BiovelocITA brings together best-in-class scientists, entrepreneurs and investors to foster the development of pioneering companies. BiovelocITA is co-founded and promoted by Sofinnova Partners, a historical player on the Italian biotech market. Active in Italy for more than fifteen years, Sofinnova Partners is in a privileged position to help Italy grow into one of Europe’s strongest biotech market.

BiovelocITA

Trentino Invest Srl

Sofinnova Partners is a venture capital firm that invests in the life sciences sector, from seed to later-stage. The firm manages over €2 billion of assets dedicated to life science investing and actively partners with ambitious entrepreneurs as a lead or cornerstone investor to develop transformative innovations that have the potential to positively impact its collective future. It invests in start-ups, early-stage companies, corporate spin-offs, and occasionally turnaround situations with a focus on Life Sciences (Biopharmaceuticals / Biotech, Medical Devices, Industrial Biotechnology). Sofinnova Partners proactively sources deals, takes a lead role, is most often the first institutional investor in Round A financings, and leads its portfolio companies until exit. They sit on the boards of their portfolio companies and play an active role from the formation phase alongside entrepreneurs. It was headquartered in Paris, Ile-de-France in 1972.

Sofinnova Partners

Indaco Venture Partners SGR is the Italian venture capital asset management company, with a strong specialization in companies that innovate in electronics and robotics, medtech, digital, and new materials. The company focused on growth-stage companies whose competitive advantage stems from cutting-edge proprietary technologies and innovations. In terms of teams and technologies, the focus is on Italian enterprises, although investments are made throughout Europe, the USA, Canada, and Israel. Indaco Venture Partners was founded in 2018 and is headquartered in Milan, Lombardia, Italy.

Indaco Venture Partners

Alia Therapeutics

Alia Therapeutics is developing next-generation gene-editing medicines to cure rare genetic diseases.

<div>Italian biotech startup Alia Therapeutics, founded in October 2018, is a spin-off of the University of Trento. The company focuses on R&amp;D of&nbsp; therapeutic approaches based on gene-editing techniques for incurable genetic diseases.&nbsp;</div>

Reinventing gene therapies with next-generation technologies

Human Gene Editing

Alia Therapeutics–Nada A: board, 202305– independent Board Director main org CEO + Co-Founder at GentiBio

<div>
 
 Italian biotech startup Alia Therapeutics, founded in October 2018, is a spin-off of the University of Trento. The company focuses on R&amp;D of&nbsp; therapeutic approaches based on gene-editing techniques for incurable genetic diseases.&nbsp;
 
 
 
 
 The company's main discovery is a genome-editing tool called &ldquo;evo-Cas9,&rdquo; which was found using yeast screening. The company has a non-exclusive license for this technology with Intellia Therapeutics.&nbsp;
 
 
 
 
 The company also addresses autosomal dominant disorders by focusing on single nucleotide polymorphisms (SNPs) and specific alleles, regardless of the underlying genetic mutations.&nbsp;
 
 
 
 
 Funding and financials&nbsp;
 
 
 
 In April 2023, the company raised EUR 4.4 million (USD 4.7 million) in a 
 <a href="https://sofinnovapartners.com/news/alia-therapeutics-secures-eur4-4-million-seed-financing-extension-led-by-sofinnova-partners">
 seed funding
 </a>
 round led by Sofinnova Partners. The funds were used to strengthen Alia's gene-editing technology and support the company's expansion.
 
</div>

<div>
 
 Editas Medicine specializes in gene editing to develop novel therapeutics for a variety of areas including oncology, ocular diseases, and blood diseases. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 and Cas12a enzymes.&nbsp;
 
 
 The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of March 2024, Editas has seven gene editing programs in its pipeline.&nbsp;
 
 
 One of the company&rsquo;s candidates was in the late clinical trial stages as of March 2024. The Phase I/II clinical trials on Editas&rsquo; in vivo gene editing therapy EDIT-101 have been ongoing since 2019 and the company announced that it had achieved proof of concept and identified a responder population in November 2022. For the first time ever (in April 2022), the company delivered its experimental in vivo CRISPR therapy to a 
 <a href="https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-dosing-first-pediatric-patient">
 pediatric patient
 </a>
 to treat Leber congenital amaurosis 10 (LCA10), an inherited early childhood eye disease. Its ex vivo gene editing therapy EDIT-301 holds two rare pediatric disease designations granted by the FDA for transfusion-dependent beta-thalassemia and sickle cell disease. The candidate also received 
 <a href="https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-receives-fda-orphan-drug-designation-edit-301">
 orphan drug designation
 </a>
 in May 2022 for beta-thalassemia.
 
 
 Key customers and partnerships
 
 
 Founded in September 2013, Editas has secured several collaborations with other pharmaceutical and drug developers including 
 <a href="https://www.catalent.com/catalent-news/catalent-enters-into-strategic-partnership-with-editas-medicine-to-support-gene-editing-medicine-pipeline/">
 Catalent
 </a>
 (for therapy development and manufacturing support),
 <a href="https://ir.editasmedicine.com/news-releases/news-release-details/adverum-biotechnologies-and-editas-medicine-announce">
 Adverum Biotechnologies 
 </a>
 (to develop technologies to deliver gene editing medicines to the eye), 
 <a href="https://www.askbio.com/editas-medicine-and-askbio-enter-strategic-research-collaboration-to-explore-in-vivo-delivery-of-genome-editing-medicines-to-treat-neurological-diseases/">
 Asklepios BioPharmaceutical
 </a>
 (AskBio) (to research in vivo gene editing therapies for neurological diseases),
 <a href="https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-and-bluerock-therapeutics-enter-strategic">
 BlueRock Therapeutics
 </a>
 (to develop medicines by combining cell therapy and gene editing technologies), and big pharma such as Bristol-Myers Squibb (to develop cell therapies for oncology).&nbsp;
 
 
 The company expanded its partnership with Azzur Cleanrooms on Demand (COD) in July 2023 with regard to continuing to manufacture Editas' candidate EDIT-301. 
 
 
 Funding and financials
 
 
 The company last raised funds in June 2023 when the company raised USD 125 million in a common stock offering. Editas Medicine did not disclose what the raised funds would be used for. The company has been listed on the Nasdaq since February 2016 and was the first CRISPR gene editing company to go public. 
 
</div>

Editas Medicine

<div>
 
 CRISPR Therapeutics specializes in developing gene editing-based novel therapeutics for a variety of areas including immuno-oncology and hemoglobinopathies. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 enzymes, and focuses predominantly on treating somatic cells that do not transfer DNA to the next generations, preventing future generations from contracting a genetic disease.&nbsp;
 
 
 The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of March 2024, the company had 18 drug candidates in its pipeline. 
 
 
 In a milestone event in November 2023, the company&rsquo;s gene-editing therapy exagamglogene autotemcel, CASGEVY 
 <a href="https://sp-edge.com/updates/24093">
 (exa-cel)
 </a>
 , developed with Vertex Pharmaceuticals to treat sickle-cell disease and beta-thalassemia, received the world&rsquo;s first authorization from the UK's Medicines and Healthcare products, Regulatory Agency (MHRA). In December 2023, it received FDA approval for patients aged 12 and older. The drug for beta-thalassemia patients is also nearing its market entry in the US, with the FDA&rsquo;s decision expected on March 30, 2024.&nbsp;
 
 
 
 
 
 Key customers and partnerships
 
 
 
 
 The company has secured strategic collaborations with several companies to co-develop and co-commercialize gene editing medicines that include 
 <a href="https://investors.vrtx.com/news-releases/news-release-details/vertex-pharmaceuticals-and-crispr-therapeutics-amend">
 Vertex Pharmaceuticals
 </a>
 (2015) to develop a therapy (CTX001) for various indications including beta-thalassemia, sickle cell disease, and Duchenne muscular dystrophy; and 
 <a href="https://viacyte.com/press-releases/crispr-therapeutics-and-viacyte-announce-strategic-collaboration-to-develop-gene-edited-stem-cell-derived-therapy-for-diabetes/">
 ViaCyte
 </a>
 (September 2018) to develop VCTX210, a regenerative medicine for diabetes.
 
 
 Big pharma Bayer also signed an option agreement in 2019 with the company to co-develop and co-commercialize two candidates, after entirely handing over the joint venture Casebia Therapeutics to CRISPR.&nbsp;&nbsp;
 
 
 
 
 
 Funding and financials
 
 
 
 
 Founded in 2013, CRISPR has been listed on the Nasdaq since 2016. For 2021, the company reported a revenue of USD 913.1 million earned via milestone payments, collaborations, and licensing.
 
 
 
 
 
</div>

CRISPR Therapeutics

<div>
 
 Intellia Therapeutics specializes in gene editing to develop novel therapeutics for various areas, including immuno-oncology, autoimmune, and genetic diseases. It leverages the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology, specifically Cas9 enzymes.&nbsp;
 
 
 The company develops both in vivo (gene editing done inside the body) and ex vivo (gene editing done in a specialized laboratory and returned to the body) therapeutics. As of March 2024, it was working on 3 internal drug programs and four collaborative programs. As of March 2024, one program was in the late clinical stage, one program was in the early clinical stage, and five were in the research and preclinical stage. The company considers its lipid nanoparticle delivery system to be a key strength in minimizing genotoxicity risk and random insertion.
 
 
 Intellia acquired biotechnology company Rewrite Therapeutics for USD 45 million in February 2022 to leverage its gene editing and DNA-writing technologies. Intellia would also pay up to USD 155 million in additional milestone payments.
 
 
 
 
 
 Key customers and partnerships
 
 
 Intellia has partnered with a number of biotech and pharma companies, including: 
 1) Big pharma Novartis (January 2015), to research and co-develop CRISPR/Cas9-edited therapies to develop an ex-vivo treatment for sickle-cell disease and a number of undisclosed programs 
 2) Kyverna Therapeutics (January 2022), for the development of a next-generation CD19 CAR T-cell therapy for autoimmune diseases 
 3) ONK Therapeutics (February 2022), to develop engineered natural killer (NK) cell therapies for cancer treatment.&nbsp;
 
 
 In October 2023, the company expanded its research collaboration with 
 <a href="https://sp-edge.com/updates/22377">
 Regeneron Pharmaceuticals
 </a>
 (entered in April 2020) to develop gene-editing therapies for neurological and muscular diseases.
 
 
 
 
 
 Funding and financials
 
 
 Founded by Caribou Biosciences and Atlas Ventures in November 2014, Intellia has been listed on Nasdaq since May 2016. In 
 <a href="https://sp-edge.com/updates/15140">
 November 2022
 </a>
 , the company raised USD 300 million via an underwritten public offering&mdash;it did not disclose the purpose of the financing. For 2022, the company reported a revenue of ~USD 52&nbsp; million, earned through collaborations.
 
</div>

Intellia Therapeutics

<div>
 
 Based in the US and France, Cellectis develops off-the-shelf gene editing cancer immunotherapies based on allogeneic Chimeric Antigen Receptors T (CAR-T) cells dubbed UCARTs by leveraging the transcription activator-like effector nucleases (TALEN) technology.
 
 
 As of March 2024, the company had seven therapeutics in development; two targeting Leukemia, one for the treatment of non-Hodgkin lymphoma, one for Large B Cell Lymphoma, two for Multiple Myelnoma, and one for Renal Cell Carcinoma. Its Leukemia therapeutics were in Phase I of clinical trials, while its non-Hodgkin lymphoma solution was just entering Phase I of clinical trials. The company's therapeutics for Multiple Myeloma, another for Lymphoma, and Renal Cell Carcinoma, had been licensed out to other pharmaceutical companies.&nbsp;
 
 
 
 
 Key customers and partnerships
 
 
 In 
 <a href="https://sp-edge.com/updates/23389">
 November 2023
 </a>
 , Cellectis entered into a partnership with AstraZeneca to accelerate the development of gene editing therapeutics, with a focus on oncology, immunology, and rare diseases.&nbsp;
 
 
 Cellectis also partnered with Primera in January 2023 to develop in-vivo gene therapies for mitochondrial disease. Through the partnership, both entities would collaborate to genetically edit mutations in mitochondrial DNA and develop an engineering toolbox that can be used to develop therapeutics for mitochondrial diseases. 
 
 
 
 
 
 
 Funding and financials
 
 
 The company entered into several strategic collaboration agreements with AstraZeneca in November 2023. These agreements included an investment agreement relating to an initial equity investment of USD 80 million by subscribing for 16 million ordinary shares. Cellectis stated that it planned to use the proceeds received to develop gene editing tools, for research and development expenses incurred in developing its programs, and other general corporate purposes.
 
 
 
</div>

Cellectis

Caribou Biosciences specializes in gene editing to develop novel therapeutics for hematologic oncology indications using the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology. It develops allogeneic off-the-shelf gene edited therapies based on Chimeric Antigen Receptors T (CAR-T) cells and CAR Natural Killer (CAR-NK) cells.The company leverages its proprietary platform CRISPR hybrid RNA-DNA (chRDNA), which uses hybrid guides—both ribonucleic acid (RNA) and DNA nucleotides—to edit DNA. As of March 2024, Caribou had four candidates in its clinical pipeline, of which its lead candidate CB-010, a CAR-T therapy to treat relapsed/refractory B cell non-Hodgkin lymphoma, was in the Phase I clinical trial stage (March 2024). Its other CAR-T candidates, CB-011 for the treatment of relapsed/refractory Multiple Myeloma and CB-012, for the treatment of relapsed/refractory Acute Myeloid Leukemia, were also in Phase I of clinical trials. Its alternate application for CB-010 to treat autoimmune diseases was still in the discovery stage.The company intended to extend ex vivo (gene editing done in a specialized laboratory and returned to the body) therapies toward other indications outside oncology and implement its Cas12a chRDNA platform in in vivo (gene editing done inside the body) applications.Key customers and partnershipsThe company entered into a partnership with big pharma company <a href="https://news.abbvie.com/news/press-releases/abbvie-and-caribou-biosciences-announce-collaboration-and-license-agreement-for-car-t-cell-products.htm">Abbvie </a>in February 2021 for licensing and co-development of two CAR-T therapies. Caribou also entered into licensing agreements with <a href="https://www.businesswire.com/news/home/20201118005128/en/Caribou-Biosciences-Announces-Licensing-Agreement-for-scFvs-Targeting-CD371-to-Enable-Development-of-Allogeneic-Cell-Therapies">Memorial Sloan Kettering Cancer Center </a>(MSKCC) (November 2020), Corteva Agriscience, and Intellia Therapeutics. It also has a sales and assignment agreement with ProMab Biotechnologies Inc. (February 2020).Funding and financialsCaribou Biosciences went public on the Nasdaq Global Select Market in July 2021 and raised USD 304 million. The IPO proceeds were earmarked to fuel the company’s clinical development. For 2021, it recorded revenue of USD 9.6 million earned through licensing and collaborations.The company last raised funding in July 2023, when it raised USD 125 million in an upsized underwritten public offering. Caribou did not disclose what the funds would be used for.

Caribou Biosciences

Companies that develop novel therapeutics based on the CRISPR/Cas9 gene-editing technology

Companies that develop novel therapeutics based on other types of gene editing technologies including TALEN and ZFN

Companies that develop systems that enable the diagnosis of gene-edited therapies. Also includes systems that deliver gene-editing components into the human body

Companies that offer their gene-editing platforms to other pharma companies to develop therapeutics

Companies that offer support services to gene-editing companies, which include genomic research companies, gene-editing component manufacturing, and designing tools

Startups in the hub develop gene-editing treatments to cater to specific patients and patient profiles

Genomics enables precise changes in the DNA sequence of living organisms to develop treatments

Alia Therapeutics

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