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LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> Idorsia’s Jean-Paul Clozel retires as CEO, plots next steps; CRISPR Therapeutics nabs CMO from Sanofi: Jean-Paul Clozel → Jean-Paul Clozel is ready to pass the torch at his Actelion follow-up Idorsia, but he doesn’t want to walk away from the company altogether. Clozel will retire as CEO on June 13, and Idorsia has tapped his longtime lieutenant André Muller to replace him. The 69-year-old Clozel will now stand for election as chairman, while Mathieu Simon moves into the vice chairman post. The changes on the board don’t stop there. Jörn Aldag, Felix Ehrat and Peter Kellogg are stepping down, and Idorsia has nominated ex-Galapagos president Bart Filius. “It has been an absolute privilege to serve as CEO of Actelion and Idorsia for the past 24 years. I can think of no greater endeavor than discovering new medicines that help patients lead better lives,” Clozel said in a statement. “Having devoted so much energy to Idorsia, I want to stay engaged and support the company to realize its potential.” Muller was CFO at Actelion and followed Clozel to Idorsia as finance chief after J&J bought Actelion for $30 billion in 2017. Muller had a 17-year career at Pierre Fabre, where he was CFO from 2002-11. Idorsia has notched two FDA approvals in its seven-year history: insomnia med Quviviq and high blood pressure drug Tryvio. Naimish Patel → CRISPR Therapeutics has found a new medical chief and promoted one of its execs. Naimish Patel takes over as CMO from PK Morrow, who resigned on Jan. 26 and joined Takeda as head of the oncology therapeutic area unit. Patel quickly moved up to global program head for the megablockbuster Dupixent early in his tenure at Sanofi, where he was recently the head of global development in immunology and inflammation. Vertex’s Casgevy partner has also elevated Julianne Bruno to COO. She left McKinsey for CRISPR in 2019 and had been SVP and head of programs & portfolio management since March 2023. → Bavarian Nordic will be looking for a CMO after Laurence de Moerlooze’s departure at the end of the month. The GSK and Takeda vaccines alum succeeded Chris Heery as medical chief in the spring of 2020. “We have an operational setup that is robust enough for people to continue doing their jobs until a replacement is in place,” Bavarian Nordic CEO Paul Chaplin told MedWatch about the search. “They can manage that for six months or more, so I have no concerns at all.” Hanneke Schuitemaker → Meanwhile, at another vaccine developer, Valneva has welcomed Hanneke Schuitemaker as CSO. As J&J’s global head of viral vaccine discovery and translational medicine, Schuitemaker was involved in the development of vaccine candidates for Covid-19, HIV, RSV, Ebola and more. Valneva overcame a nearly two-and-a-half-month delay and received an… #lucidquest #genetherapy #celltherapy

We are delighted to provide our first business update since delisting from AIM earlier this year and continue our journey from a discovery to an immuno-inflammation therapeutics company. We're making important progress with our small molecule drug discovery portfolio in immuno-inflammatory diseases, focusing on areas with significant unmet need and commercial potential. Our lead oral a4b7 integrin inhibitor for inflammatory bowel disease is nearing pre-clinical candidate selection, with planning for IND-enabling studies well underway. And we announce two new pipeline programmes which are progressing towards Lead Optimisation, with first-in-class and best-in-class potential. Elsewhere we are applying our PatientSeek platform to define a genetic fingerprint as a predictive biomarker for a4b7 response that will allow us to specifically design our pivotal clinical trials for success. Read more here:  https://lnkd.in/dbBTEKZx #C4XD #immuno_inflammation #biotech #innovation 

LucidQuest Strategic Insights (lqventures.com) >>> Gene&Cell Therapy >> Roche makes ADC deal with China's MediLink Therapeutics for $50M upfront: Antibody-drug conjugates, one of the creams of the crop in 2023 biopharma dealmaking, are riding into 2024 on a strong note as Roche pays $50 million upfront to collaborate with a biotech in China, where many of the ADC deals have been made recently. The Swiss Big Pharma is pairing up with Suzhou-based MediLink Therapeutics, which teamed with BioNTech last fall for $70 million upfront and with Zai Lab last April for an undisclosed amount. In the new deal, Roche’s China Innovation Center will work with MediLink (also known as Suzhou Yilian Biopharmaceutical) on the c-Met-targeted ADC candidate dubbed YL211. They’ll work together on Phase I studies and then Roche will handle all other development and marketing globally, the companies said Tuesday. The tie-up starts with $50 million in upfront and near-term payments and could grow to nearly $1 billion over time, plus tiered royalties. The partners will test YL211, which is currently in the “clinical application stage,” in solid tumors, MediLink said. The biotech’s candidate goes after c-Met, part of the receptor tyrosine kinase family, which is also targeted by ADCs like AbbVie’s Teliso-V, for which the Big Pharma plans to request accelerated approval. You get an ADC, you get an ADC: Phar­mas bite on on­col­o­gy’s buzzi­est class Tuesday’s move comes amid a flurry of deals in the ADC space, with the most recent high-profile bid coming from AbbVie’s $10.1 billion acquisition of ImmunoGen, which is expected to close in the middle of this year. GSK, Merck, Bristol Myers Squibb and Pfizer, among others, all made multiple ADC splashes last year as they try to carve their place in the hottest area of oncology R&D, which seeks to create more powerful, but more precise, versions of chemotherapy. Although Roche markets two ADCs — Kadcyla for breast cancer and Polivy for hematology — its pipeline is relatively devoid of ADC candidates. The pharma giant has been busy inking acquisitions in the inflammatory and cardiometabolic spaces in recent months with its purchases of Telavant and Carmot. The latest deal marks a small departure from Roche’s previous stance on ADCs. In June 2022, then-CEO Severin Schwan told journalists that Roche had “rather limited interest” in the field, noting it was a packed space already, Reuters reported at the time. Thomas Schinecker replaced Schwan last year and presented an updated R&D road map last September, when the company said it was “exploring different modalities,” including ADCs, allogeneic CAR-T and other treatment types. Charlie Fuchs But Charlie Fuchs, Roche and Genentech’s global head of product development for oncology and hematology, noted that what makes ADCs “decidedly interesting” is that a… #lucidquest #genetherapy #celltherapy

On the back of signing its first out-licensing deal, Roquefort Therapeutics plc (LSE:ROQ, OTCQB:ROQAF) said it is also "engaging with multiple big pharma partners" interested in its siRNA programmes in immunology and inflammation. Also known as gene silencing, siRNA, or small interfering RNA, and is a field of research that has attracted a lot of attention industrywide with some big deals inked for early-stage assets. In an update alongside interim results, Roquefort said the focus going forward will be finalising its first deal with the contract research group PDC, worth an initial $10 million. CEO Ajan Reginald told investors: "During the first half of the year we continued with our strategy to select novel medicines in high-value pharmaceutical markets and developed them to deliver the results required to attract big pharma partners. More at #Proactive #ProactiveInvestors http://ow.ly/pzyv105K4rY

FDA Issues Comprehensive Guidance for Industry and Academic Sponsors Developing Ex Vivo-Manufactured CAR T Cell Products in Oncology: Expert Recommendations for CMC, Pharmacology, and Clinical Trial Design The guide includes recommendations tailored to both autologous and allogeneic CAR T-cell products. In addition, it provides guidance on comparative analytical studies for CAR T-cell products. Although the focus is on CAR T-cell products, certain information and recommendations may also be relevant for other genetically modified lymphocyte products, such as CAR natural killer (NK) cells or T-cell receptor (TCR)-modified T-cells. It is important to note that for these related product types, further considerations beyond those suggested in this guidance may be required depending on the product and manufacturing process. https://lnkd.in/ePCkxmgh https://rea.works/

Gene&Cell Therapy >> Century Therapeutics buys Clade; Eliem announces $120M PIPE and a deal: Plus, news about Leap Therapeutics, Allay Therapeutics, Harmony Biosciences, Rallybio and CSL Behring: Century Therapeutics acquires Clade: Century is purchasing the off-the-shelf cell therapy startup for $35 million upfront in cash and stock in a deal that comes after Clade laid off employees and closed its Dutch subsidiary, as Endpoints News reported in February. Clade launched with $87 million in 2021 and was founded by CRISPR Therapeutics co-founder Chad Cowan. Century also announced that it’s expanding its lead iNK cell therapy into autoimmune disease, beginning with a form of lupus called SLE, and it raised $60 million from a private placement in light of the autoimmune expansion. — Lei Lei Wu Eliem acquires Tenet, plans to advance its lead drug: The company will now focus on TNT119, an anti-CD19 antibody designed to treat autoimmune diseases, including systemic lupus erythematosus. In conjunction with the buyout, Eliem raised $120 million in a PIPE financing, which likely gives the company runway into 2027. Most of Eliem’s board will remain, and Tenet CEO Stephen Thomas has been named interim CEO of the newly combined company. — Max Gelman Leap Therapeutics also announces a PIPE: The oncology biotech sold approximately $40 million in shares to institutional investors including Gilead, Samsara BioCapital, 683 Capital Partners, Laurion Capital Management and Rock Springs Capital. It plans to use the proceeds to fund its lead program, an anti-DKK1 antibody that it’s studying in second-line colorectal cancer. — Lei Lei Wu Allay Therapeutics’ pain drug sails through Ph2b: The biotech said ATX101 achieved sustained post-surgical pain relief for up to four weeks in patients following knee replacement. The non-opioid drug candidate also outperformed the standard of care — bupivacaine — in durability. Allay is planning to start a separate, registrational Phase 2b test later this year. — Ayisha Sharma Harmony Biosciences nabs license for OX2R agonist: Harmony will pay $25.5 million upfront to French pharma company Bioprojet to “develop, manufacture and commercialize” TPM-1116, per a press release. If everything works out, Harmony is also on the hook for up to $357.5 million in development and sales milestones. — Max Gelman MIT-founded DeepCure raises $24M: The company, which has put together about $72 million so far, is calling the raise a “Series A-1″ round. DeepCure researchers are working to develop new small-molecule drugs and identify new targets with the help of artificial intelligence. — Max Gelman Rallybio’s non-traditional deal with J&J: Johnson & Johnson is teaming up with Rallybio, which is developing a “complementary” treatment for a rare disease affecting fetuses and newborns. In addition to a $6.6 million equity… #lucidquest #genetherapy #celltherapy

Eder Therapeutics and xcube.bio have announced a strategic cooperation to facilitate the market entry and commercialization of biotech assets in Canada and Europe. The two companies will coordinate their offerings to biopharma innovators in their respective regions and leverage the numerous synergies that exist between the healthcare systems of Europe and Canada. The collaboration will specifically focus on product profiling, regulatory processes, Health Technology Assessment (HTA), and medical communication, and commercialization - ultimately benefiting patients and biopharma partners.   "Eder Therapeutics is thrilled to combine its unique depth in the Canadian market with the breadth of a European platform like xcube.bio. This partnership will enhance access to innovative treatments for Canadian patients, which is our ultimate goal," said Jared Rhines, CEO of Eder Therapeutics.   Pierre-Henri Belin, CEO of xcube.bio, stated, "xcube.bio, a European market-entry specialist, has found a valued like-minded partner in Eder Therapeutics. We look forward to expanding this cooperation to accelerate patient access and provide added value to our biopharma partners."   An initial phase of expert cooperation will begin with Eder Therapeutics' investigational gene therapy for Lipoprotein Lipase Deficiency.

We can clearly see where CRISPR is at now, but where could it be five years from now? CRISPR Therapeutics, in collaboration with Vertex Pharmaceuticals, recently gained approval for its gene-editing therapy Casgevy, targeting blood-related diseases. The $2.2 million-priced treatment, with regulatory nods in the U.K., U.S., and Bahrain, is poised to tap into a market exceeding $70 billion, positioning CRISPR for steady revenue once sales ramp up. Moving forward, CRISPR's innovative wheel is set to spin further, with potential label expansions for Casgevy and promising progress in clinical programs such as CTX112 and CTX131. Expect the company to secure approval for additional CRISPR-based gene-editing treatments, solidifying its position as a top biotech giant for the foreseeable future. #CRISPR #GeneEditing #Genomics #Biotech

ExoXpert, a CDMO launched by EXO Biologics to service increasing worldwide demand for exosomes - https://lnkd.in/gw_ad42J EXO Biologics  SA, a Belgian biotech company committed to developing biopharmaceuticals using exosomes to treat rare diseases with high unmet medical needs, today announces the worldwide launch of ExoXpertTM, a contract development and manufacturing organization (CDMO) specializing in exosomes. ExoXpert offers a MSC-based exosome manufacturing platform used in European clinical trials. ExoXpert is a wholly owned subsidiary of EXO BiologicsTM. “The potential of exosomes continues to be demonstrated across the cell and gene therapy sector. As a result, exosome-based clinical trials have the potential to significantly increase in number, if industrial supply of exosomes can be met. Currently there is a lack of exosome production, manufacturing specialists and facilities to supply exosomes for both R&D and clinical trials,” said Romain du Hecquet de Rauville, Chief Business Officer, EXO Biologics. Read more at: https://lnkd.in/gw_ad42J #exosomes #researchanddevelopment #stemcells

Eder Therapeutics and xcube.bio have announced a strategic cooperation to facilitate the market entry and commercialization of biotech assets in Canada and Europe. The two companies will coordinate their offerings to biopharma innovators in their respective regions and leverage the numerous synergies that exist between the healthcare systems of Europe and Canada. The collaboration will specifically focus on product profiling, regulatory processes, Health Technology Assessment (HTA), and medical communication, and commercialization - ultimately benefiting patients and biopharma partners.   "Eder Therapeutics is thrilled to combine its unique depth in the Canadian market with the breadth of a European platform like xcube.bio. This partnership will enhance access to innovative treatments for Canadian patients, which is our ultimate goal," said Jared Rhines, CEO of Eder Therapeutics.   Pierre-Henri Belin, CEO of xcube.bio, stated, "xcube.bio, a European market-entry specialist, has found a valued like-minded partner in Eder Therapeutics. We look forward to expanding this cooperation to accelerate patient access and provide added value to our biopharma partners."   An initial phase of expert cooperation will begin with Eder Therapeutics' investigational gene therapy for Lipoprotein Lipase Deficiency. #biopharma #partnering #commercialization