’ GLP-1 agonist product candidate for type 2 diabetes now belongs to i2o Therapeutics, which will make the treatment’s case for approval at an upcoming FDA advisory committee meeting. The startup’s $46 million Series A financing is one of several recently announced funding rounds.

The post Twice Rejected Diabetes Drug/Device Product Gets New Chance at Startup i2o appeared first on MedCity News.

Pear Therapeutics was once regarded as a digital therapeutics pioneer, but its spiral into bankruptcy has the industry searching for the best path forward. While some favor going direct to consumers, others say the solution is in generating more robust clinical trial data to persuade hesitant payers.

The post Rigorous Data Are Key to Convince Payers, Investors in the World of Digital Therapeutics appeared first on MedCity News.

Eli Lilly’s partnership with Verve Therapeutics covers the development of a preclinical therapy addressing a protein associated with cardiovascular disease risk. The in vivo gene-editing therapy is a potential one-time treatment.

The post Eli Lilly Puts Up $60M for In Vivo Gene-Editing Med for Cardiovascular Disease appeared first on MedCity News.

Ironwood Pharmaceuticals is paying $1 billion to acquire VectivBio, a biotech whose lead program could become a blockbuster treatment for a gastrointestinal disorder. Preliminary Phase 3 data are expected by the end of 2023.

The post Ironwood Places $1B Bet on Biotech That Could Bring Its Next Blockbuster GI Drug appeared first on MedCity News.

The FDA approved Omisirge, an allogeneic cell therapy from Gamida Cell that could improve access to stem cell transplants as treatments for blood cancers. The biotech makes its therapy from umbilical cord blood.

The post How Gamida Cell’s FDA Approval Could Reduce Racial Disparities in Stem Cell Transplants appeared first on MedCity News.

Pierre Fabre is paying Scorpion Therapeutics $65 million to begin a partnership on clinical development and potential commercialization of two targeted therapies for non-small cell lung cancer. The Scorpion drugs could have safety advantages over approved Johnson & Johnson and Takeda therapies that address the same rare genetic signature.

The post Bio Startup Scorpion Partners With Pierre Fabre for Trials of Targeted Lung Cancer Drugs appeared first on MedCity News.

Vertex Pharmaceuticals aims to use CRISPR Therapeutics’ gene-editing technology to develop type 1 diabetes cell therapies that don’t prompt the immune system to reject them. This approach could compete with a Sana Biotechnology program expected to reach its first test in humans this year.

The post Vertex Pays CRISPR Therapeutics $100M to Bring Gene-Editing to Type 1 Diabetes appeared first on MedCity News.

Pear Therapeutics isn’t meeting the commercialization goals for its prescription digital therapeutics, so it’s now seeking strategic alternatives for the business. Prospective buyers can pick up Pear’s FDA-cleared products for substance use disorder, opioid use disorder, and insomnia.

The post Pear Therapeutics’ Cuts Are Not Enough; ‘Strategic Alternatives’ Among Next Steps appeared first on MedCity News.

GV led Chroma Medicine’s Series B financing round, which the biotech will apply toward epigenetic medicines that could offer advantages over other editing technologies. Other recent financings include rounds from Cargo Therapeutics and Transcend Therapeutics.

The post Aiming for an Epigenetics Edge, Chroma Raises $135M to Move Closer to the Clinic appeared first on MedCity News.

Hemab Therapeutics will apply its Series B financing toward clinical development of its lead drug candidate, a potential treatment for Glanzmann thrombasthenia. The capital will also support the rest its pipeline addressing rare bleeding and thrombotic diseases with no approved therapies.

The post Blood Clotting Biotech Hemab Hauls In $135M to Drug Rare Bleeding Disorders appeared first on MedCity News.

Chiesi drug Lamzede is an engineered version of an enzyme that’s lacking in patients with the rare disease alpha-mannosidosis. FDA approval of the Chiesi drug comes about five years after European regulators authorized the product.

The post Chiesi Wins FDA Approval for First Drug to Treat Ultra-Rare Enzyme Deficiency appeared first on MedCity News.

Karuna Therapeutics has licensed two small molecules that shuttering Goldfinch Bio had developed for kidney disorders. These compounds target a pathway that could also treat neurological disorders, giving clinical-stage Karuna the opportunity to compete against a drug candidate from Boehringer Ingelheim.

The post Karuna Picks Up Kidney Drugs for Chance to Challenge Boehringer Ingelheim in the Brain appeared first on MedCity News.

Elicio Therapeutics dipped its toe in the IPO waters but decided to go public by merging with publicly traded Angion Biomedica. Clinical-stage Elicio is developing immunotherapies, including cancer vaccines, that work by targeting immune cells located in the lymph nodes.

The post Cancer Biotech Elicio Finds Path to Public Markets Via Angion Reverse Merger appeared first on MedCity News.

In rejecting Spectrum Pharmaceuticals drug poziotinib, the FDA said the biotech needs to generate more data from another clinical trial. Instead, Spectrum is turning the company’s focus to commercializing its recently approved product for treating a common cancer complication.

The post Biotech Firm Slashes 75% of Staff After FDA Rejects Drug for Rare Lung Cancer appeared first on MedCity News.

Ascidian Therapeutics is developing therapies for inherited disorders that work by editing RNA with an approach that could offer advantages over currently available genetic medicines. The biotech’s lead program is in preclinical development for Stargardt disease, a rare eye disorder caused by multiple genetic mutations.

The post Sea creature-inspired biotech Ascidian surfaces with $50M and a new way to edit RNA appeared first on MedCity News.

Merck is paying $25 million to kick off a research partnership with Cerevance, a startup that analyzes donor brain tissue to find novel targets for new neurological disorder drugs. While Cerevance’s lead program is a Parkinson’s disease drug, Merck wants to see if the startup’s technology can help it discover and develop new Alzheimer’s disease drugs.

The post Merck turns to biotech Cerevance to feed neuro pipeline with Alzheimer’s drugs appeared first on MedCity News.

Pear Therapeutics is implementing a corporate restructuring projected to save about $28 million. The goal is to extend its available cash as the company presses on with commercialization efforts for its digital therapeutics.

The post Pear Therapeutics pares down its plans as restructuring cuts 9% of workforce appeared first on MedCity News.

Technological advances are moving drug discovery work to computers, but experimental medicines still must be tested in animals. Startup Manifold Bio is developing technology that enables the testing of hundreds of molecules in a single mouse, bringing drug hunters valuable in vivo data much earlier in the drug discovery process.

The post Harvard spinout lands $40M to bring in vivo drug discovery to cancer appeared first on MedCity News.

Vertex Pharmaceuticals already has a cell therapy candidate for type 1 diabetes but the company says acquiring rival ViaCyte will provide it with complementary assets and technologies. The two companies already share a partner in common: both have therapeutic candidates that use the gene-editing capabilities of CRISPR Therapeutics.

The post Vertex builds up pipeline of type 1 diabetes cell therapies with $320M buyout appeared first on MedCity News.

Two private equity firms have reached an $890 million deal to buy Radius Health, a company whose main asset is the commercialized osteoporosis drug Tymlos. In other M&A news, cancer biotech F-star Therapeutics is being acquired in a $161 million deal.

The post Biopharma firm Radius Health is going private in an $890M M&A deal appeared first on MedCity News.

Cell and gene therapy research has been hot the past few years but the overall market has cooled down in recent months. During the World Medical Innovation Forum in Boston, a panel of venture capitalists discussed capital formation and what biotech firms need to do to win over investors and advance their research.

The post How cell & gene therapy startups can warm up to investors in this “biotech nuclear winter” appeared first on MedCity News.